BC Treatment & Clinical Trials Archives

When it comes to breast cancer treatment, patients and their care partners have much to consider. Some people may seek breast cancer treatment and clinical trials, others may have few feasible options. Understanding treatment options, goals, and what to expect are vital to achieving the best possible outcome for you.

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When Is a Full Mastectomy Appropriate for Metastatic Breast Cancer Patients?

When Is a Full Mastectomy Appropriate for Metastatic Breast Cancer Patients? from Patient Empowerment Network on Vimeo

Dr. Stephanie Valente discusses mastectomy for metastatic breast cancer patients, including common misconceptions around breast cancer surgery.

Dr. Stephanie Valente is the Director of the Breast Surgery Fellowship Program at Cleveland Clinic. More about this expert here.

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Transcript:

Dr. Valente:                

So, there are a lot of reasons that a woman undergoes a mastectomy. The first one is choice. So, anytime somebody is diagnosed with breast cancer, they actually have the choice of whether or not they want to remove their whole breast. So, even if their cancer is small, they do have the option of removing the whole breast. If the cancer is smaller, they might have the option to save the breast, which is called a lumpectomy.

Sometimes cancer is found, and it’s a little bit more advanced where saving the breast is not an option. So, the cancer is larger than a lumpectomy would allow. And sometimes that’s what’s called the extent of disease. So, the amount of breast tissue that’s involved requires a majority of the portion of the breast to be removed.

So, just because a woman has breast cancer that’s made its way out of the breast, into the lymph nodes, or beyond – so, metastatic cancer – doesn’t necessarily mean that she needs a mastectomy. So, just because you’ve got metastatic cancer doesn’t necessarily mean that the breast needs to be completely removed.

So, I think that one of the biggest misconceptions is that the more aggressive somebody is with their surgery, the better their chances with survival.

And again, taking a step back and saying you can choose a more aggressive surgery, but a more aggressive surgery doesn’t necessarily mean it gets you out of chemotherapy or it gets you out of radiation therapy. Those things are recommended, independent of a woman’s choice for the type of surgery that she may or may not pick.

Insist! Metastatic Breast Cancer Resource Guide

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Metastatic Breast Cancer: Accessing the Best Treatment For YOU

Metastatic Breast Cancer: Accessing the Best Treatment For YOU from Patient Empowerment Network on Vimeo.

How could genetic testing results impact your metastatic breast cancer treatment options? In this INSIST! Breast Cancer webinar, Dr. Julie Gralow discusses essential testing, the latest targeted therapies and emerging breast cancer research.

Dr. Julie Gralow is the Jill Bennett Endowed Professor of Breast Medical Oncology at the University of Washington, Fred Hutchinson Cancer Research Center, and the Seattle Cancer Care Alliance.

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Transcript:

Katherine:

Welcome to Insist Breast Cancer, a program focused on empowering patients to take an active role and insist on better care. Today, we’ll discuss the latest advances in metastatic breast cancer, including the role of genetic testing and how this may affect treatment options. I’m Katherine Banwell, your host for today’s program, and joining me is Dr. Julie Gralow. Welcome, Dr. Gralow. Would you introduce yourself?

Dr. Gralow:   

Hi, thanks, Katherine. I’m Dr. Julie Gralow. I’m the Jill Bennett Endowed Professor of Breast Medical Oncology at the University of Washington, Fred Hutchinson Cancer Research Center, and the Seattle Cancer Care Alliance.

Katherine:    

Excellent, thank you. Before we begin the discussion, a reminder that this program is not a substitute for seeking medical advice. Please refer to your own healthcare team. Well, Dr. Gralow, let’s start by helping people understand how breast cancer is staged. Could we go through those stages?

Dr. Gralow:     

The staging of breast cancer has traditionally been by something we call anatomic staging, which has the tumor size, the number of local lymph nodes involved, and whether it has metastasized beyond the lymph nodes. So, that’s TNM – tumor, nodes, metastases. And so, that’s the classic staging, and based on combinations of those things, you can be a Stage 0 through Stage 4. Stage 0 is reserved for ductal carcinoma in situ, which is a noninvasive breast cancer that can’t generally spread beyond the breast, so that’s Stage 0, and then we go up for invasive cancer.

Interestingly, just a couple years ago, the big group that oversees the staging of cancers decided that in breast cancer, that TNM – the size, the lymph nodes, and the location beyond the lymph nodes – is not good enough anymore, so they came up with a proposal for what we call a clinical prognostic stage, which is a companion to the traditional TNM staging.

What they were getting at here was it’s not just how big your cancer is, how many lymph nodes, or whatever, it’s also at the biology of your cancer. So, this new clinical prognostic stage takes into account the estrogen and progesterone receptor of your cancer, the HER2 receptor at the grade, which is a degree of aggressiveness, and then, if your tumor qualifies, one of the newer genomic testing profiles that we use in earlier-stage breast cancer, such as the Oncotype DX 21-gene recurrence score or the MammaPrint 70-gene assay.

So, all of that goes into account now, and the whole point here is that the estrogen receptor, the HER2, the grade, and some of these genomics may actually make more difference than how many lymph nodes you have, where the cancer is, and how big it is, so it’s not just the size, but also the biology of the cancer that we’re trying to include in the new staging systems.

Katherine:    

In this program, Dr. Gralow, we ’re focusing on metastatic breast cancer. Would you explain when breast cancer is considered to have metastasized?

Dr. Gralow:  

That’s a great question because technically, if the lymph nodes in the armpit – the axillary area – are involved, that does represent spread beyond the breast, but if it stays in the local lymph node areas, it’s not technically called a metastatic or Stage 4 breast cancer. So, metastatic breast cancer would have traveled beyond the breast and those local lymph nodes, and some common sites would be to the bone, to the lungs, to the liver, less commonly – at least, up front – to the brain, and it could also travel to other lymph node groups beyond those just in the armpit and the local chest wall area as well.

Katherine:   

What about subtypes? How are they determined?

Dr. Gralow:   

The main way that we subtype breast cancer right now is based on the expression of estrogen and progesterone receptor, the two hormone receptors, and the HER2 receptor, the human epidermal growth factor receptor. So, to date, those are the most important features when we subtype, and so, a tumor can either express estrogen and progesterone receptor or not, and it can overexpress or amplify HER2 or not, and if you think that through, you can come up with four different major subtypes, in a way, based on estrogen receptor positive or negative and HER2 positive or negative.

When all three of those are negative, we call that triple negative breast cancer, and that’s about 18-20% of all breast cancers as diagnosed in the U.S. And then, when all three are positive, we sometimes call it triple positive, and the reason that we subtype is because we know that those different subsets act differently and that we have different drugs to treat them with, and we’ve got great drugs in the categories of hormone receptor positive and HER2 positive, and increasingly, some recently hope in a new drug approval or two in triple negative breast cancer as well.

Katherine:     

For a patient to get diagnosed, what are the essential tests?

Dr. Gralow:  

So, we’re talking about metastatic breast cancer here, and in the U.S., maybe up to 10% or slightly less of breast cancer is technically Stage 4 or metastatic at diagnosis. That means at the time we first found it in the breast, it had already spread beyond. So, an important thing that we’ll do with a newly diagnosed breast cancer is especially if there are a lot of lymph nodes are involved or the patient has symptoms that might say there’s something in the bone, liver, or lung is staging.

So, we’ll use scans – maybe a CAT scan, bone scan, or PET scan – and we will look at whether the disease has gone beyond the breast and the lymph nodes, and if so, where. So, maybe 8-10% of breast cancer diagnosed in the U.S. already has some evidence that it has spread beyond the breast, but the most common way that metastatic breast cancer happens is that a patient was diagnosed possibly years and years ago, treated in the early-stage setting, and now it comes back, and that is the most common presentation for metastatic breast cancer, and sometimes that can be due to symptoms.

As I said, if it comes back in the bone, maybe that’s bone pain. If it’s in the lung, it’s a cough. There are symptoms. Sometimes, it’s because we’ve done a blood test or something and we find some changes there.

And so, when a breast cancer has recurred, it’s really important to document that it’s really breast cancer coming back, first of all, and so, if we can, we generally want a biopsy, and we want to stick a needle in it if it’s safe to do, and look and verify that it looks like breast cancer, and also, it’s really important that we repeat all those receptors that we talked about from the beginning because it can change.

So, a cancer up front 10 years ago could have been positive for estrogen receptor, but the only cells that survived – mutated, changed – were estrogen receptor negative, so what comes back could be different. So, it’s really critical to get that biopsy, repeat the estrogen/progesterone receptor and HER2, and also, in an ideal world, now that it’s 2020 and we’re moving more toward genomics, to do a full genomic profile and look for other changes and mutations that could drive our therapeutic options.

So, staging, knowing where the cancer is, getting a good baseline by understanding where it is and how big it is so that we can follow it and hopefully see that it’s responding to treatment, and then, repeating all of the biology components so that we know what the best options are for treatment are really critical.

Katherine:  

Right. How can patients advocate for a precise breast cancer diagnosis, and why is that important?

Dr. Gralow:    

Well, all those things I just mentioned are key. Knowing exactly where it is so that we can monitor it – for example, if the cancer has come back in the bones, we would add what we call a bone modifying agent, a drug like zoledronic acid or denosumab – Zometa or – which can suppress bone destruction from the cancer, but if it’s not in the bone, we wouldn’t add that.

And, we want to have a good look everywhere so that we can see if it’s responding because sometimes, the tumor can respond differently in one area than another. Also, I think it’s really important to know what your treatment options are by doing that biopsy, getting a full panel, and looking at potentially hundreds of genes that could be mutated, deleted, or amplified so that we know what our treatment options are.

And, we’re not going to use all the treatment options up front, so it’s helpful for knowing that if this treatment doesn’t work or is too toxic, what are the second-line or third-line options? So, we make sure that there’s what we call good staging up front so we know where the cancer is, and then we make sure that we’ve looked at it as best we can in 2020 with all the genomics.

That would give us the best chance of being tailored – individualized – to the tumor. Sometimes, if we can’t biopsy it, like with a needle that would go into a liver spot, then increasingly, we’re looking at what we call liquid biopsies, and that can be drawing the blood and seeing if we can find parts of the tumor, whether it be the DNA or the RNA that’s floating around in the blood, and sometimes we can get that information out of the blood as well.

Katherine: 

All right. Dr. Gralow, when you meet with patients, what are some of the more common misconceptions that you hear related to diagnosis?

Dr. Gralow:  

Well, I think people do confuse – especially at an early diagnosis – that the metastases, the travel to the local lymph nodes, is not the same as a metastatic breast cancer, so we spend some time talking about how it’s still curable and not considered a distant metastasis if the lymph nodes are in the armpit or up above the collarbone, and so, that’s something that we spend some time talking about.

This whole term of “metastatic recurrence” – unfortunately, when you start looking online and get your information from Dr. Google, you read right away that it’s no longer curable, and in 2020, yes, that’s true. That’s probably the most specific statement that we can make. We are not going with curative intent, which means we treat for a defined amount of time, and then all the disease goes away, and we stop treatment, and then you go on with your life, and it never comes back. That would be cure.

But, I think it’s really important to point out that much of metastatic breast cancer can be highly treatable, and what we hope to do – and certainly, at least a subset of metastatic breast cancer – we want to convert it more to what we would call a chronic disease, and so, think of it more like hypertension, high blood pressure, or diabetes. These are diseases that we generally don’t cure with treatment, but that we can control with drug therapy, which sometimes has to be adjusted, and if we don’t control it, we can get some bad complications.

So, that’s not all metastatic breast cancer, unfortunately – we can’t convert all of it do something where we can use a therapy for a long time that keeps it in check and where you have a pretty good quality of life – but we’re hoping that more and more, we’re getting targeted therapies and more specific treatments to patients so that we can convert more patients to a more chronic kind of situation.

Katherine:

So, it’s something that patients live with.

Dr. Gralow:  

Right.

Katherine:  

Many people are confused about genetic testing. They often think that it relates to ancestry or physical traits like hair and eye color. What’s the role of genetic testing in breast cancer?

Dr. Gralow:    

Well, you can do genetic testing of the patient’s inheritance, which is how most people think of genetic testing, and that’s actually really important and increasingly important in metastatic breast cancer to do your own inheritance. Have you inherited a gene that was associated with how your cancer developed? Because now, we actually have a class of drugs called PARP inhibitors that are approved for tumors that have a BRCA1 or BRCA2 mutation with them. Most of those mutations were inherited, but not all. Sometimes they can develop as well.

So, now, when my patient – if she didn’t previously have genetic testing for an inherited risk for breast cancer either coming from mom or dad’s side of the family, a lot of people do have that up front, especially if they’re younger at diagnosis or they have a lot of family members with breast cancer. If she didn’t have that genetic testing done previously, at the time of the metastatic occurrence, I’m going to recommend that that be done because knowing if the cancer is associated with one of these DNA repair genes – BRCA1, BRCA2, some other genes – we have a new treatment option, which is an oral pill that actually is highly effective if the tumor has a mutation in one of these.

But, we can also – so, that’s genetic testing of the patient’s own DNA, but we can also do what we call genetic testing – or genomic testing, if you will – of the genes of the cancer. What were the changes in the DNA at the gene level that caused a normal breast cell over time to develop into a cancer cell that’s now growing without responding to our body’s checks and balances? So, what were those mutations, deletions, or amplifications in the tumor itself?

So, we’ve got the patient’s genetics, we’ve got the tumor’s genetics, and both of those come into play when we’re making our best treatment recommendations and trying to understand what the right approach is.

Katherine:       

How is testing administered?

Dr. Gralow: 

So, for our inherited testing, those gene changes can be found in every cell in the body, so we can do that from a simple blood test where we just look at the blood cells. We can actually do it with our sputum and with a cheek swab, even. You can get enough of the DNA from the inside of the mouth to do that.

For a tumor’s genetics, we need some of the tumor, so that’s either done with a biopsy into the metastatic site or, as I mentioned before, increasingly, we’re exploring the potential for a liquid biopsy – so, drawing some blood and then trying to find pieces of the tumor that are shed into the blood.

Katherine:      

What advances have there been in testing?

Dr. Gralow: 

Well, it used to be – just going back a couple of years ago – that we didn’t do a lot of this genetic testing or genomic profiling of the tumor because we didn’t have many – the term is an “actionable mutation.” So, if we found something, would we do something with it? Did we have a drug we could use to do it? But, more and more and more, even in breast cancer, we’re finding actionable mutations that would drive therapy.

For example, in estrogen receptor positive breast cancer, we have a new class of targeted therapies called PI 3-kinase inhibitors – a drug called alpelisib or Piqray was approved in the last couple of years in that category – and it only is effective in estrogen receptor positive breast cancer that has a mutation in the PI 3-kinase gene. So, that would be something we’re looking for in the tumor’s genes, and actually, we need to know that there’s a mutation to even get the drug approved for treatment because it doesn’t work if you don’t have that mutation.

Increasingly, we’re finding some changes that can happen in the estrogen receptor gene and the HER2 gene, interestingly, so that you can have estrogen receptor expressed on your tumor, but over time, that tumor might develop an estrogen receptor mutation so that it stops responding to certain drugs that target the estrogen receptor.

And so, that’s called an ESR1. That’s the name of the estrogen receptor gene – an ESR1 mutation – and that would tell me probably not going to respond as well to a drug in the class we call aromatase inhibitors, but might respond better to a drug in the class that we call the selective estrogen receptor degraders like fulvestrant or Faslodex, is the name of a drug in that class.

We’re also finding that you can have what we call activating mutations in HER2, and they can be present whether the tumor overexpresses HER2 or not, and we’ve got some ongoing clinical trials looking at if the tumor doesn’t have extra HER2 on its surface – so, it doesn’t have extra HER2 protein, but at the gene level, it’s got an activated HER2 gene – we can use certain types of HER2 therapy to treat it, and we’re testing that right now in clinical trials.

So, could we even use some HER2 drugs even though technically, the tumor would be classified as HER2 negative? So, fascinating increasing information that we’re understanding, and I also mentioned before we can inherit mutations in genes such as BRCA1 and 2, but fascinatingly, the tumor can acquire those mutations. Even if we didn’t inherit a mutation, we can see mutations in the BRCA1 and 2 gene – we call those somatic as opposed to germline mutations. So, “germline” means it’s in every cell in your body, but “somatic” means the tumor somehow acquired this over time.

And so, we’ve done – we just presented some very early results of a trial, and we’re expanding this trial, looking at if you didn’t inherit a BRCA1 or 2 mutation, so technically, you don’t qualify for a PARP inhibitor, but if the tumor acquired a mutation and we can prove that with testing the tumor’s DNA, then we have seen responses from these PARP inhibitors, so that opens up another whole class of treatments, and there are other DNA repair genes that actually may be qualified as well that we can inherit or that can be acquired by the tumor.

So, more and more, we’re doing this genomic profiling, and it is leading to results that would give us possible treatment options.

Katherine:  

Dr. Gralow, the goal of this program is to provide the confidence and tool for patients to advocate for the essential tests to get best care personalized to them. Are there specific tests that patients should make sure they have?

Dr. Gralow:  

Well, there are a lot of assays out there to do this genomic profiling or genetic testing of the tumor, so I don’t promote any one. Various institutions do it and do it well, various companies do it, but I think every metastatic patient should have the tumor looked at in this kind of profiling.

I also think every metastatic patient should advocate for having a biopsy of their cancer, and if a biopsy cannot be done safely in the recurrence, then see if they could get a liquid biopsy – have blood drawn to find it. So, I think that patients should be asking about this. Sometimes, insurance won’t always cover it, and so, my job as a treating physician is to advocate for that, to do an appeal.

More and more, because we have so many actionable mutations in breast cancer now, I’m not having insurance decline, but occasionally, it does, and then it’s our job as the healthcare providers to make the case that yes, this will impact the patient, and yes, it should be covered by insurance.

Katherine:  

You’ve been referring to a number of terms. Patients may have heard the BRCA or “braca” that relate to breast cancer in genetics. Would you give us an overview of common mutations in breast cancer?

Dr. Gralow:    

So, of the mutations that we can inherit, the first two that were discovered were BRCA1 and BRCA2, and for all breast cancer – not just metastatic, but all breast cancer – we think that maybe 5-10% of breast cancer is the direct result of the inheritance of a strong gene that gives you a high – not 100%, but a high likelihood of developing breast cancer.

So, for BRCA1 and 2, these two genes are associated predominantly with breast and ovarian cancer, and if you live out your normal lifespan, you could have up to a 75-80% chance of getting one of those two cancers, and breast cancer being more common. Also, some association with some other cancers including, interestingly, prostate cancer, which we’re learning more about.

So, BRCA1 and 2 are the most common, and they tend to be found – because they have such a high association with the risk of breast and ovarian cancer, they tend to be found in families that have a lot of other breast cancers, and also breast and ovarian cancer presenting at a younger age. So, you’ve inherited a gene that leads to a high predisposition, and the cancer occurs earlier.

So, whereas the average age of diagnosis of breast cancer in the U.S. is 61-62 most commonly, in a patient who’s inherited a BRCA1 or 2 gene mutation, it’s closer to 40-42 – so, a lot younger. And then, there are a variety of other genes that can be inherited that are either much less common or have a weaker link. So, for example, there are genes called CHEK2 or PALB2, ATM, P53 – I just mention that because some of the listeners will potentially have one of those mutations or have heard it. Those are either rarer or they’re associated with a weaker chance of getting cancer.

So, those might be more commonly found in a family that doesn’t have a lot of cancer in it because a carrier – the mother or the father – and their other relatives would have maybe only a 30% chance of getting breast cancer in some cases. So, there would be a lot of carriers who don’t get cancer.

So, as I mentioned earlier, I think it’s really important – especially right now in metastatic breast cancer – that pretty much everybody, even if you didn’t have a strong family history, even if you weren’t diagnosed at a young age, get tested because if we find one of these inherited mutations, we now have some additional treatment options, especially right now, approved for BRCA1 or 2, but clinical trials going on for many of these other genes.

Katherine: 

How do these mutations affect disease progression and prognosis?

Dr. Gralow:          

So, most of the genes I’ve mentioned – in their normal state, they’re critical, actually. They’re called DNA repair genes, and their job in our life is when we accidentally make a mistake when we’re replicating our DNA and two cells are dividing, if there’s a mistake in the DNA, they go in and repair it. And, we’ve got all kinds of mechanisms to try to prevent mutations from happening as cells divide, and BRCA1 and 2 are a key part of that, and so, they’re fixing it.

So, if you inherit a mutation in one of those genes, you still have some ability to repair any routine mistakes that are being made, but over time, you have less ability, and then, if you get a cancer that has a deficiency in BRCA1 or 2, those cancers can be more sensitive to certain kinds of chemotherapy that affects DNA repair.

So, for example a class of chemotherapy agents called the platinum drugs – carboplatin and cisplatin – may be more effective in BRCA1- or 2-mutated cancers, also more generally in triple negative breast cancer because they can be more similar to BRCA1-mutated cancers in a lot of ways.

So, to go back to your original question, once a cancer has developed in a patient who has a BRCA1 or 2 mutation, we treat that cancer for what it is. So, it might have developed estrogen – have estrogen receptor on the surface or HER2, so we treat it as the subtype that developed, and actually, the chance of cure is just the same for BRCA1-associated breast cancer as it would be for one that doesn’t have a BRCA.

But, the chance of getting a second breast cancer – a totally new breast cancer – would be higher unless you chose to remove both of your breasts and the bulk of your breast tissue. So, decisions like surgery – if you had a known BRCA1 mutation, we’d treat the cancer you have now aggressively and for cure, but when you talk about your surgery options, we’d say doing more aggressive surgery, like removing both of your breasts – that’s not gonna improve your chance of surviving the cancer you have now, but it will markedly reduce the chance of getting a second breast cancer.

So, you could consider that as an option for surgery – not to improve your chance of this cancer, but to reduce the chance of another breast cancer. So, your surgery decisions might be impacted by knowing your BRCA1 or 2 mutation. And then, clearly, if you had metastatic breast cancer, knowing if you had the option of a PARP inhibitor, one of the drugs in that class could be – you could have a different treatment option for drug therapy.

Katherine: 

Well, Dr. Gralow, what other factors should be taken into consideration with a treatment route?

Dr. Gralow:   

I always like to think of the treatment decision as relying on three factors, and the first relates to the tumor factor, the cancer factor.

So, we talked a lot about the biology, the estrogen receptor, the HER2, the genomic profiling. So, that’s critical, but there are two other components that we need to really strongly consider when trying to devise the right treatment regimen. One of those is patient factors, and not just the patient’s genetics, but are they pre- or post-menopausal?

What is the age? Where are they in life? Are they young with young kids? Are they working, and is that an important priority for them? Are they older and with grandchildren, and they don’t need to work? What is it that would be critical? What are the patient’s priorities here, and what are their fears, what are the things they would – what would be really important as we plan a regimen? And so, the patient factors which would be patient priorities and where they are in life right now.

And then, there’s factors related to the treatment itself, which would include not just how effective it is, but – and, this is really important when trying to decide regimens – what are the side effects of a regimen? For some patients, hair loss is a big deal, and we can put it off as long as possible – maybe choosing the first couple regimens don’t cause hair loss sometimes.

But, for other people, that doesn’t matter to them. For some, we have oral – some regimens, and that could keep them out of the infusion room, and others actually – I’ve had patients who actually like coming into the infusion room regularly so that they can review the side effects and get the reassurance provided by it. So, we’ve got different route of administration of the drugs, different side effects. If you already had, for example, a neuropathy – a numbness/tingling of fingers and toes – from treatment that you might have gotten for early-stage disease, we’d probably want to avoid drugs where that’s their major side effect in the metastatic setting and that would increase that even further.

We’ve got some drugs that cause a lot of toxicity to our GI system – nausea, vomiting, or diarrhea – and other drugs that don’t. And so, understanding what symptoms the patient already has and actually tailoring the treatment based on some of the side effects of the drug could also be done, as well as how they’re administered. So, again, patient factors, tumor factors, and then, factors related to the treatment itself all come into play when we make decisions.

Katherine:    

There have been so many advances in breast cancer research. What are you excited about in research right now?

Dr. Gralow: 

Well, every single drug that’s been approved, every single new regimen that’s been approved in breast cancer is the direct result of clinical trials, and this is a major part of my career, is to help patients get access to clinical trials and run important clinical trials that could lead to new discoveries – is this regimen better? What’s the toxicity?

Because until we have a cure for breast cancer, we need to do better, and we need to research better treatment options. So, doing trials, having access to clinical trials where you can participate, help move the science forward is key.

I think where we’re moving with breast cancer is the more we’re understanding the patient and the tumor, the more we’re realizing every single breast cancer is different, actually, and whereas when I started my training 20-plus years ago, breast cancer was breast cancer – we weren’t even using HER2 yet, we were just learning how to use estrogen receptor, and we kind of treated everything the same – now, we’re subsetting, and subsetting, and subsetting. Even in triple negative breast cancer now, which is about 18-20% of breast cancer, we’re subsetting.

Does that triple negative breast cancer have PD-L1, which is associated with being able to get immunotherapy drugs? Does it express androgen receptor? Because sometimes, even a breast cancer that doesn’t have estrogen or progesterone receptor can express the androgen receptor, like prostate cancer, and we can use some prostate cancer drugs. So, even triple negative breast cancer we’re subsetting and subsetting, and could that triple negative breast cancer be associated with a BRCA1 or 2 mutation, and then we can use the PARP inhibitors?

So, I’m actually really excited about that we’re learning more and more, and subsetting, and not treating breast cancer as one size fits all, and if we can better tailor the treatments to the patient and the tumor, that we are going to get to the point where I can tell my patients yes, we can get cures in metastatic breast cancer.

Katherine:    

For patients who may be hesitant to speak up – to advocate for themselves in the process – I’m gonna start again. For patients who may be hesitant to speak out for themselves and advocate for their own care and treatment, what advice do you have?

Dr. Gralow:   

You have a whole team who’s behind you, and I’m the MD on the team, but I’ve got a nurse practitioner, and a nurse, and a scheduler, and a social worker, and a nutritionist, and a physical therapy team, and financial counselors. I’ve got a whole team who works with me. And so, a patient might be hesitant to speak up during the actual appointment with their physician. It’s a short amount of time. I would recommend come into it with written-down questions because things go fast. You don’t get a lot of time with your doctor.

Things go fast, but don’t come in with 25 questions, either. Pick your top few that you want to get taken care of this visit because if you come in with 25 or 30, you’re gonna lose the answers to most of them. Maybe bring somebody with you who’s an advocate and a listener for you who could be taking notes, so you can process and you don’t have to write it down, or ask if you can record it. It’s really important if you’re newly diagnosed or maybe there’s a progression and you’re going on a new treatment. That’s okay too.

But, I would also say you have a whole team behind you, so sometimes, if you don’t have time or if you’re hesitant to speak up in your doctor’s visit, you can ask the nurse, or maybe you can ask the social worker for help, even. See if there’s support groups around.

Interestingly, we’ve got a peer-to-peer network where patients can request to talk to somebody else who’s matched to them by some tumor features, and their stage, and things like that. Maybe finding somebody else who’s gone through something similar, and somebody independent to talk to instead of relying on your family.

It can also be really helpful to talk to a therapist or a psychologist about your fears, and sometimes, you want to be strong for your family, strong for your children and all, but you need a safe space with somebody that you can just express your fears and your anger if that’s what’s going on, or your depression or anxiety to while you’re trying to hold a strong face for others in your family. So, I would encourage patients to look at who is the whole team and talk to the other members of the team as well, and sometimes, they can help advocate.

Also, find somebody who might be able to come to your appointments with you, somebody who will help you advocate or remind you – “Didn’t you want to ask this question?” – or be another set of ears that you can process it with afterwards.

Katherine:     

Dr. Gralow, we’ve covered a lot of useful information today for patients. Thank you so much for joining us.

Dr. Gralow:    

Thank you, Katherine.

Katherine:       

And, thank you to all of our partners. To learn more about breast cancer and to access tools to help you become a proactive patient, visit powerfulpatients.org. I’m Katherine Banwell.

Confused About Immunotherapy and Its Side Effects? You Aren’t Alone

“You don’t look like you have cancer.”

More than one patient undergoing immunotherapy to treat cancer has reported hearing statements like that. Immunotherapy is one of the recent advances in cancer treatment that belie the stereotypes about the effects of cancer treatment. 

The side effects of immunotherapy are different from those associated with chemotherapy and radiation. However, that does not mean immunotherapy does not have side effects. Patients and care partners need to be aware of these potential side effects and to be vigilant in addressing them with their oncologists because they can signal more serious complications if left untreated.

What is Immunotherapy?

Despite the increase of immunotherapy treatment options in recent years and considerable media attention paid to advancements in this field, there remains confusion about immunotherapy and its side effects. Many cancer patients are unaware of whether immunotherapy treatments are available for their specific diagnosis. Others don’t know that genetic profiling of their tumors is usually required to determine if immunotherapy is an option and not all treatment centers routinely conduct genetic profiles of tumors. A  survey by The Cancer Support Community found that the majority of patients who received immunotherapy knew little to nothing about it prior to treatment and were unfamiliar with what to expect.

Immunotherapy works by manipulating the patient’s immune system to attack cancer cells. It is perceived as gentler and more natural than chemotherapy and radiation, without the same destructive effect on the body’s healthy tissues.  This, combined with a lack of prior understanding of immunotherapy, can lead patients and care partners ill-prepared for possible side effects.

Furthermore, immunotherapy is a category of therapies, not a single type of treatment. There are a variety of immunotherapy drugs, most of which are administered via infusion.  Side effects will vary by drug, the cancer and its location, treatment dose, and the patient’s overall health.

The following are the most common types of immunotherapy.

  • Checkpoint inhibitors use drugs to block proteins in the patient’s immune system that would otherwise restrain the immune system, often referred to as taking the “brakes” off the immune system.
  • CAR-T therapy modifies the patient’s T-cells in a lab to enhance their ability to bind to cancer cells and attack and kill them.
  • Oncolytic virus therapy uses genetically modified viruses to kill cancer cells.
  • Another therapy uses cytokines (small proteins that carry messages between cells) to stimulate the immune cells to attack cancer.

Immunotherapy can be part of combination therapy. It might be combined with chemotherapy. It might be used to shrink a tumor that is then surgically removed.  Or multiple immunotherapy drugs might be used simultaneously.

What Are The Side Effects?

With immunotherapies, side effects typically occur when the immune system gets too revved up from the treatment. The most common side effects for immunotherapy treatments are fatigue, headache, and fever with flu-like symptoms. Some people also experience general inflammation often in the form of a rash. Many melanoma patients report blotchy skin discoloration, called vitiligo, during treatment. These milder side effects can usually be managed with over-the-counter remedies and adjustments to daily activities.

For checkpoint inhibitors, the fastest growing segment of immunotherapy treatments, mild side effects occur in 30% – 50% of patients. Serious side effects typically occur in less than 5% of patients. (See “Understanding Immunotherapy Side Effects” from the National Comprehensive Cancer Network and the American Society of Clinical Oncology.)

Less common side effects are blisters, joint pain, thyroid inflammation, and colitis (inflamed colon resulting in diarrhea with cramping). Some patients who receive CAR T-cell therapy develop a condition known as cytokine release syndrome, which causes fever, elevated heart rate, low blood pressure, and rash. 

In rare cases, immunotherapy has resulted in lung inflammation, hepatitis, inflammation of the pituitary, and detrimental effects on the nervous and endocrine systems. In most cases, the conditions clear up when treatment ends.  However, there have been outcomes in which immunotherapy caused diabetes or tuberculosis.

“Overall there are fewer side effects [with immunotherapy],” explained Dr. Justin Gainor, a lung and esophageal cancer specialist at Mass General during an Immunotherapy Patient Summit hosted by the Cancer Research Institute. “But the immune system can affect anything from the top of the head down to the toes. Any organ has the potential to be affected.”

As the application of immunotherapy has expanded, so has our understanding of the potential side effects. Like most medical treatments, how one person responds to immunotherapy can be different from another even when the cancer diagnosis and drug therapy are the same.

The essential thing patients and care partners need to know about side effects is they should always be reported to their oncologist or nurse oncologist.

Why Patients Should Talk to Their Provider About Immunotherapy Side Effects

Because immunotherapy has created newer therapy options, there isn’t the volume of experiences as with older treatments. The infinite number of variables that patients provide once a treatment moves beyond clinical trials and into the general patient population generate more diverse outcomes.  And, as most therapies are less than 10 years old, there hasn’t been an opportunity to study the long-term effect of these therapies. This is why oncologists advise patients and their caregivers to be extra vigilant in noting any changes experienced during and after treatment.

Many side effects are easy to treat but medical providers want patients to be forthcoming in discussing any and all side effects. This is in part to improve understanding of side effects, but also because a mild cough or a case of diarrhea might be harbingers of a more systemic issue that will grow worse if left untreated.

Patients should not be hesitant to discuss side effects because they fear they will be taken off immunotherapy.  Sometimes a pause in treatment might be necessary, but the earlier the oncologist is made aware of a side effect, the less likely that will be necessary.

In addition, patients undergoing immunotherapy should always take the name(s) of their immunotherapy drugs and the name of their oncologist when seeing medical professionals outside of their cancer treatment team. This is especially important when visiting the ER.  Because immunotherapy drugs are newer and highly targeted to certain cancers, many medical professionals remain unfamiliar with drug interactions and treating related side effects.

Immunotherapy On The Rise

Immunotherapy treatments have resulted in reports of remission in cases that would’ve been deemed hopeless just five or 10 years ago.  The Federal Drug Administration (FDA) has approved various immunotherapy treatments for melanoma, lung cancer, head and neck cancer, bladder cancer, cervical cancer, liver cancer, stomach cancer, lymphoma, breast cancer, and most recently bladder cancer.  (Here is a list of  immunotherapies by cancer type from the Cancer Research Institute.)

“It’s revolutionized how we treat our patients,” says Dr. Gainor of Mass General about immunotherapy’s impact on lung and esophageal cancer.

Advances in immunotherapy research and trials continue to generate optimism and excitement. A clinical study in Houston is looking at using immunotherapy to prevent a recurrence. Researchers in Britain recently announced a discovery that might lead to advances in immunotherapy treatments to a much broader array of cancers.

While there is excitement around the field of immunotherapy and it has resulted in unprecedented success in treating some previously hard-to-treat cancers, it remains an option for a minority of cancer diagnoses.  It works best on solid tumors with more mutations, often referred to as having a high-mutational load or microsatellite instability (MSI) high. And it is not universally successful for every patient.

With hundreds of clinical trials involving immunotherapy alone or in combination with other therapies, it is certain more treatment options are on the horizon. As more therapies are developed and more patients with a greater variety of conditions undergo immunotherapy, we will also increase our understanding of potential side effects.

Side effects should not dissuade patients and care partners from considering immunotherapy if it is available or from advocating for genetic tests to deteimine if it is an option. Many patients undergoing immunotherapy have previously undergone chemotherapy and report that the side effects are fewer and milder by comparison.  The important thing is that patients and their partners know what to expect and communicate with their treatment team.

If the next 10 years in immunotherapy research and development are anything link eth elast 10, we can expect more exciting advancements in the battle against cancer. For more perspective on what’s ahead for immunotherapy see the Cancer Research Institute’s article: Cancer Immunotherapy in 2020 and Beyond.

Breast Cancer Treatment and Side Effects

This was originally published by breastcancer.org here.


In recent years, there’s been an explosion of life-saving treatment advances against breast cancer, bringing new hope and excitement. Instead of only one or two options, today there’s an overwhelming menu of treatment choices that fight the complex mix of cells in each individual cancer. The decisions — surgery, then perhaps radiation, hormonal (anti-estrogen) therapy, and/or chemotherapy — can feel overwhelming.

Breastcancer.org can help you understand your cancer stage and appropriate options, so you and your doctors can arrive at the best treatment plan for YOU.

In the following pages of the Treatment and Side Effects section, you can learn about:

  • Planning Your Treatment: What types of treatment are available, the most likely sequence of treatments, treatment options by cancer stage, and fitting treatment into your schedule.
  • Getting a Second Opinion: Reasons for getting a second opinion about your treatment plan, how to go about getting one, and what to do once you’ve got it.
  • Surgery: Breast-conserving surgery (lumpectomy), mastectomy, and lymph node dissection, and what to expect from each. Also included: Prophylactic surgery and breast reconstruction.
  • Chemotherapy: How chemotherapy works, who should get it, different types and combinations, and side effects and how to manage them.
  • Radiation Therapy: How radiation therapy works, who it’s for, advantages, side effects, and what to expect when you get it.
  • Hormonal Therapy: The link between hormones and breast cancer and how different groups of drugs — including ERDs, SERMs, and aromatase inhibitors — can affect that link. Also covered: Side effects of hormonal therapies.
  • Targeted Therapy: How different drugs work, who should get them, how they’re given, side effects, and major studies.
  • Immunotherapy: What is immunotherapy, different types of immunotherapy, and who it’s for.
  • Complementary and Holistic Medicine: How complementary medicine techniques such as acupuncture, meditation, and yoga could be a helpful addition to your regular medical treatment. Includes research on complementary techniques and ways to find qualified practitioners.
  • Drugs for Treatment and Risk Reduction: A reference list of drugs used to treat and reduce the risk of breast cancer, including how they work, to whom they are typically given, and side effects.
  • Treatments for Pain: Ways to treat cancer- and treatment-related pain, including types of medications and tips on talking to your doctors about pain.
  • Treatment Side Effects: A reference list of side effects and ways to manage them.
  • Lymphedema: All about lymphedema, including who is at risk, what to watch out for, how to reduce risk of lymphedema flare-ups, and how to find a lymphedema therapist.
  • Clinical Trials: What clinical trials are and how they work, why they’re important, and how to find trials that may be appropriate for you.

A New Standard of Care for HER2-Positive Metastatic Breast Cancer?

This podcast was originally published by Dr. Rashmi Murthy on December 11, 2019 on breastcancer.org here.


Dr. Rashmi Murthy, assistant professor of breast medical oncology at the University of Texas MD Anderson Cancer Center in Houston, discusses the results of the HER2CLIMB study that she presented at the 2019 San Antonio Breast Cancer Symposium showing that the experimental medicine tucatinib offers benefits to people diagnosed with HER2-positive metastatic breast cancer and may be a new standard of care.

Listen to the episode to hear Dr. Murthy explain:

  • A summary of the study results
  • Why this study included people with brain metastases
  • The side effects of tucatinib
  • Why she thinks the results are practice changing

Understanding Clinical Trials: A Jargon Buster Guide

When it comes to cancer treatment you or a loved one may be considering participating in a clinical trial as a treatment option.  Clinical trials are designed to evaluate the safety and effectiveness of a treatment. They may involve researchers administering drugs, taking blood or tissue samples, or checking the progress of patients as they take a treatment according to a study’s protocol.

Learning about clinical trials can be a steep learning curve – not least because the process comes with a lot of new terms, acronyms and jargon.  To help you, I’ve put together this list of the most common terms you will find when you are researching clinical trial information. This is not an exhaustive list but it is a helpful starting point. At the end of this article you will see links to find more information.

Adverse Effects (AE)

Also called Adverse Events, or Adverse Drug Reaction, AEs are any harmful event experienced by a person while they are having a drug or any other treatment or intervention. In clinical trials, researchers must always report adverse events, regardless of whether or not the event is suspected to be related to or caused by the drug, treatment or intervention.

Arm

Subsection of people within a study who have a particular intervention.

Bias

Bias is an error that distorts the objectivity of a study. It can arise if a researcher doesn’t adhere to rigorous standards in designing the study, selecting the subjects, administering the treatments, analysing the data, or reporting and interpreting the study results. It can also result from circumstances beyond a researcher’s control, as when there is an uneven distribution of some characteristic between groups as a result of randomization.

Blinding

Blinding is a method of controlling for bias in a study by ensuring that those involved are unable to tell if they are in an intervention or control group so they cannot influence the results. In a single-blind study, patients do not know whether they are receiving the active drug or a placebo. In a double-blind study, neither the patients nor the persons administering the treatments know which patients are receiving the active drug. In a triple-blind study, the patients, clinicians/researchers and the persons evaluating the results do not know which treatment patients had. Whenever blinding is used, there will always be a method in which the treatment can be unblinded in the event that information is required for safety.

Comparator

When a treatment for a specific medical condition already exists, it would be unethical to do a randomized controlled trial that would require some participants to be given an ineffective substitute. In this case, new treatments are tested against the best existing treatment, (i.e. a comparator). The comparator can also be no intervention (for example, best supportive care).

Completed

A trial is considered completed when trial participants are no longer being examined or treated (i.e. no longer in follow-up); the database has been ‘locked’ and records have been archived.

Control

A group of people in a study who do not have the intervention or test being studied. Instead, they may have the standard intervention (sometimes called ‘usual care’) or a dummy intervention (placebo). The results for the control group are compared with those for a group having the intervention being tested. The aim is to check for any differences. The people in the control group should be as similar as possible to those in the intervention group, to make it as easy as possible to detect any effects due to the intervention.

Efficacy

How beneficial a treatment is under ideal conditions (for example, in a laboratory), compared with doing nothing or opting for another type of care. A drug passes efficacy trials if it is effective at the dose tested and against the illness for which it is prescribed.

Eligibility Criteria/ Inclusion and Exclusion Criteria

Eligibility criteria ensures patients enrolling in a clinical trial share similar characteristics (e.g. gender, age, medications, disease type and status) so that the results of the study are more likely due to the treatment received rather than other factors.

Follow-up

Observation over a period of time of participants enrolled in a trial to observe changes in health status.

Informed Consent

A process (by means of a written informed consent form) by which a participant voluntarily agrees to take part in a trial, having been informed of the possible benefits, risks and side effects associated with participating in the study.

Intervention

The treatment (e.g., a drug, surgical procedure, or diagnostic test) being researched. The intervention group consists of the study participants that have been randomly assigned to receive the treatment.

Investigator

A person responsible for the conduct of the clinical trial at a trial site. If a trial is conducted by a team of individuals at a trial site, the investigator is the responsible leader of the team and may be called the principal investigator (PI).

Multicentre Trial

A clinical trial conducted according to a single protocol but at more than one site, and therefore, carried out by more than one investigator.

Number needed to treat (NNT)

The average number of patients who need to receive the treatment or other intervention for one of them to get the positive outcome in the time specified.

Outcome Measures

The impact that a test, treatment, or other intervention has on a person, group or population.

Phase I, II, III and IV Studies

Once the safety of a new drug has been demonstrated in tests on animals, it goes through a multi-phase testing process to determine its safety and efficacy in treating human patients. If a drug shows success in one phase, the evaluation moves to the next phase

  • Phase 1 tests a drug on a very small number of healthy volunteers to establish overall safety, identify side effects, and determine the dose levels that are safe and tolerable for humans.
  • Phase II trials test a drug on a small number of people who have the condition the drug is designed to treat. These trials are done to establish what dose range is most effective, and to observe any safety concerns that might arise.
  • Phase III trials test a drug on a large number of people who have the condition the drug is designed to treat. Successful completion of Phase III is the point where the drug is considered ready to be marketed.
  • Phase IV trials can investigate uses of the drug for other conditions, on a broader patient base or for longer term use.

Placebo

A fake (or dummy) treatment given to patients in the control group of a clinical trial.  Placebos are indistinguishable from the actual treatment and used so that the subjects in the control group are unable to tell who is receiving the active drug or treatment. Using placebos prevents bias in judging the effects of the medical intervention being tested.

Population

A group of people with a common link, such as the same medical condition or living in the same area or sharing the same characteristics. The population for a clinical trial is all the people the test or treatment is designed to help.

Protocol

A plan or set of steps that defines how something will be done. Before carrying out a research study, for example, the research protocol sets out what question is to be answered and how information will be collected and analysed.

Randomized Controlled Trial (RCT)

A study in which a number of similar people are randomly assigned to 2 (or more) groups to test a specific drug, treatment or other intervention. One group has the intervention being tested; the other (the comparison or control group) has an alternative intervention, a placebo, or no intervention at all. Participants are assigned to different groups without taking any similarities or differences between them into account. For example, it could involve using a computer-generated random sequence. RCTs are considered the most unbiased way of assessing the outcome of an intervention because each individual has the same chance of having the intervention.

Reliability

The ability to get the same or similar result each time a study is repeated with a different population or group.

Sample

People in a study recruited from part of the study’s target population. If they are recruited in an unbiased way, the results from the sample can be generalised to the target population as a whole.

Subjects

In clinical trials, the people selected to take part are called subjects. The term applies to both those participants receiving the treatment being investigated and to those receiving a placebo or alternate treatment.

Trial Site

The location where trial-related activities are conducted.


References

The Canadian Institutes of Health Research (CIHR)

TROG Cancer Research

ICH.org

NICE

Further Resources

American Society of Clinical Oncology’s Cancer.Net trials site

National Cancer Institute (NCI) Clinical Trials lists open and closed cancer clinical trials sponsored or supported by NCI. 

ClinicalTrials.gov database of privately and publicly funded clinical studies

CenterWatch Clinical Trials Listing

Does Cannabis Oil Have a Role in Cancer Treatment?

Does Cannabis Oil Have a Role in Cancer Treatment? from Patient Empowerment Network on Vimeo.

Is it just a trend or could cannabis oil truly have a role in cancer care and treatment? Dr. Sangmin Lee share his perspective.

Dr. Sangmin Lee is a hematologist-oncologist specializing in blood disorders and blood cancers at Weill Cornell Medicine and New York Presbyterian Hospital. More about Dr. Lee here.

See More From The Fact or Fiction? AML Series


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Transcript:

Patricia:

The use of cannabis oil is becoming prevalent. Does this have a role in cancer care and treatment?

Dr. Lee:

Absolutely. So, we use it for a lot of side effect management. So, cannabis can be helpful, in terms of appetite and nausea, for example. So, we often use it in conjunction to manage some of the side effects that patients can have throughout their treatment.

You should consult with your medical team, and of course, I should say that laws differ state by state, so it doesn’t apply to every state. But when it’s available, it can be a valuable addition.

Patricia:

Sure. Discuss that with your physician.

Are Clinical Trial Participants Monitored More Closely?

Are Clinical Trial Participants Monitored More Closely? from Patient Empowerment Network on Vimeo.

Dr. Sangmin Lee discusses the monitoring of clinical trial participants and the measures taken for patient safety.

Dr. Sangmin Lee is a hematologist-oncologist specializing in blood disorders and blood cancers at Weill Cornell Medicine and New York Presbyterian Hospital. More about Dr. Lee here.

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Transcript:

Patricia:

How about this next one? I am monitored more closely in a clinical trial.

Dr. Lee:

In some cases, it’s true. Clinical trials do have certain monitoring visits, in terms of doctor’s visits, laboratory tests, and physical exams.

The purpose of that is to make sure that it is safe. So, the purpose of monitoring closely, in a lot of cases, is for the patient’s safety. We are testing drugs in a lot of clinical trials, for which the complete safety profile, as well as efficacy profile, is not known. So, the purpose of closer monitoring is to make sure whatever we’re doing is safe, and if there are any unexpected side effects, then it allows us to address the side effects, as well. So, it’s mainly for patients’ safety.

Will Clinical Trials Cost You? The Facts.

Will Clinical Trials Cost You? The Facts. from Patient Empowerment Network on Vimeo.

Dr. Sangmin Lee reviews the financial impact associated with clinical trials, including a discussion of what expenses are covered for participants.

Dr. Sangmin Lee is a hematologist-oncologist specializing in blood disorders and blood cancers at Weill Cornell Medicine and New York Presbyterian Hospital. More about Dr. Lee here.

See More From The Fact or Fiction? AML Series


Related Resources

Confronting Fears About Clinical Trials

AML Research and Internet Claims Program Resource Guide

Second Opinions in AML: The Importance of Moving Swiftly

Transcript:

Patricia:

All right, how about this one: I may have unexpected costs if I join a clinical trial.

Dr. Lee:

So, typically, that’s actually, usually not true, because how it works is that the clinical trial drugs, and that there may be extra procedures or visits associated with clinical trials.

And what usually happens is that the sponsor of the clinical trial provides the cost of the drug, intervention, and anything extra that are required for the clinical trial. So, in the end, the cost of participating in a clinical trial should not be any more than receiving standard care treatment.

In some rare cases, there may be stipends associated with the clinical trial, especially with travel. So, if you participate in a clinical trial, and you live far away, then you should ask to see if there is any stipends available, especially for travel.

Is It Safe? Breaking Down the Clinical Trial Process

Is It Safe? Breaking Down the Clinical Trial Process from Patient Empowerment Network on Vimeo.

The idea of a clinical trial can be intimidating and confusing for many patients. Dr. Sangmin Lee explains the phases of clinical trials, including the safety protocols in place to protect patients.

Dr. Sangmin Lee is a hematologist-oncologist specializing in blood disorders and blood cancers at Weill Cornell Medicine and New York Presbyterian Hospital. More about Dr. Lee here.

See More From The Fact or Fiction? AML Series


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Transcript:

Patricia:

What is the process for getting medicine to patients during clinical trials?

Dr. Lee:

So, clinical trials are basically what’s needed to prove that drugs work. So, a lot of times, we test drugs in a test tube in AML cell lines, and they show great promise. But just because a drug works in a test tube setting, doesn’t actually mean that it will work in humans, because human bodies are much more complicated. So, we need to test promising drugs in humans to make sure they are safe and effective.

And that’s what the purpose of clinical trials are. Once they demonstrate safety and efficacy, then a drug then gets to be approved, and is available commercially. So, that’s the purpose of clinical trials.

To be involved in clinical trials, what it involves is, basically, you have to meet a sort of criteria, called eligibility, because different clinical trials have different criteria for selection. So, we have to look into that. And then, once you fit an eligibility or selection criteria, then you typically undergo certain diagnostic tests to enroll on a clinical study. And then, you get whatever drug or intervention that is designed to test in that setting.

So, there are numerous steps to actually enroll in a clinical study.

Patricia:

And like you mentioned, there’s a long way between rat studies and human trials. What are the phases of clinical trials?

Dr. Lee:

So, there are three phases for clinical trials, commonly. There’s phase one, and phase two, and phase three. Phase one is the earliest part of the clinical trial process. So, goal of a phase one study is to make sure a drug is safe in a human. So, phase one studies are usually the first time that you are testing the drug in humans, and the main purpose is to demonstrate that it’s safe. So, typically, in a phase one study, typically, you test a drug at a lower dose or dose levels to demonstrate safety. What it means is that you’re enrolling a few patients at a time.

Once a drug is proven to be safe, then you move on to phase two, which is basically testing the drug in more patients. And the purpose of phase two is to get a preliminary assessment of how effective a treatment would be.

So, typically, a phase two study involves many more patients in that setting. And then, if a phase two study shows that a drug is very promising, then the drug may move on to phase three, where, basically, in phrase three, you are comparing one intervention or a drug compared to the standard of care. And, typically, in a phase three setting, a computer decides randomly which intervention you get, whether it’s an intervention or new drug versus standard of care. And standard of care may include either placebo or chemotherapy intervention, that is standard of care. So, it’s not always placebo in phase three.

Breast Cancer Before 40: What You Should Know

Breast Cancer Before 40: What You Should Know from Patient Empowerment Network on Vimeo.

Dr. Stephanie Valente reviews key information for women under the age of 40 with breast cancer, including risk, treatment approaches and the role of genetic testing.

Dr. Stephanie Valente is the Director of the Breast Surgery Fellowship Program at Cleveland Clinic. More about this expert here.

Related Program:

Breast Cancer Before 40: How Can I Preserve My Fertility?


Transcript:

Sure. So, when we talk about breast cancer in young women, usually we are referring to women under the age of 40. And the prevalence for breast cancer in general is one out of eight women. For women under the age of 40, it’s about a seven percent incidence of breast cancer. So, it’s about a seven percent incidence in the general population in the United States. So, that’s about 12,500 women per year are diagnosed under the age of 40 with breast cancer.

So, the prevalence has pretty much been the same over the years. We used to think that women diagnosed at a younger age had a more aggressive breast cancer. But the more we look at things, the more we realize that women under the age of 40 usually are diagnosed at a later stage because it’s not something that somebody in their 20s or 30s is thinking that a breast lump equates to cancer. So, these women unfortunately present at a later stage.

Many times, it’s because they are pregnant, breastfeeding or just not having any family history where the first thing they think of when they get a lump is that they actually have breast cancer.

So, the first thing is women under the age of 40 usually present at a later stage, meaning that they have larger cancers, and the cancers because they are larger have had more time to spread to the lymph nodes. So, these women they don’t necessarily have more aggressive breast cancers than older women, it’s just that it’s found at a later stage.

So, the treatment options are the same for young women with breast cancer. So, depending on the size of the tumor and the size of her breast, women are given the option for a lumpectomy which usually is follow by radiation versus a mastectomy. And studies have shown that either surgical choice is a good option for women. And that one surgery doesn’t make a young woman, or an older woman live any longer.

That the survival for breast cancer is based on stage not the choice of surgery that they pick.

So, one of the first things is that women under the age of 50 – the average age for breast cancer in the United States is between 64 and 68.

So, if you are under the age of 50 or under the age of 40 there’s a higher chance that maybe these women carry a gene that would increase the risk of getting breast cancer. And so nowadays, we test over 21 genes. But these genes can increase their risk of getting breast cancer not only in the breast they have the breast cancer in, but in their other breast as well. It also increases the risk for other types of cancers such as ovarian cancer. And they could potentially pass this gene on to their kids.

So, sometimes women – and this is the great thing about academic medicine. Everything is changing so quickly with the modern research. So, a lot of times a woman who is triple negative, which is an estrogen receptor negative breast cancer.

If they are genetic positive for the BRCA gene, they qualify for certain medications or chemotherapy that we know targets specifically that type of cancer and their gene. So, that’s why it is important for some of these women to get genetic testing to see if the certain chemotherapy regimens or medicines that we have would benefit their type of cancer.

So, one of the things for any woman diagnosed with breast cancer at a young age is to offer those women genetic testing. And sometimes it can be a relief or sometimes it can be very challenging for these women to think, “Oh my gosh. I have this gene. I’m at increased risk for more cancers.” And that potentially they could pass that on to their children.

So, having the women who come in meet with a genetics counselor to go over the risks. And the reality is that of all the women under the age of 50 that test for the gene, only 10 percent actually carry the gene.

So, the good thing is that 90 percent most likely don’t have the gene. But it still is an anxiety provoking thing for these women to go through.

Another thing is that these women a lot of times are younger. So, they have either two things – young children to take care of which is extremely difficult to mange an already stressful motherhood. You throw in a diagnosis of cancer, whether or not there is a dad involved or a father that needs to help out with these kids. A lot of times their families need to help out. So, we have a psych oncologist that’s part of our team. And it’s really great. How do you tell your kids you have cancer? And how do you manage kind of day-to-day life with going through this? So, that’s another great program that is offered. And importantly for a woman who doesn’t have children but maybe desires to have children or even to have more children than the ones that she has,
looking at fertility options for young women is huge.

So, we know that some of the chemotherapy that we give breast cancer patients decreases their ability to have children in the future. So, the chemotherapy can shut down the ovaries. So, sometimes women – and this is the great thing about academic
medicine. Everything is changing so quickly with the modern research. So, a lot of times a woman who is triple negative, which is an estrogen receptor negative breast cancer.

If they are genetic positive for the BRCA gene, they qualify for certain medications or chemotherapy that we know targets specifically that type of cancer and their gene. So, that’s why it is important for some of these women to get genetic testing to see if the certain chemotherapy regimens or medicines that we have would benefit their type of cancer.

So, the one thing is that I tell these women that breast cancer takes a good year between chemotherapy if they need it, surgery, radiation of they need it and the whole process of recovery. That is really takes a good solid year before they are kind of done going to their doctor’s appointments. But the reality is that for an early stage breast cancer, studies have show for young women the overall survival is over 92 percent. So, I say, “This is going to be a tough year. We are going to get through this together. And the good thing is that you are going to be alive in five, 10, 15, 20 years. And so, our goal is to get you the best quality of life not only now but in the future.”

As far as hope for young women with breast cancer, things are changing so fast. The medicine that’s out there – we’re doing these studies where women are getting chemotherapy and by the time they get to surgery about 40 to 60 percent of the tissue that I take out has no residual cancer. That’s phenomenal. That means that the medications that these women are getting are working really well for their cancer. And so, the hope is that in 10 years I don’t have a job because the medicine that they are getting works so well that the cancer can be removed without needing surgery. And so, I think in our lifetime we will find either a cure or a complete resolution of breast cancer.

Is It Difficult to Participate in a Clinical Trial?

Clinical Trial Mythbusters

Clinical Trial Mythbusters: Is It Difficult to Participate in a Clinical Trial? from Patient Empowerment Network on Vimeo.

Downloadable Program Guide

Three experts discuss the clinical trial process and the difficulty in participating in a trial. Our expert panel includes:

  • Ken Getz, MBA – Founder and Board Chair, CISCRP
  • Andy Lee – Senior Vice President and Head, Global Clinical Trial Operations, Merck
  • T.J. Sharpe – Melanoma Survivor and Patient Advocate

Transcript:

Andrew Schorr:

And greetings from Southern California. I’m Andrew Schorr from Patient Power. And welcome to this Patient Empowerment Network program, another in our series of Clinical Trials MythBusters. Our goal, of course, is to help you get the treatment for you or a loved one that you need and deserve. I want to thank the financial supporters for this program to the Patient Empowerment Network; AbbVie, Inc., Celgene Corporation. Daiichi Sankyo and Novartis for their support. They have no editorial control and we’re going to have a very freewheeling discussion today. And really what it’s about is how can a clinical trial be made easier for you to participate? Are there barriers? We’ve talked about it in previous programs. But specifically, what are the companies—the pharmaceutical industry mostly, who sponsor trials all around the world, what are they doing to make trial participation easier? For you to know about trials. For the people at your clinic to know about it and what to say and how to administer it. For you to have documents that are understandable for you and your family to know whether you want to participate. To keep you informed. And also related to the requirements of trials. How can they be relaxed a little so that there may be a trial that would benefit you, that you and your doctor agree on, and the requirements of it allow you to be in the trial. Okay, and the logistics of it are not so tough either. All right, I’ve been in two clinical trials, and I believe I’m alive today because of that. So, I’m very grateful. We have some wonderful panelists with us over the next hour. Now as you have questions, send them to questions@patientpower.info. And some of you have. So, you’ll be able to interact with us as we go along. First, I want to go to Ft. Lauderdale, Florida, and T.J Sharpe. And T.J. has been on programs with me over the years. Stage four melanoma patient having been in trials. And T. J., you would agree, you’re alive today because you were in trials, right?

T.J. Sharpe:

Absolutely, Andrew. I think both of us are very fortunate that we found a trial that was the right treatment for us and gave us the ability to combat our disease in areas may not have been available to us if we just waited for standard of care therapies.

Andrew Schorr:

Right. And here you are—we should say that you were diagnosed a number of years ago with melanoma, went through trials. And now you’ve had two years without treatment, right?

T.J. Sharpe:

Yes. It’s been five years of treatment followed by now two good years of a clean bill of health.

Andrew Schorr:

Well, great. And I should mention for our audience, many people are familiar with T.J. T.J. goes around the country, gives speeches. He’s been at many events, consults with industry that are developing trials to try to bring the patient perspective forward. So, T.J., thank you for all you do. We really appreciate it.

T.J. Sharpe:

You’re welcome. It’s my honor to be able to represent all these patients.

Andrew Schorr:
Well, most every family—certainly most have been touched by cancer. But our other guests are not cancer patients but are in national leadership programs. And so, let’s go up to Medford, Massachusetts at Tufts University outside Boston, Ken Getz. Ken, welcome to the program. Ken, ladies and gentlemen, is a true national leader when it comes to clinical trials and really helping us move forward with better processes, better understanding. Ken, tell us a little bit about your organization there, CISCRP. What does that stand for?

Ken Getz:

Thank you. And I have to say your pronunciation was nearly perfect. It’s hard to pronounce it. It’s an acronym and it stands for The Center for Information and Study on Clinical Research Participation. It’s a non-profit organization. It was founded 18 years ago. And it’s really there to help patients and their families navigate the whole clinical research progress which for many is completely unfamiliar terrain until they’ve been diagnosed with an illness or when they have exhausted all other treatment options. So, CISCRP really helps people become more educated and informed so that they can really think of the clinical research process with more confidence. And they can navigate this unknown terrain.

Andrew Schorr:

All right. I’m going to come back to you in a minute because you have such an overview, and you’re also an Associate Professor at Tufts. And so, you study all this, and you’ve written books. But I want to introduce the third guest. And that is a leader from the pharmaceutical industry and one of our most respected and venerable companies in the field, and this is Merck. So, joining us in a senior vice president of clinical operations there around the world. And that’s Andy Lee. Andy, welcome. Thank you so much for being with us.

Andy Lee:

Andrew, thank you. And pleasure to be with some prestigious panelists, both of whom I know. And I’ve met you over the last two weeks. And thank you to T.J. and yourself who have been trial participants and who are representing that part of the organization.

Andrew Schorr:

Okay, and we should mention that both T.J. and Andy are working on a couple of levels. And Ken sounds off on this too. There is a group called TransCelerate where pharmaceutical industry is working together on some of the issues they face in having the proliferation of trials. More trials sites, more accessibility, procedures for that. And then, of course, Andy has helped lead that effort at Merck related to breakthrough therapies that they have been trying to develop there in supporting patients who might be in Merck trials. So, we are going to come back to that. But I want to go to you for a second, Ken. Ken, how low is the participation among adults in clinical trials, at least in the U.S. Now, I’ve heard really low percentages. Where are we now with that?

Ken Getz:

Right, it’s a great myth for us to start with, this notion that only three to five percent of patients—eligible patients, participate in clinical research. That’s actually a statistic that was published by the National Cancer Institute in the early 1990’s. The latest research really shows that it varies widely. For example, when we look at pediatric cancers, the participation rates are extremely high, 80 to 90 percent in same cases—pediatric leukemia. In part because those communities have very engaged healthcare providers, very engaged families that really share their information. It’s just an enabled community where all of the stakeholders support participation. And then there are other areas of course. Some cancers where we do see relatively low participation rates. But I want to point out that low participation is driven by so many factors, Andy, including the strict eligibility criteria. And the demanding protocol designs which are a real burden for some people, and they choose not to participate. As well as low awareness, very low accessibility to trials among minorities and underserved communities. So, there are many factors that contribute to this variation in the participation rates.

Andrew Schorr:

Yeah, you’ve ticked off some now. T.J., in your own experience, one of the breakthrough trials you were in you had to go from Ft. Lauderdale in South Florida and move your whole family to Tampa in central Florida, right. I mean that was a big deal.

T.J. Sharpe:

Absolutely. When you have a young family and a stage four cancer diagnosis, relocating simply across the state during the holidays especially, is no big deal. We were fortunate because we had the means to be able to move there with work situation, with family. But too many people can barely go across the county, much less the state or the country to find a trial that might be the best match for them.

Andrew Schorr:

Andy, so we’ve ticked off some of the obstacles, and Ken touched on some about even the proliferation of trials. Is that a lot of what you do is how can we have trials be more accessible, be more widely distributed to a clinic near you?

Andy Lee:

Yes, let me just explain. When we look at a new cancer therapy, we look at the various cancers that may be affected. And what we do is we go for high probabilities of success. And the challenge is if you bring a new cancer agent. You normally start off in very advanced disease. So, patients would have failed multiple lines of therapy, and often it is a last gasp. And you have to show some sort of clinical efficacy. And then you move sort of backwards in the disease, and you go from sort of third-plus line, second line and first line.

And then you may work downwards into earlier stages of the disease into an adjuvant setting and maybe a neoadjuvant setting. So, as we sit down and design a trial, what we need to look at is what is the population that is most likely to show any benefit at all. And quite often when you are developing a new therapy, it’s difficult to show benefit because many of the patients are very ill. So, what we have to do is optimize the opportunity for success of a compound by going to the right target patients.

And quite often as we have learned a lot more about cancer, this does not mean we test a product broadly in anyone with cancer. We typically try and find a profile of a patient that is likely to respond. And many patients now will realize their predictor biomarkers or prognostic biomarkers. So, for example, with immunotherapies, those that work through the PD1 mechanism would probably want to have a PD1 ligand receptor positive patient who is likely to bind to the drug.

And that gives a higher probability of success. So, it sounds counterintuitive that while we want to develop therapies for all cancer patients, when we start clinical trial development, we have to show efficacy in a population that will benefit. And that’s normally predefined and makes the inclusion criteria fairly strict. As we show efficacy and as we can move into broader populations, it makes it a lot easier for us to design more liberal clinical trials. And then we can actually spread those in the geographic domains.

I could talk more about geographic allocation, but let’s hold that for the time being, and let’s see if there’s time later on.

Ken Getz:

Can I just add to what Andy said because I think it’s really important for your viewers to understand just how active drug development activity is today. We’re looking at over 4,000 pharmaceutical and biotechnology companies, some of them very, very small. But in total, we’re looking at nearly 6,000 drugs that are in active clinical trials. And to Andy’s point, many are really targeting a patient with a very specific genetic profile or a specific biomarker. But it should give anyone who believes that a clinical trial may be an important care option for them, they should recognize that there may be many, many trials out there.

In total we estimate as many as 80,000 clinical trials, nearly 50 just conducted in the U.S. alone—50,000. So, it’s just important that we keep all this activity in perspective.

Andrew Schorr:
Right. So, T.J., that’s why all of us as patients need to ask about them, right? Go to different resources, whether it’s an advocacy group that you ultimately spoke with other patients, and obviously quizzing the doctors we go to. Is there something that may line up with my situation, right T.J.?

T.J. Sharpe:

Absolutely. There is a both top down and bottom up approach here that patients as they become educated—and every patient should be the owner of their healthcare as they become educated. Hopefully they are coming across advocacy organizations, other informed patients, patient support groups—all of which will help inform them different options for disease treatment, including hopefully as Ken mentioned, clinical research as a care option. At the same time, there is certainly very much an opportunity from the top down from the sponsors who develop the trials and from the sites that execute them to educate patients as they come in.

Not just at their own site, but at any site, at any medical facility. That if you have a diagnosis and you are looking into your care options, that you should be asking the question. And we should be giving you more information on the possibility of clinical trials and where you may find clinical trials that are appropriate for you.

Andrew Schorr:

Right, the whole enchilada, if you will, of all your options. Andy, so you mentioned about trial requirements. So, first of all, what efforts either at Merck or are you aware in the industry are being made to really talk to patients early on as you are designing trials? Whether it’s the requirements—how many CT scans you’re going to have. How often you are going to have to go to the main trial site. All the different things that sometimes get in the way.

Andy Lee:

Well, firstly we start with design. And we believe in exquisite trial design, quality by design as well. So, what we want is to run the experiment once and not have a sloppy trial design. We want to make it really robust in terms of scientific integrity and operational execution. So, we have a lot of internal design committees and what we do is we co-op with many groups external to our company. So, we speak to people who run clinical trials at cancer institutes.

We speak to the doctors who manage this. We speak to the trial coordinators. We speak to people involved with the transporting and shipping of medicine how they would do that. And then we of course speak to people in the ecosystem. We quite often speak to investigational review boards before we start trials. We talk to them about our design and what would be best to protect the rights and well-being of patients. And then, of course, the patient-centric approach says that we need patient insights.

And I’ve chosen my words very carefully because the insights are really important. Not all patients—and I’m very respectful that some patients are very intelligent and actually may be involved in this. Some patients can contribute to design, not all can. And so, what we do is we take the insights and we impute those. We often have focus groups. We talk about this disease. We talk about the burden of the disease. And then we talk about how that disease is managed in an ecosystem. And quite often in different countries it’s managed differently.

And so, we have to appreciate the global clinical trials have to navigate a path that may not be a linear path as we’d see it at an exquisite elite cancer center in the United States. It’s community-based, it’s all the rest. So, we take that input, and what we try to do is unburden the trial for the patient. We say, “How can we design a trial that requires the least visits to the clinic—the hospital, the least burden for them. And how can we take some of that burden from the clinic and actually transfer that into an easier environment.

So, document reading and review. Perhaps filling in questionnaires about quality of life. These are things that don’t have to be done in the clinic itself. And then often when we work with clinics, we work with them to help them understand how we as sponsors can make their life easier. And some of those things might be simplifying the informed consent. But I want to stress just one point here is that we can do whatever we like in the design at a company.

One of the things is, the patients are not sponsor patients. Okay, we sponsor clinical trials. The patients are managed by a doctor and a professional. And underneath that principal investigator is a whole oncology team. And it involves radiology. It involves pharmacists. It revolves around a 360 multidisciplinary team. They’re exquisite. They help manage the patient, not the sponsor. We provide the enabling functions for them. And then also that the oversight of the patient’s right, safety and wellbeing is the responsibility of an institutional review board.

And while we may provide templates and simplify templates in text and language, we rely heavily on the institutional review boards to help us with things that may make things easier, such as reimbursement for parking, transport, all of these things. And by and large, the institutional review boards are very supportive of these things. But they are very difficult to quantify in exact terms because of different geographic regions and different norms in different places. So, we rely heavily on exquisitely well-trained 360 team who manages oncology patients with a great PI. They manage patients.

And we work collaboratively with the sites who work with patients on our behalf. So, I just wanted to say the myth is that sponsors interactive with patients. That’s a myth. And the truth is that we engage with clinical sites, and we try and make our design and all the elements—the enabling elements, simpler for the trial sites in order to manage the patients in a simpler way.

Andrew Schorr:

Okay. Thank you for that. So, Ken, I want your comment on that. Because okay, we are downstream patients. We have a doctor, healthcare team. And we know somewhere in the background there’s a sponsor that tried to enable good things to happen to get reliable data and hopefully a cure for us. So, how do we—what’s happening? Are we improving things there in that interaction between clinic and patient?

Ken Getz:

Yes, we absolutely are. And I’ll start by just echoing and acknowledging that Andy has really laid out just an incredible amount of input that goes into the design of a protocol. And that’s really for a really large company. We see many, many examples now of patient advocacy groups or smaller companies turning to a variety of approaches to solicit input from patients and healthcare providers. Some virtual approaches through a social media or digital community. So, there’s lots of ways that feedback is being channeled.

And that’s really important. The flip side, to really answer your question, is that our protocol designs are becoming more and more complex, more and more demanding. A much larger proportion of drugs are now targeting rare diseases that have been stricter inclusion and exclusion criteria. And the designs of the studies—the number of procedures and the number of visits. The number of investigators that are involved, all of that has also continued to grow. And as a result, we do see that our trials are taking longer.

We have yet to see a year when we actually witnessed a reduction in the cycle time to conduct a clinical trial. And we just have to figure out new ways of making the participation process less burdensome and more efficient.

Andrew Schorr:

Oh, my. So, T.J., you had been living with stage four melanoma, a life-threatening condition. We have people even on our team who are living with stage four disease. So, when Ken talks about things slowing, that’s not what we want to hear. We want to hear two things. One is, we can accelerate a development of new medicine. And ideally—because this is an issue certainly in the U.S., but I think worldwide, that by speeding the process, cutting through red tape, improving procedures and us participating, the cost can be less as well.

And when we talk about cancer, the costs are going through the roof as you know for people living with chronic cancer. And you know so well, Andy, people who are on some of the medicines that you’ve come out with at Merck. Where people used to die unfortunately in short order, are living a much longer life thanks to new medicines. We want it to happen faster and be financially achievable. Andy, any comment about the pace of science?

Andy Lee:

Yeah, I would like to make a couple of comments about that. We often hear the sort of story that 80 percent of clinical trials don’t recruit on time, et cetera. We do immense feasibility. Once we have designed a protocol, we send it out to all of the countries that could potentially work with us. We have staff in 47 countries. And they look at two areas of interest. One is the medical durability, is the comparator the one we use in our country. Is the protocol designed the way we practice clinical medicine, not clinical research medicine?

And will that enable us to recruit the patients? That’s the first level. The second level we look at is to ask the question, is this operationally feasible? Can we source the comparator? Do the clinical sites have the equipment? How would we have to ship the biological samples around the world? And based on medical durability and the operational durability, we do a site selection. And we run the indicators through a Monte Carlo simulation. And we simulate this trial. What if we took three countries out? What if we added this more sites? What if we changed this inclusion?

And we come up with a model of what the recruitment would look like. And recruit about 80 percent of our trials according to our model. So, about 80 percent of our trials recruit on our model time. And then if we look at the typical time for drug development, it has been from eight to 10 years for many years in the industry. And when we look at some of the development timelines now—the cycle times. Pembrolizumab (Keytruda), for example, from first study until first approval, was 60 percent reduction in time.

We were looking in the four-year time period. And we are looking at five or six years for many indications. And so, we’ve halved that cycle time for some of the newer oncology products. And there are a number of reasons we’ve done that. One is we have found operational efficiencies. Two is the trial design has enabled us to interim analysis with independent data monitoring committees to assist with that. I’d also like to put in a positive plug for the regulators.

I do believe—and I’ll talk specifically about the FDA, because they are the agency for the United States. They have revolutionized the way they approach the designs and the way they review the data. And they have breakthrough designation status they’ll give to compounds that are really looking like they have strong efficacy. And so, the approval process through the agency has improved remarkably. And they’re open to adaptive designs. And they are open to interim analysis. And they are open to all sorts of things.

So, I really wanted to give credit to our agency who has said, “Where there’s a need for breakthrough medications, we’ll try to find the path.” And so, I do believe there’s a real positive side to this. The challenge is the market is saturated. We have now more than 25 PD1s in development. And to put the 25th one in there, they are so far behind in development. I wonder what that does. It clogs up the system. So, when you look at how can we influence sites, at the top sites we only get one or two patients.

And we compete with 50, 60, 70, 80 other sponsors. And so, it becomes so saturated that, that site has to learn to do systems and process with 70 companies. And what they are doing is almost hedging. They are not focusing on certain things. So, in those cancer centers, they offer treatment for all lines of therapy and all types of cancer, the specialized and nonspecialized. And we are moving out of that sort of geography and moving it community-based oncology practices where it’s less saturated, and we can actually have more traction there and be able to engage more with the clinical trial enterprise for the good of the patients.

Andrew Schorr:

Ken, you write books about all kinds of issues around this. So, if we are getting—particularly in oncology to have trials offered at the community practice where those doctors work night and day—the nurses. They are really stretched. More and more cancers, genomic subtypes, most sophisticated testing. How—what would you say the patient can do. T.J. talked about it a little bit. What would you recommend to patients so that at that community oncology practice the patient and the family can kind of discover what may be available for them as Merck and other companies try to get these trials distributed?

Ken Getz:
Right, well you—talk about the whole enchilada, Andrew. You’re really touching on it. It’s also very exciting times for patients, not just cancer patients, but patients that are dealing with any chronic and severe illness today. And it’s really all about more of a partnership with the clinical care environment and clinical research. And of course, at the heart of it is the patients and their family being as informed as possible, sharing their electronic health and medical information so that they can be connected to trials that might be appropriate for them.

But it’s moving—as Andy said, away from the classic places where trials used to be conducted. And in many cases, they were at these dedicated centers that only conducted clinical trials. It’s a very competitive environment now for patients. So, many sponsor companies like Merck and others are looking at clinical care settings and moving into communities or, in some cases, large health systems where you can have clinical research professionals who will supplement and provide support to the healthcare providers, so they’re not stretched too thin.

But so that they have the clinical research capability onsite at the point of care. For patients it’s a great opportunity because now they have the opportunity to get their own healthcare or treating physician and treating nurse involved in a clinical trial as part of their overall care. And we expect to see more of that over time. We expect to see other virtual trials or opportunities for patients to participate in the comfort of their own home tied in with their clinical care setting.

And all of this is relatively new to the whole world of clinical trials and the investigation of experimental medications.

Andrew Schorr:

You touched on something I just want to follow up on. I’ve heard of this term site-less trials where you said you participate in your home. So, T.J. had to go from Ft. Lauderdale to Tampa. I had to go from Seattle to Houston. There are not—this is a big deal, especially if you have little kids as I did, he has. So—and away from work and whatever your situation is. So, is technology going to come in play so Andy can get the data he needs for the FDA, but that we can have technology help accrue that data in a more efficient way.

Ken Getz:

And I’ll say absolutely. And my colleagues here today I’m sure can comment on this as well. But absolutely. We are seeing wearable technologies and mobile applications that now have the ability measure vital signs and other important baseline information in a validated manner. There are ways that you can access a specific facility for a highly specialized test, specialized imaging for example where the technician can evaluate it remotely. Blood can be drawn at remote locations as well.

So, there are lots of places where we have sort of this more flexible environment that can cater more to the patients and less about a specific physical facility where you have to go to participate in a trial.

Andrew Schorr:
T.J., I want to talk to you about diversity. So, you and I are kind of middle-class white guys. But we want to know how new medicines work for a variety of populations, ethnically, economic groups, et cetera. And Andy needs that data. And he goes to the FDA, and the FDA says, “Well, do you have Hispanic people? Do you have Asian people? Do you have African American people?” or whatever the country is because he works globally. And they say, “We want to understand are there differences?”

How are we doing with that. How can we make a difference there so that we really know what medicines make a difference for broader and also distinct populations?

T.J. Sharpe:
I’m sure Ken can back up some of these things with more hard data than I can. I know that different populations have different levels of trust with the medical system. One thing that you and I both experienced was a lack of options—a lack of good options. And when you get into dire straits, you tend to be a little more trustful of anything that comes along. But we have serious or chronic conditions that have proven treatments that might not be the most effective for certain populations.

And we’re not able to broad the scope to these minority populations or populations that don’t have access to NCI designated cancer centers or top-notch medical facilities. They are not able to get either in a trial that is looking for a drug that would help them or even get access to medicines that have been recently approved simply because their healthcare situation doesn’t allow it. Whether that’s a lack of insurance, a lack of healthcare literacy or simply a mistrust of—there’s a lot of generational mistrust I think in some communities of the clinical trial system.

So, as an advocate, I certainly push caretakers especially—and children caregivers for older populations who are maybe first or second-generation Americans to help facilitate a conversation between the medical professional who’s trusted and a patient that might not be able to get or rely on the information they’re given. Because it really will speak to populations that don’t get the opportunities that you and I have gotten simply because they are either not aware, or there is a barrier there to get to that medical professional.

Ken Getz:

I appreciate, T.J., you mentioned CISCRP. That’s one of the things that we’ve focused on for 18 years is bringing clinical research education into major metropolitan areas around the U.S. and parts of northern and western Europe where we plan for several months, and then we put on what we call an Aware for All events. And we really work very hard to encourage participation by—or from patients based within minority or underserved communities.

And I’m happy to say that we’ve had a lot of success with that. These are really difficult communities to reach through a lot of the traditional approaches. We have to rely on community centers and clergy and other approaches to really help these communities, for a lot of the reasons T.J. mentioned, trust the educational information, and come out to learn more. And I’m happy to say we’re seeing more and more people of diverse backgrounds that are curious and interested in learning more about clinical research, especially knowing that representative populations provide more information that can inform treatment for different types of patient sub-populations.

Andrew Schorr:
I want to go to Andy in a second. Andy, just one second. I wanted to mention and call out—and Andy’s company has been a leader in this. He was talking about PD1 and all of that. But drugs that have been breakthrough in immunotherapy for people like T.J. where—and it’s being explored in broader cancers where otherwise life was going to be short. And how to activate the immune system and really fight the cancer in people living long term. So, the people in those trials—and certainly there were people in the melanoma trials like yourself T.J.

Lung cancer trials and increasingly now others who did get tomorrow’s medicine today. Andy talked about accelerated approval which is great. So, that’s the impetus for the patient and the family. Is there the chance to get tomorrow’s medicine today? Now the obstacles may be distrust. You talked about that, Ken. And also, is maybe accessibility. Is it as a clinic near you? And Andy you talked about pushing that out. And then sometimes it’s related to cost.

Now is there anything that sponsors can do, Andy, related to the costs that people may have in being in certain trials? Where do we stand with that?

Andy Lee:

Yeah, so I’ll just touch on the distribution first and then get into the costs because they are linked. When we prosecute global trials—we’ve had a very U.S.-centric discussion so far. But cancers present differently in different geographic regions of the world. And so, when we want speed out of our trials. You want me to shorten that timeline and get drugs to market quickly. I do it internationally and in some cancers like esophageal cancer or some of the gastrointestinal cancers, Asia has a much higher prevalence of these cancers.

And we do a greater proportion of work there. We always include multi-country studies. And U.S. may have a greater proportion in other areas. So, we balance that out to optimize speed. Of course, with clinical trials the cost structure around the globe is very different. But let’s talk about U.S. We have spoken about a saturated core of clinical trial sites that we all go to. And I speak generally now for all sponsors. And we are all looking to optimize and get great efficiency.

At the same time, we realize we have many underrepresented geographies and ethnic groups—and not just ethnic groups, but under resourced populations. And so, what we’ve been thinking about is how can we support people, and support people at all levels. And so, we start off with thinking about the cost structure, and we obviously pay clinical sites for what they do. But we will support all sorts of things. We’ve been negotiating with Uber and Lyft, so we can build that into automated transport for patients.

Again, the IRB has to approve that. We are looking at ways to augment that they are not out-of-pocket for things. And we’ve been talking a lot with a group called Lazarex Foundation who has really expanded into under resourced communities and found ways to ensure that they have daycare and different access for those patients. We have worked extensively now to look at outreach programs into communities that typically wouldn’t be in trials. We are focusing in two areas right now as we speak.

One is next generation of HIV medicines, and the other one is in prostate cancer. And we’ve got a large program rolling out in prostate cancer. So, what we are doing is going into sites and we have put together training videos and training materials. And we are looking at cultural competency. So, it starts at the site. Are they culturally competent to engage a different community? And we’ve spoken about working with the community churches, community education systems.

And so that starts with cultural competency. I have a woman, Madelyn Goday, who works on this day and night in my organization. And she’s very strong at this. It’s early days, but if we can show that it works in one or two therapeutic areas and cancer types, we’d expand it further and further. But we can’t just have a shotgun approach and just go and do 100 sites and hope it works. Hope isn’t a good strategy. We are working systematically to engage different people. And as appropriate and approved by ethics committees, we will support all of these communities and help build infrastructure and capacity.

Those are important things for us. But as I said, where appropriate and where it’s sustainable. We can’t just throw money at something in the hopes something sticks. We have to have something sustainable and it goes to what Ken says, and that’s education and providing resources and materials. And we’ve used quite a lot of Ken’s materials in multiple clinical trials. Thank you for that, Ken. It’s been really helpful for us.

Andrew Schorr:

Great. I wanted to note for your audience. If you have a question, send it to questions@patientpower.info. We have expert panelists here. And this is really—we are all in this together. I think you hear the dedication from Andy at Merck and T.J. as a patient advocate and Ken as a professor and founder of organizations devoted to this. We want obviously accelerate medicines, but have the accurate data of how it affects different people, who is it right for so that the regulators—and thank you for what you said about the FDA here in the U.S., has the information to make a decision on should this medicine be available for people with that diagnosis.

Okay, so what about staying in the trial. So, T.J., how long—let’s take with the Keytruda trial or one of them. How long were you in to for?

T.J. Sharpe:

Nearly four years. Three-and-a-half years.

Andrew Schorr:

Were there ever times when you said, “I’m done. I want to bail out.” You know.

T.J. Sharpe:

I’ll be very careful how I answer this question for Andy’s sake.

Andy Lee:

It’s okay, T.J., we’re friends.

T.J. Sharpe:

No, probably the biggest crossroads I ever came to was when one of my tumors started growing about a year into it. And we weren’t sure if the medicine stopped working or not. We didn’t know what to do. And as it turned out, it was still working. And I think was just one spot that wasn’t responding. But everything else had responded great. However, at the point, as a patient, you’re thinking about yourself first and your family first and the trial second. It’s easy to stay compliant on a trial when things are going well.

But when you’re ahead of the medicine in some ways, and I think patients with chronic illnesses or in some cases rare diseases, are almost more knowledgeable than some of their doctors or the trial protocols about when they’re stopping. They don’t have the luxury of finishing out a protocol and seeing where their disease journey takes them. And the best example I can give of this is a very passionate advocate by the name of Jack Wheelen who we unfortunately lost a couple of years ago, but whose influence has kind of dominated the patient advocacy world for the last decade or so.

And Jack was able to monitor his health almost better than a doctor. And he knew when his trials weren’t working. When we get to that point in a clinical trial setting where we know the medicine is not being effective or where a patient would be better served to move on to another treatment. That’s when we are going to take the next step in clinical research, because now we’re aligning the trial design and the trial goals with a patient and a patient’s family’s treatment goals. And as those two points merge, that’s where clinical research becomes that much more effective as a care option.

Andrew Schorr:

That was well said. And I think with all those trials, you’re right, the team—that care team, what’s right for you at that time. Obviously to get the data, but also not at all costs. In other words, if the data is showing something is no longer effective for you, is there another treatment or a trial? I’ll just share my story for a second. So, I was in a phase two trial of combination therapies—which are increasingly common certainly in oncology. And after three months—halfway in the trial, my blood was kind of cleaned up.

And I had nausea and some other side effects. And I said to the trial coordinator, “You know, I think I’d like to stop.” And she said, “You know, our belief is that you still have microscopic illness in your bone marrow—in this case with the blood cancer, and the additional three months in this protocol will make a long-term difference for you. That’s what we believe.” They didn’t have the answer, but that’s what they believed. You know what? I stuck it out. She was right. I had 17-year remission.

If I’d stopped after three months, would I have? So, it’s a dialogue with the care team Andy, right? It’s this ongoing discussion not just entering the trial, but remaining in the trial, correct?

Andy Lee:

Yes. Absolutely. And I just wanted to impress a really important thing. People talk about people dropping out of trials. In cancer trials we see extremely low drop out. I mean these are potentially lifesaving medicines for all of the companies. But what we do want to make sure about is that when there is progression of disease, and it’s shown that the drug—whichever it is, the control arm or the active arm or the new agent, where there is progression of disease that they get the best available therapy.

And so that often contaminates trials because we have the crossover effect that now they are getting maybe the experimental agent in the standard of care type of thing. But most important thing for us is to track the survival of the patient, regardless of whether they go on another therapy. And we have put a tremendous amount of effort into looking at the informed consent and making sure we work with IRB to track patients long term survival.

Because as you’ve said, you may have a short-term issue that shows that the drug may not be working short term, but long term it may have prolonged and profound effects. Positive or negative, we don’t know that. And so, what we like to do is get long term survival. And we ask patients to consider when they sign the consent for whatever trial and whichever sponsor is sponsoring this, is to consider that knowing their status throughout their treatment—whether it’s on a sponsor’s drug or another sponsor’s drug or x therapy. It is really important — and I ask people to think about that.

Because that really helps us get as much data out of the individual treatment as possible. And that may prevent nonrequired trials in the future or it may say, “Wow, that really informed.” And we’d like to inform all cancer patients. If data we generate can inform other therapies, we certainly want to do that. We do not want to do wasteful clinical trials. So, tracking patients long term or patients—the message to patients is being cognizant of letting the sponsor—and the sponsor could be an institution. Letting them know your status is really important. All they want to know is are you dead or alive.

Andrew Schorr:

In the end, just one thing is, are we partners. In the end, our viewers here, are we your partner? And can we feel that not just for their doctor but you guys behind the scenes with the labs and everything, that in the end we are partners. And unless we see it that way, we won’t get anywhere.

Andy Lee:

Absolutely. I’m glad you used the term partners. Because when we’ve done a prep for this people have said, “Are they investors in the thing?” So, yes, patients invest their time and everything, but they are partners in research. They are contributing so much. They are contributing—they are going into the absolute unknown. And there is an immense trust level that is there. And we owe that back as research professionals is to treat people with respect, dignity and as partners, to make information available, to publish our data to get it out there as quickly as possible. And to make sure we get that back into the participant’s sort of hands.

Andrew Schorr:

So, Ken, how are we doing on that because you go back over the years and people say, “I don’t want to be in a trial because I’ll be a guinea pig,” and respect was not seen as part of it.

Ken Getz:
Well, that’s also a bit of a myth, right? You had a few that claimed that they felt the process made them feel like a guinea pig. The vast majority of people, over 90% of people who participate in a trial, would do it again. So, once they get past that unfamiliar area where they’ve perhaps only heard a few case examples or a few very vocal people who had bad experiences. Once they’ve done it themselves or they’ve been able to work with a group of advocates that really help them think about this process, and they become more educated, generally they’re very impressed with the level of professionalism, the compassion that exists at all levels.

I work with so many professionals—science professionals and pharmaceutical companies and at the research centers, and they all share that kind of commitment that Andy just mentioned. There’s a real desire to partner with the patient to really inform them. I would say one place where we need to see much, much more however is in the return of clinical trial results in a plain language to people who’ve been in trials. That’s a place where as an enterprise—government, research sponsors as well as industry have not really made this a standard practice at this point. And that’s one thing that we’re really working on actively.

Andrew Schorr:

Right. Great. So, T.J., you and I are investors—and Ken used that term and Andy used it, and I’ve always believed it. We are investors of our tissue, our body, our future to help other people and hopefully help ourselves. And certainly, for profit companies that may greatly benefit if they have a blockbuster therapy. But we need to be kept informed in the long term, right T.J.? We want to know what a difference our participation made.

T.J. Sharpe:

Certainly. And I think to echo what both Andy and Ken said is that patients do become partners. Patients who are involved in clinical research, a significant chunk become altruistically invested. I’ve heard more than once, “Even if this doesn’t help me, I’m glad I participated because it might help somebody else.” I know I’ve felt like that, and I’d venture that you’ve had some of that too, Andrew on your journey. So, it’s only—it’s at the very minimal fair, and it’s certainly very justified to expect as a co-participant in this.

And as kind of a co-creator of science with sites and sponsors that we understand what has come of our sacrifice and our time dedication to helping science out. We shouldn’t have to find it out through press releases from ASCO or hope that we hear about it on the nightly news. We deserve to hear what has happened. Not just because it can affect us as people and as patients, but that we put a lot into this too. And then we did our part to further medical research and we want to be part of the—whatever the end of the trial ends up being. We want to be aware of that. Not just for personal knowledge, but to know that it’s going to help this many other people.

Andrew Schorr:

Right, to be honored. So, Andy, at Merck you’ve established some internet platforms in particular related to keeping people informed, right?

Andy Lee:

Well, we’ve got an internet platform that people can log onto. I’m happy to share that with you; in which they can get access to a list of our trials. So, I didn’t prepare this but especially, but I did make a handmade note. And if anyone wants, it’s a very simple log on. Andrew Schorr:
You’re a great artist.

Andy Lee:

And it’s a simple one. What that will get you access to is two main important things. One is it gives access to information about clinical trials. We have a tab on there that tells everyone about the phases of clinical trials and what to expect in a trial. So, it’s an educational part. Then we have a lot of information about the Keytruda clinical trials were, are running, and they’re called keynote trials. And there you can look at the different indications. And you can look up and it has a telephone number you can call.

Now I must stress is that we run over 1,000 clinical trials in oncology. But many of them are not sponsored by us, they are investigator sponsored trials. So, you can go to clinics, and they run their own clinical trials that are not sponsor-related. And the NCI runs their clinical trials. So, there are a lot of different sources. And many companies will have clinical trials. We also have the website clinicaltrials.gov. I’ve had to use that in the last two days for a colleague.

And you can navigate that and look for different types of trials. And you can look at different products and everything. It’s not perfect. But at least it’s a place to go to. And I don’t want to sound as if I’m one sponsor centric. Many other companies have access to websites, and they really want to try and enhance and direct people to the clinical trials sites at which they are working.

Andrew Schorr:

Right, absolutely. And then you were working at the industry level with a group called TransCelerate, and I know T.J. is involved too, to try and establish common procedures as you establish trial sites, as you have communication, as you have training, right? So, that hopefully all boats will rise, right?

Andy Lee:

That’s correct. TransCelerate is a group that formed about eight or nine years ago. There were 10 initial member companies. I was a founder member of that. And we got together to say, “We have to improve operational efficiency.” So, we do not collaborate on molecular structures and those types—that’s competitive. We collaborate on what we call precompetitive, procompetitive aspects which says, “If we all work together to improve something, we’ll all get the benefit of this.” And we share it publicly.

There’s a website, you can look at it. But we’ve looked at standardizing protocols. We have a common protocol template. We’ve adopted that at our companies, so have other sponsors. The protocol can be developed in a standardized way. We’ve looked at standardizing ways where we can improve monitoring. We’re looking now at ways that we can work with investigative sites through i-platforms, shared investigative platforms. So, a clinical trial site has to provide the information for us as a sponsor and use the exact same standardized questionnaire and information for any other sponsor through a standard portal.

So, we are trying to reduce the burden on clinical trial sites. And we’ve plugged away for many years, and we are seeing greater traction there. We are seeing more efficiency, more standardization. We are seeing greater quality, less rework. And so, while it’s hard to quantify this, what we believe is that the sites are freed up of some of the more burdensome things, and they can direct their attention towards patients, patient safety, and access to clinical trials. So, the work may not be directly related to access for a cancer patient into a cancer trial, but there’s a lot of tangential spin-off of making a site more efficient so they can put their resources and energy in the right place.

Andrew Schorr:

Well, thank you for that effort and your leadership. So, Ken, you’ve been around this a long time. And you’ve deal with all the companies and the government and the various agencies. And as you know, in some quarters there’s a distrust for pharma. We mentioned cancer that you get the price tag of a drug, and it’s very expensive. And some people are struggling to pay for it. And there’s just frustration about it. And often in the news media they are the bad guys who are called out for unethical procedure or something that went awry.

So, how are we doing there in overcoming that because we talk to Andy, he seems very ethical, dedicated guy representing a company that’s been around I think well over 100 years. So, how are we doing to move clinical trials on in this area when people aren’t sure what to make of pharma.

Ken Getz:

Yeah. It’s a huge issue, Andrew. And I think part of the challenge is that all it takes is one questionable behavior, and it makes it difficult for the reputation of the entire industry. Right now, we are dealing with major pharma companies that are actually being fined for having contributed—a judgement, having contributed to the opioid crisis. And when you start looking at some companies aggressive marketing tactics, right? It really sort of sheds a darker light on a lot of the great work that companies do.

What we look at, at the Tufts Center for the Study of Drug Development at the School of Medicine. We look at the overall output, the level of innovation that’s coming from the industry today. And we look at the number of complaints that have been filed with the FDA and other regulatory agencies around the world. And what we see is tremendous growth in the innovation and the quality of the innovation—drugs like Keytruda and other cancer immunotherapies. What an exciting area.

We see that the vast majority of companies really support and live by highly ethical, highly professional, highly compassionate approaches because they all know that it takes just one questionable issue that can really tarnish the reputation of every company operating in the industry. So—again, Andy also mentioned just how regulated we are as an industry, the fact that we have ethical review committees and data safety monitoring boards and so many other external agencies that help to oversee the work that’s done here.

So, I would say for patients who are thinking about clinical trials, it’s good to know the history. It’s good to know what you need to do to protect yourself. But the vast majority find that the people they deal with are ethical, they are professional, they are compassionate. And, as I mentioned, over 90% of people who get involved in trials say that they would do it again.

Andrew Schorr:

Thank you. That was a wonderful response. Andy, you mentioned earlier about starting research with the sickest people basically, where there are no options. But one of the questions that came in is, “Are trials only for the sickest people or are there of all those trials you talked about opportunities for people who maybe are newly diagnosed or could be their fairly initial therapy?

Andy Lee:

Yeah, great question. And thank you to the person who asked that. And the answer is that we start in people—because we don’t know if our experimental agent will work. And everyone assumes that new medicines are all going to succeed. And we work in research and researcher because of that many things fail very early on. They fail in phase one before anyone hears of it. It’s normally a code number at that point. And we may just not make the drug soluble enough, or it may not be distributed enough.

So, we may have a thing that works in a test tube or a petri dish. But to get that into humans and make sure that it’s safe at the dosage we use often fails. We just don’t progress far enough. So, what we want to make sure of is that firstly the drugs are safe. And there’s a trade-off between safety and efficacy. We’re constantly trading off. And so, what we do is we look at that and say when someone has no option and we want to get an option going, that’s where we start.

We’ve actually moved down the disease scale, and we’ve come into adjuvant treatment or secondary prevention. And we’ve gone into newer adjuvant is when you have a small tumor is we pre-treat to manage that tumor before surgery is done. And post-surgery we hope that there’s limited treatment or no treatment. And we actually have removed the cancer, and there would be no evidence of disease. But. of course, using the word cured is something we try not to do, because we prefer to use no evidence of disease.

But absolutely. And the next strategy is prevention of cancer. Our company does a lot of vaccinations in women’s health. We have a product that protects against human papilloma virus which is a precursor for cervical cancer. So, people who are vaccinated with this particular product—and I’m deliberately not using brand names for obvious reasons. But when you vaccinate for HPV, you essentially are preventing the likelihood of a cervical cancer. And there are now prospects in many disease areas where either vaccination or early treatment gives you a tremendous positive prognosis of not getting the disease later on in life.

The answer to your question is yes, we are absolutely looking at ways to prevent getting to a very advanced stage which is very costly to manage and very emotional and stressful and difficult.

Andrew Schorr:

I want to thank you. I just want to get a final comment on what you would say to patients or family member. And I want to start with you, Ken. What do you want patients right now to know so that—what tips would you give them so that they’d consider being part of clinical research or stay in clinical research and the benefit it could be for them.

Ken Getz:

I will say really two things. The first is there’s just a tremendous amount of information out there, and we recommend education before participation. So, do your homework and engage family and friends and people you meet and trust to help you make the decision. And the second point comes off of that. And that is this is not a decision you make alone. Really bring in your treating physician, your nurse. Bring in your support network. And chances are you will learn a lot, and you might even find a trial that is right for you.

Andrew Schorr:

Right. And Andy, what about you? A final point—what would you say to a friend or family member or colleague related to considering trials today.

Andy Lee:

We get this question every single day. And we get it from patients in need. And my answer is we are all patients. We are all going to face this as professionals in our job or professionals outside. And so, I say community of practice. And disease hits all of levels of society in all education professions, et cetera. And so, my thing is to encourage people to do what Ken has said. Work as a team. Get multiple inputs.

And I am sponsor agnostic. Get the best therapy that is available. And that may be the best care option—as I said, the ecosystem in which you get the care is really important as well as the medicines that you get. So, have the discussion. Trust the medical professionals, they are very skilled out there. They are extremely well educated. And I just urge people, “Don’t think on two clicks on Google you are going to solve what your treatment option is.” Really discuss it with people because not all the options are public, and there is not enough information available about how to manage the whole disease through the entire enterprise. Trust the professionals.

Andrew Schorr:

Well said. And T.J., you and I are alive today because of trials. What do you want—what’s the thing you want to leave our viewers with?

T.J. Sharpe:

That they don’t have to be involved in clinical research. I think that’s an important distinction to make. And it’s going to pull together what Andy and Ken said that clinical research should not be considered a hail mary or last gasp option. If you are a patient—and we are all going to be patients as Andy mentioned. You want the best care for you. You want to be able to weigh all of your options. And if you are not considering clinical research, if you don’t know about it or aren’t able to get the information you need about it, then you are not going to be able to make the best healthcare decision long term for your health.

So, take that information that you can get. Find the trusted sources. Be able to reach out to advocates or colleagues or someone that you know that would have the disease or can connect you with good information. And be your own advocate—a little cliché, but really own that healthcare information. And once you are able to collect all of the different treatment options, then you consult with your professional medical team as to what the plan forward—the best plan forward for your individual situation would be.

Andrew Schorr:

Right. T.J., my friend, thank you. It’s a delight to see you again. Andy, with Merck, thank you so much for being with us and bringing your years of expertise. And, Ken, being at an independent non-profit center and also at Tufts University there, thank you for all the work you do. I want to thank the Patient Empowerment Network for pulling this all together. And the sponsors who supported us in this effort, AbbVie Inc., Celgene Corporation. Daiichi Sankyo and Novartis.

All these companies and I’m sure many more, working so that research can move forward. We can be true partners in it. And hopefully get tomorrow’s medicine today to make a difference for the community and live a long life, and hopefully a cure, right? I’m Andrew Schorr in California. Remember, knowledge can be the best medicine of all.


Please remember the opinions expressed on Patient Empowerment Network (PEN) are not necessarily the views of our sponsors, contributors, partners or PEN. Our discussions are not a substitute for seeking medical advice or care from your own doctor. That’s how you’ll get care that’s most appropriate for you.

Clinical Trial Mythbusters: How Does Medicare or Medicaid Impact My Ability to Participate in a Clinical Trial?

 

Clinical Trial Mythbusters: How Does Medicare or Medicaid Impact My Ability to Participate in a Clinical Trial? from Patient Empowerment Network on Vimeo.

Downloadable Guide

Cancer patients are living longer as a result of clinical trials that test new treatments, therapies, procedures, or new ways of using known treatments.

Watch along as a panel of experts from the Diverse Cancer Communities Working Group (CWG) Sustainable Healthy Communities, LLC, Baptist Memorial Hospital–Memphis, and the American Cancer Society Cancer Action Network (ACS CAN) explore the questions:

  • What do patients and their support networks need to know about clinical trials as an option for cancer treatment if they are insured through Medicare or Medicaid?
  • What requirements differ from region to region and what is covered or not covered?

Transcript:

Laura Levaas:             

Hello, and welcome to this Patient Empowerment Network Clinical Trial MythBusters program on a very, very important topic, what impact does Medicaid or Medicare have on a patient’s ability to participate in a clinical trial. My name is Laura Levaas, and I’m the lung cancer community manager for Patient Power. I’m also a Stage 4 lung cancer survivor. I’m two years out from diagnosis, and I’m also on Medicaid. So, this is a topic that’s really important to me on a personal level.

This program is produced by Patient Power.  We want to thank the following companies who provided financial support to make this possible. While they don’t have editorial control, we appreciate the support of AbbVie Inc., Celgene Corporation, Daiichi Sankyo, and Novartis for their support.

Today we are joined by some really amazing guests, the first being Mark Fleury from the American Cancer Society Cancer Action Network out of Washington DC, followed by Jeanne Regnante, also out of Washington DC, and Jeanne is with the Diverse Cancer Communities Working Group, Sustainable Healthy Communities, and last but not least, nurse navigator Laura McHugh from the Baptist Cancer Center in Memphis, Tennessee. Welcome to all of our guests today. Thank you for joining us.

So, Mark Fleury, Mark is interesting because he has an understanding, a very deep understanding, about this issue from a regulatory and research perspective. He’s going to share with us what he’s learned about barriers in clinical trial participation and solutions to overcome some of those options.

Jeanne is going to share her viewpoint as part of the Diverse Cancer Communities Working Group. She helps share information about access to care treatment and inclusion in clinical trials for underserved populations.

And Laura McHugh who is joining us by phone (she is a friend of a friend of mine, and she’s really amazing) is a nurse navigator who has worked in the cancer space for 24 years. And she helps guide people in underserved communities every day as part of her working life. She works with Medicare and Medicaid patients on the daily. So, we’re looking forward to hearing from her.

So, back to our program, patients are living longer as a result of clinical trials that test new treatments, therapies, and procedures, or new ways of using known treatments for new ways. The myth here behind Clinical Trial MythBusters today is that being in a clinical trial isn’t covered by medical insurance particularly for Medicaid or Medicare patients. I know for me personally I’m interested in being in a clinical trial and I’m on Medicaid but I don’t even know what that means. So, I definitely need some guidance.

So, as we’re talking about this today, if you have any questions about if you’re a patient yourself or you’re a support person for a patient that has cancer or any kind of disease wanting to know about clinical trials on Medicare or Medicaid, we’re here to help you. Send your questions to questions@patientpower.info. So, viewers who are joining us today thank you again. If you’re on Medicare or Medicaid, what do you even do if you’re presented with the option to participate in a clinical trial to treat your condition? Let’s talk with Mark Fleury. Hi Mark.

Mark Fleury:              

Hello Laura. Thanks for having me on.

Laura Levaas:             

Yeah. We’re so, so grateful to have you on our program today because you have such a deep knowledge in this industry and on this topic. Can you tell us real briefly what exactly you do for the Cancer Action Network? And then I’d like to talk to you about barriers around Medicare and Medicaid.

Mark Fleury:              

Sure. So, I work for the American Cancer Society Cancer Action Network. We’re the policy and advocacy arm of the American Cancer Society, and we focus on public policy, so that’s regulation, laws that impact cancer patients. And specifically, my work deals with policies around research and drug and device development, so how can we get those findings that happen in the laboratory into the clinic. And specifically, that goes through clinical trials. So, I’ve spent the last couple of years with a large partnership of other stakeholders taking a really deep dive into looking at clinical trials and all of the challenges patients have in getting themselves enrolled as a part of those trials.

Laura Levaas:             

Good. We look forward to hearing more. Can you tell us a little bit about the current state of clinical trial participation in the US right now?

Mark Fleury:              

Sure. So, there’s not real solid numbers, but we believe somewhere between 6 to 7 percent of US cancer patients participate in a clinical trial right now. So, that’s a fairly low lumber overall, and it’s also a fairly low proportion of the patients who would be interested. Research has found that between 50 and 70 percent of patients would say yes to participating in a clinical trial if they were asked. But unfortunately, many are not asked. And some of those who are asked are unable to enroll for a variety of external reasons. One of the things that we do know is that the people who do enroll in clinical trials tend to be less diverse and better off financially than the overall population with cancer.

Laura Levaas:             

Okay. What are some of the barriers around Medicare and Medicaid patients who want to get involved in a clinical trial?

Mark Fleury:              

Sure. So, obviously, first of all, there has to be a clinical trial for the patient based on your clinical characteristics. But assuming that that is the case, for a patient to enroll in a clinical trial, it’s critical that their insurance cover the routine care costs of that clinical trial. In other words, there are costs in a clinical trial that a patient would see regardless if they were in a clinical trial not. Say, for example, the first step of any treatment is a surgery and then the second step in normal care would be one drug but in a clinical trial it’s a different drug.

Well, regardless, you’re always gonna get the surgery. It’s important that insurance cover that routine part of the clinical trial. And unfortunately, historically, that’s not always been the case. Fortunately, in Medicare, they have covered that since 2000. That is not the case universally for Medicaid. And we can talk a little bit more about that later if you’d like.

Laura Levaas:             

Okay. Perfect. I would definitely like to follow up on that topic seeing as I’m a Medicaid person myself. Can you touch briefly on what actually is different between the two programs in terms of clinical trial, the actual coverage? You mentioned routine care; is that for both programs?

Mark Fleury:              

Well, so what’s important to note is that Medicare is a federally administered program. And so, there is one universal federal policy, and if you’re in Medicare, it doesn’t matter if you’re in Florida or if you’re in Idaho, the policies are identical. Medicaid is an insurance program that while partially funded by federal dollars, it’s administered by each state. And as such, each state has quite different policies. So, if you’ve see one Medicare policy, it’s uniform. If you’ve seen one Medicaid policy, it’s only relevant in the state in which you happen to be. So, it could vary significantly from state to state.

Laura Levaas:             

Right. And so, depending on your state, you would need to follow up with your local maybe human services office to get specific questions answered.

Mark Fleury:              

That’s correct. Yeah. There are some resources (and I think we can provide those at the end of the webinar) where generally speaking some states have passed laws or signed agreements in which their Medicaid programs have to cover those routine care costs in Medicaid. And we can certainly make available those states. But even within those states, it’s important to look closely at the policies. For example, in Medicare, Medicare also covers any adverse events. So say, for example, while you’re being treated, you had to be admitted to an ICU for heaven forbid a heart attack or something like that. Medicare pays for all of those unexpected expenses. And that coverage may vary state by state in Medicaid.

Laura Levaas:

Okay. Thank you, Mark. We’re looking forward to those resources. And for those of you watching, we will definitely be providing a downloadable guide with all sorts of resources to help you. Thanks Mark.

Mark Fleury:              

You’re welcome.

Laura Levaas:             

Hi Jeanne.

Jeanne Regnante:        

Hey Laura.

Laura Levaas:             

Okay. I can’t wait to talk to you about this. I have so many questions. I feel like we could talk for an hour. So, aside from the myth, I came into this thinking, “I’m on Medicaid; I probably can’t get into a clinical trial when and if I get to that point.” And then also, “If I am, it’s probably cost prohibitive because I’m on a fixed income.” So, is participating in a clinical trial expensive or cost prohibitive if you’re on Medicare or Medicaid like I thought? I mean, I know Mark touched on some of the issues, but what would you say? How would you answer that?

Jeanne Regnante:        

For low-income patients, the cost of routine care and logistic support needed during a clinical trial is certainly a barrier to participation. And Mark pointed out some of these costs. But specifically in patients in rural communities, remote communities, aging population, children, patients with cognitive disabilities or physical disabilities. These are the same patients who have low access to care in general.

And covering the cost for routine care in a clinical trial and also the logistic support is a clear barrier to participation. So, there are clear barriers there, travel, housing, parking, paying for food, on having access to clinical trials not only for routine care costs like Mark alluded to but also logistical support being included in the clinical trials. So, all of those things are barriers.

Laura Levaas:             

And would you say that seniors are also part of this underserved population?

Jeanne Regnante:        

Absolutely, especially seniors that live alone, that are in remote rural areas in the United States. And remember, that’s 20 percent of the population, aging population, in those areas. So, clearly, we need to do better to engage those patients in care and also clinical trials.

Laura Levaas:             

So, is it possible for us to draw any conclusions about how many people are on Medicare or Medicaid right now in the US? I did a little bit of internet sleuthing mainly through the Centers for Medicare and Medicaid, and it seems like there – the numbers that I came up with were pretty high, and it’s almost like 40 percent of the population is on Medicare or Medicaid. And so, has it –

Jeanne Regnante:        

That’s absolutely true. Look at by the numbers, there is 329 million people living in the United States, and that’s according to the last census, which is a hot topic these days. There is 60 million people on Medicare, beneficiaries, and about 66 million people Medicaid. So, together, that represents about 40 percent of the population. And we have to remember kids. So, there are 7 million patients on CHIP, which is part of the Medicaid program. So, if you include percentage of people on Medicaid plus kids on CHIP, that’s 22 percent of the population.

Laura Levaas:             

So, then circling it back around to clinical trial participation, how can we connect the dots here?

Jeanne Regnante:        

So, I think one of the main issues is clinical trial sponsors and the clinical trial operations folks in the sites working together to do a better job of reaching out to patients, ensuring that everybody is asked to participate, and not just selecting the ones who people think can participate but asking everybody to participate and understanding the eligibility of all patients and working together to help to cover their costs to keep them in chart.

Laura Levaas:             

Got it. Mark, I’m gonna pull you back into the conversation here for a minute. Can you touch briefly on what’s happening in the news right now around Medicare and Medicaid that could potentially impact clinical trials? Or maybe, Jeanne, you can speak better to that.

Jeanne Regnante:        

I’ll let Mark take that one.

Mark Fleury:

Certainly, so, Medicaid traditionally has been a program that has served primarily children in many states, children and pregnant women. Starting close to 10 years ago with the passing of the Affordable Care Act, states had the ability to expand Medicaid eligibility beyond those kids and pregnant women. And now we see many states who have expanded the roles of Medicaid recipients to healthy adults who just happen to be lower income.

And so, what that really has changed is the number of people obtaining their insurance through Medicaid. Obviously, there has been a lot of – it’s a state-by-state decision whether or not Medicaid is expanded. The Affordable Care Act as a whole is hanging in the balance in a court case, and there’s obviously been a lot of discussion about whether it should continue or not. So, certainly, the number of people who are supported through Medicaid is a dynamic number, and that certainly is subject to changing policies that are still under active discussion.

I will say that Medicare, again, the coverage for routine care costs in clinical trials for Medicare, long-standing policy since 2000 that has been relatively stable. And I would expect that to continue unchanged.

Laura Levaas:             

Thank you, Mark. And Jeanne, I’m gonna come back to you in a minute. For viewers that are watching, thank you for hanging in there with us. If you have any questions that you would like us to address in the program, we’ll get to that at the very end after we’ve talked with all of our esteemed panelists. Send your questions to questions@patientpower.info. So, now I would like to talk with Laura McHugh. Are you with us, Laura?

Laura McHugh:          

I am. Thank you so much for having me.

Laura Levaas:             

Hi. I am so excited to have you. I met Laura McHugh because she is a nurse navigator for a friend of mine who is ALK positive, which is the type of lung cancer that I have. And she works very closely with my friend and speaks so highly of Laura. So, I’m excited to have her on the program today. I wonder, Laura, if you could tell us why you think that clinical trials are important.

I wanted to share why they’re important to me personally. The medication that I’m on right now of course went through a clinical trial process, and it wasn’t even around before the year 2011. I was Stage 4 when I got discovered, which happens often with non-small cell lung cancer because many folks are asymptomatic. So, for me, what that means is if I didn’t have people going through the clinical trial process ahead of me, I probably wouldn’t be here today. So, on that level, is there anything that you can say why you think that clinical trials are important especially for people on Medicare or Medicaid?

Laura McHugh:          

Absolutely. I believe that the clinical trials pave the way. All of the genetic testing that’s done now, all of the testing that’s been done all the way down to a molecular level. So, with these clinical trials and all of the things that have been tested, it’s opened up doors beyond what we ever thought we would have for lung cancer. There are so many opportunities and lines of therapy that you never had before.

And across the board, I think clinical trials and participation in clinical trials, all of the people that have done that, just opened the doors for all of the people in the future. We had a lady who was in her 90s, and she met all of the requirements, participated in a clinical trial. And all the way through, she said, “I want to stay on this. I want to do this. It may not help me, but it will help everybody after me.” And that’s just profound.

Laura Levaas:             

Right. And so, Laura, tell the audience who you work with. I know that you specialize in thoracic cancers, and I know that clinical trials don’t always just focus on cancer. They deal with multitudes of diseases and conditions. But can you let us know who you work for because he’s famous in a way, right?

Laura McHugh:          

Absolutely. I’m actually the physician nurse for Dr. Raymond Osarogiagbon. He is well known in the field of lung cancer. That’s our specialty. We have a multidisciplinary meeting every week and a conference. He sits on the board for NCCN and multiple, multiple other things as far as paving the way for lung cancer. I’ve been actually privileged to be his nurse since he came in 2005. We’ve built our practice together, and, oh, the changes are just – the changes that I’ve seen in the years that we’ve done this are amazing. And he is brilliant; he is. He’s known all over the world. And our focus is lung cancer.

Laura Levaas:             

That’s great. Can you shed some light on the role of the patient navigator or the nurse navigator in what you do on a daily basis with your patients especially around clinical trials and folks who are on those government-supported insurances like me?

Laura McHugh:          

Sure. So, we base all of our care – we – or I’m blessed to have a research department and two really dedicated research coordinators that I work with very closely. They’re not nurses like myself, but they do all of the coordinating for the care on the studies and all of the above from patients that are uninsured that are on Medicaid, Medicare, even private insurance. And what we do, we see primarily all of our new patients insurance allowing through our thoracic program.

So, I actually have a coordinator with me when I’m in clinic. And so, if we even think a patient is potentially eligible – not even just for a drug study. There are smoking cessation studies that we have, different protocols for that. So, it really starts at the beginning. There’s the surgical studies, different things like that. And every Wednesday is that clinic. And even during the week, if there’s anything going on, they come to our regular clinics as well and do follow up with the patient.

Laura Levaas:             

So, I hear chatters here and there – when I bring up the subject of clinical trials, I hear things like, “Oh, trials are only for young people,” or, “Trials are only for old people,” or, “Trials are only for this type of person.” Can you speak to that a little bit?

Laura McHugh:          

Wow. Yeah. Well, part of it is if you look at where we sit, there’s always – until now, in recent years, you heard about research but you didn’t really hear about research. So your older population, they were skeptical. It’s a different generation of, “Are you experimenting on me?” And part of your underserved communities, a lot of people didn’t know anything about it. They’re limited on getting to a physician in general much less being able to participate or being in a center that even focuses on clinical trials.

So, I think all of that in the past was very, very real. I believe now people are coming around and seeing, “Wow, anybody can do this.” I think people are still limited. Some people don’t have computer access. It’s hard in a day of electronics, we sit down and we can pull up all of this information, but not everyone can do that.

Laura Levaas:             

Right. We do make a lot of assumptions when it comes to those type of factors. So, being that you’re a nurse navigator, I imagine that when you’re seeing a patient, you’re thinking, “Okay, is there a trial that this person might be good for?” I don’t want to say convince, but how do you help people learn about clinical trials and the importance of it because when you and I spoke yesterday, you said you want to make it clear to patients it’s always voluntary, “We’re not dragging anybody into a study. We want to make sure that you want to be there”?

Laura McHugh:          

Absolutely. So, again, all of our patients are approved during a thoracic conference, and then all of the ones that we can bring to our clinic within our healthcare system we bring through that clinic, and if not, we bring them to our general oncology clinic. The physician will sit down with the patient. Of course, we’ve met with the coordinators, they’ve looked at everything. And they’ll come to us and say, yeah, they like this or this. The physician sits down and talks with them, and then I go in the room and talk with them as well. We tell them, “This is totally voluntary, something that’s open to you if you’re interested,” talk about it.

The coordinators go in and speak with them as well. We tell them to go home, “If you have any questions or concerns, call back.” And a lot of times they will. You have to be able to digest something. It’s a very overwhelming visit to walk in an oncologist office and be told all of this information and try to sort it all out on the spot. So, a lot of times they’ll go home, they’ll think about it, they’ll call back. Basically, communication, I just feel that’s the most important – it’s communication.

Laura Levaas:             

Absolutely. So, to circle back a little bit, do you feel like it’s realistic for patients that are on Medicare and Medicaid to be in a clinical trial?

Laura McHugh:          

Absolutely. I think it’s clinically appropriate for anyone that fits. If everything lines up the way it should and they’re able to participate, I think it would be wonderful if everyone would.

Laura Levaas:             

This may seem like a silly question, but do folks on those programs get the same care as somebody that has a private insurance?

Laura McHugh:

Absolutely, absolutely from our standpoint. Of course, I’m answering from my institution and what I know that we do. And they do, absolutely. And sometimes there are challenges. I mean, we’ve had patients that were uninsured, underinsured. Again, Medicaid, you have to make sure – Medicare’s a little bit different again because all of the guidelines were set state to state. Medicaid’s different because each state has its own – and if you see someone in Mississippi, sometimes they can’t come across to Tennessee to go to the hospital or to do this. So, it’s a patient-by-patient basis, but overall, I think our patients are being treated, being offered clinical trials, and should participate if at all possible.

Laura Levaas:             

Wonderful. And again, just to underline that clear and open communication is important.

Laura McHugh:          

I think communication is No. 1 for everything. People are scared. They have questions. They don’t even know what to ask immediately. So, I think all of the support you can give – everybody has a knowledge base and everybody is empowered with that knowledge. Sometimes it’s all about just listening, communicating, and then answering any question they have no matter how simple it may be to us. To a patient, it’s a very profound thing. And it could be as simple as, “How am I going to get back and forth? Do you have a way to help me?”

Laura Levaas:             

Thank you, Laura.

Laura Levaas:             

Okay. I’m gonna circle back to the group and just ask some questions. I wanted to rewind with Mark and talk about Medicare Advantage. I am on Medicaid for about another year and I’m going to be rolled into Medicare, which under typical – I mean, I’m 44 years old, and so Medicare is typically for people that are 65 and older. And so, for me, it feels a little bit strange, and I’m like, “I just want to know how are they different.” And so, I have called my local CMS office, my local Social Security disability office. And I feel like I get different information. So, it’s sifting through everything. I just wanted to call out Medicare Advantage because you mentioned that. Can you expand on that and how it ties in with clinical trials?

Mark Fleury:              

Sure, sure, happy to. So, traditional Medicare has multiple parts. You have Medicare Part A, which is the hospitalization, and Medicare Part B, which is the physician portion, and then a Medicare Part D, which is the drug portion. A few years back (understand the complexities of all the pieces and parts of Medicare) there was a decision to allow private insurance companies to administer all the programs together on an optional basis.

So, if you qualify for Medicare, you can use the traditional what’s called fee-for-service Medicare or you can go through a private insurance company. So, this might be an Anthem or a Blue Cross or another private insurance company like that who has been authorized to bundle all of your Medicare benefits together under one program. Now because it is a privately run version of Medicare, they’re required to offer the minimum benefits, but they do have some flexibilities in how they administer that.

So, a traditional fee-for-service Medicare, as long as a physician advertises that they accept Medicare patients, you can go anywhere you want to. If you live in Florida and you go on vacation into Los Angeles and become ill and you want to go visit a physician there, as long as they accept Medicare patients, that’s fine. Medicare Advantage on the other hand looks a lot more like private insurance in that they sometimes build closed networks, so, you can only go to certain systems or only go to certain doctors. So, that’s an important difference between the two.

And in terms of with clinical trials, how that’s affected, if you want to enroll in a clinical trial and you’re Medicaid Advantage, right now the current policy is for the portion of your care that is related to the clinical trial, you would revert back Medicare fee-for-service, traditional Medicare. That doesn’t mean that you are kicked off of Medicare Advantage, but anything related to that clinical trial would be handled from a payment and a billing standpoint through traditional Medicare.

So, if you’re on a cancer clinical trial, all those cancer clinical trial bills would go through traditional Medicare. But say, for example, you needed to get your flu shot or had a cold or something like that, that would still be handled under your traditional – or under your Medicare Advantage. You wouldn’t be kicked off of it. It’s just the treatment part of your clinical trial would go through traditional Medicare. So, a little confusing, but that’s where we are from a policy standpoint today.

Laura Levaas:             

Okay. Jeanne, I wanted to ask you – and again, if you want to defer this to one of our other panelists, that’s A-okay. I’m thinking of folks who have some barriers around those additional costs in a clinical trial. Is it typical or acceptable for the, for example, pharmaceutical company or the sponsor of the clinical trial to pick up some of the costs that may not be covered under Medicare or Medicaid?

Jeanne Regnante:        

The answer to that question is yes, it is appropriate for them to do so. And actually, there is an FDA guidance document (it’s Guidance for Industry) that actually reinforces their ability to do so because there has been some concern that covering costs like logistical costs or hotels or travel or giving people a gas card would create undue influence. So, I think the FDA put out a draft guidance that’s clearing that up and basically reinforcing the fact that pharmaceutical companies are able to do that.

I can tell you from our working group, we have 10 active major pharmaceutical company members in the Diverse Cancer Communities Working Group. And I asked them what they usually do in this space, and during the planning phase of the clinical trial, they go out to their sites to ask for a budget and ask them what they need in terms of routine care costs and also logistical costs. And the site sends that information in. And generally, the pharmaceutical companies cover those costs.

What I’ve found to be the case, which is interesting to me, is that the clinical trial operations team in the sites have a lot to do, they have a lot of work to do. And this was brought up to me by a couple of leaders in pharmaceutical companies, that what they’ve learned is that they also need to ask what capabilities do you need, do you need people support or FTE support to be able to adjudicate and track those costs at a site level and validate them and close them out and pay them. And a lot of times, the answer is yes and pharmaceutical companies are paying for those FTEs at the site. So, those costs are being covered when the site asks for support.

Laura Levaas:             

Got it. So, since we’re talking about this topic anyway, that draft to FDA guidance publication, I’m gonna say it. It’s a really long title. It’s a mouthful. But I’m hoping you can break down a little bit of that. So, it’s called Enhancing the Diversity of Clinical Trial Populations, Eligibility Criteria, Enrollment Practices, and Trial Designs Guidance for Industry. What is the meaning –?

Jeanne Regnante:        

So, I do want to paraphrase what the FDA says, but I’m gonna read the portions that I think are appropriate for this discussion. So, there’s a section in this guidance that was put out in June, and it’s a draft guidance, so, it’s open for public comment. And it focuses on study design and conduct considerations for improving enrollment in the industry. There’s a big section. I really would urge everybody on the call to read this section because I think it’s really great and progressive and quite empathetic of a major governmental agency to put out this guidance to industry.

It gives examples. It notes the burden for trial participants in remote and rural locations, for example, and also acknowledges the trial burden on the elderly, children, disabled, and cognitively impaired individuals who require caregiver assistance. So, what the FDA does in this guidance is they encourage industry to reduce No. 1 the number of study visits where possible and use electronic communications or mobile technology to monitor the patient for safety and efficacy because of the challenges of a number of folks in this patient population.

They also encourage industry to make sure that patients are aware of financial reimbursements, and that’s what Laura does. She manages their expectations in the recruiting stage and reinforces the fact – and the guidance also reinforces the fact that the FDA does not consider reimbursement of travel, lodging, parking, time, and other considerations to raise issues concerning undue influence. And they also reinforce that the amount of dollars that might be reimbursed should always be addressed with the local IRB. So, I think this is a very progressive guidance to give the industry so there are no questions on what they can and cannot do.

Laura Levaas:             

Okay. Thank you very much. Laura McHugh, quick question, and Mark touched on this earlier in the program, what if something goes wrong in a clinical trial and a patient has to be hospitalized or treated for an unexpected reason? That’s covered, right?

Laura McHugh:          

It has been for our patients. If it’s Medicare, what you always look at is standard of care. And the Medicaid patients that we’ve had, when they’ve been hospitalized, to my recollection, we’ve not had anyone that we’ve had difficulty substantiating why it should be covered. I mean, sometimes you have to go the extra step and go back and forth with the insurance companies or Medicaid. But we so far have been able to get it covered.

Laura Levaas:             

I have a couple of questions that have come in from the audience, and feel free, Mark, Jeanne, or Laura. I’m assuming that a nurse navigator or a doctor is going to have the best information on where to find out about a clinical trial. But where are the best resources for someone to go? And again, I’m cancer focused because I have lung cancer and I work for Patient Power. And we support all types of folks with cancer. But there are folks that are in clinical trials that are not cancer related. Mark, what would be a source where somebody can find a clinical trial?

Mark Fleury:              

Sure. So, in looking at the current cancer clinical trial landscape, we know that the overwhelming majority, probably 75 to 80 percent of patients, who end up on a clinical trial found that clinical trial because someone on their care team recommended it or someone from the clinical trial team approached them. So, it’s most common that someone from the medical system invites that patient. But we also know that a lot of patients get their cancer care at very small practices (they might be single-doc practices or things like that) where clinical research is not a normal part of what they do. And in that case, you would not necessarily hear about clinical trials from your nurse or from your physician.

In those cases, it’s up to an empowered patient to find the clinical trial on their own. And that’s obviously a little bit harder but certainly not impossible. And there are public-facing websites. Some of them are sponsored by the government, things like ClinicalTrials.gov where all clinical trials whether cancer or not are listed in the United States. And NCI has one, trials.cancer.gov, which is just NCI sponsored, which is the National Cancer Institute. So, it’s federally funded clinical trials.

But additionally, many patient organizations both have general educational materials about clinical trials – so, for example, the American Cancer Society at the website cancer.org has information about clinical trials. At the moment, we don’t have a matching window, if you will, but many patient-advocacy organizations also actively help patients one on one with matching. So, many of these are disease specific. So, there are lung cancer groups who you can call at the hotline, colorectal cancer, etc. Many patient-advocacy organizations will do the direct handholding and navigation if your own provider does not do that for you.

Jeanne Regnante:        

I just want to add to that great list that Mark gave in terms of finding clinical trial sites. So, just a shout out to Stand Up To Cancer, they have a clinical trial matching site for any type of cancer. You can contact them, and they will actually match you to a clinical trial site in your area so you can give that information to your provider so they can call them to see if you qualify. Sometimes it’s difficult for anybody, myself included, to understand what clinical trial I might be eligible for just by looking at a site. So, it’s nice to have somebody do that for you.

Also, all the major pharmaceutical companies have if you happen to know about a given therapy or that you might be looking to be on because you heard about it it’s good to ask for help from somebody to find out what company makes it go to their website. And they all have clinical trial information on their sites as well.

Laura Levaas:

Thank you. And I’d like to share a little bit about my personal experience. When I was diagnosed, I was told about a Facebook group for my specific type of lung cancer mutation. And I learned about clinical trials from that group. And if I had never, like you said, Mark, been an empowered patient and been very curious in wanting the best care for myself, I probably would not have found out about those trials because some of them are just fly under the radar; they’re doing their work.

I think these are some great resources, and thank you for sharing those. One more question that I would like to ask the group before we – we have a couple of questions that came in from the audience, which is awesome. What is one solution (Mark, we’ll start with you) that you would like to put forth to address the issue of better clinical trial participation for Medicare and Medicaid patients which really, I mean, goes out to the larger group, I mean, really for anyone?

Mark Fleury:              

Yeah. Well, I think specifically within the population of Medicare and Medicaid, as I mentioned at the outset, Medicare has a uniform national policy. So, someone like Laura, if she became a clinical trial professional in a different state, the Medicare policy would be the same it doesn’t matter what state you’re in. Whereas Medicaid, it varies so much, and that can be quite a bit of hurdle.

As I mentioned, I work in the policy and advocacy portion of ACS, and so, we focus on legislation. And so, one of the public policies that we have been advocating for (and there’s actually a piece of legislation before Congress right now), it would harmonize all 50 states plus DC Medicaid policies such that standard of routine care costs in cancer clinical trials would be covered in all 50 states in the same way and there wouldn’t be this ambiguity or uncertainty from state to state in terms of how it’s covered. So, that would be my one wish within this question if I could wave my magic wand.

Laura Levaas:             

Yeah. That would very much clarify everything. Ms. McHugh, do you have a solution? What would you like to see happen to get more folks participating in clinical trials specifically those on the Medicares and Medicaids?

Laura McHugh:          

Again, from my nursing background, a lot of it’s communication. And I think it’s sitting down with patients and explaining what some of the benefits are, what the risks are but what the benefits are because truly the benefits outweigh the risks. People worry about money and they worry about all of these things. Well, if it’s Medicare, it’s standard of care. Anything above and beyond, if there’s a problem, then you appeal back to the drug company, the provider.

Opening doors, communicating with patients, telling them, “You have a more active role in your own healthcare when you’re on a clinical trial. You’re empowered. You’re educated. You’re the first to benefit from this drug. You have your health professionals close. You’ve got a research coordinator, your nurse, your doctor, access to new drugs that may not be available.” I just feel like communication and – we’re totally sitting down with someone and explaining and taking some of the fear away from what people think about being on a clinical trial.

Laura Levaas:             

I have a friend in the lung cancer community that was in a clinical trial. I don’t remember the specific drug, but she is still on it after it came out of trials. And she’s been on it for years, which is amazingly successful. And if not for that trial, she wouldn’t be where she is. And so, that’s just amazing. Okay. And then, Jeanne?

Jeanne Regnante:        

You know what, first of all, I agree with what Mark said and what Laura said. First of all, it needs to be legislated. And No. 2, there needs to be better communication amongst trusted providers, trusted community leaders, primary care physicians to talk to patients to have them understand that a lot of these trials now include placebo versus standard of care and also help them to manage their expectations in terms of what will be covered in terms of their cost. And the folks that need to do that are the closest to the healthcare systems and patient navigators and care coordinators who can talk to an individual specific situation.

I think in addition to all those things, I think that generally industry needs to do a better job of placing trials where the patients are. Although that seems quite trite, patients that are in underserved communities or in rural communities, they don’t often have access to these cancer centers which are big academic centers that do a lot of these trials with big innovations.

And I think that we need to get much more creative to make sure that either the reach out from those academic centers go out to community centers or we do a better job placing clinical trials in community research centers to ensure better accessibility because really, logistical support, even if you cover it, even if the industry covers it or cancer care covers it or the American Cancer Society cover it or a laser X organization covers it, it’s still a challenge and a barrier.

So, I think we need to do a better job overall. The infrastructure needs to place trails where the patients are because cancers are not homogeneous across the United States. They appear in different places with higher risk and higher prevalence. And we need to use that data to place trials where the patients are.

Laura Levaas:             

I agree. I’m actually located in Denver, Colorado, and I was doing some research for a blog post recently. And I went to American Cancer Society, Mark, just to look for what are the most recent statistics by state in terms of cancer. And obviously, it’s not lung cancer specific. But I was shocked to find out that Colorado has one of the highest percentages in the country of cancer occurrence. And I was surprised. So, Laura, would it be appropriate – this article that you sent me this morning from ASCO, would this be appropriate to include in our downloadable guide for our guests after the program? This was about the Affordable Care Act because we were talking about how people can get involved if they’re interested. What do you think, should we include this, Jeanne?

Jeanne Regnante:        

Oh, I heard you say Laura.

Laura Levaas:             

Yeah. Sorry.

Jeanne Regnante:        

I think it’s a really well thought out piece to help folks understand how they can get involved with their legislators and understand that this act and this piece of legislation to advocate [inaudible] [00:50:28] specifically for patients that are on Medicaid in the United States so they can get the same benefit of routine care that Medicare patients get.

Laura Levaas:             

I do have a question from Steve, one of our audience members, and he says, “Can Medigap Plan F help with paying for clinical trials? If the clinical trial accepts Medicare, would my out-of-pocket expenses be covered? I’m worried that any extra testing would be my responsibility.”

Mark Fleury:              

Yeah. I’m happy to jump in with a quick answer on that.

Laura Levaas:             

Okay. Thanks Mark.

Mark Fleury:              

So, I mentioned a little bit before about what’s required to be covered. When you think about costs involved in a clinical trial, I’ll put them in three buckets. There is the normal routine medical care that you would get. So, for example, if you would normally get surgery and then followed up by some sort of chemotherapy, everybody’s gonna get the surgery regardless. And then say, for example, ordinarily routine care would be you would get a scan every six months after surgery, but the clinical trial because they want to collect more data wants to have a scan every three months instead of every six months. And the clinical trial is testing a new drug after surgery.

So, Medicare would pay for the routine costs, which would be the surgery and then a scan every six months. The clinical trial sponsor would pay for the drug, which is what you’re testing in the clinical trial. So, the patient doesn’t have any responsibilities for that. And since there’s basically twice the frequency of scans, the sponsor would pay for every other scan.

Now what’s important is that while Medicare covers the routine care costs, it covers them the same way it would cover any other cost. So, if you have a co-pay for a doctor’s visit that is routine, just because you’re on a clinical trial, that co-pay doesn’t disappear. So, if you have a Medigap plan that covers those co-pays, it should cover them the same way as if you were not on a clinical trial because the only responsibility for the patient is the co-pays of the routine care costs, and Medicare will pick those up.

So, anything that’s not normal from a medical standpoint will be paid for by the sponsor. Now as Jeanne aptly pointed out, if you’re coming in twice as often for tests, even if the test itself is paid for, you might be paying for the parking garage twice as often or gas to travel twice as often. And those are nonmedical costs that can add up, but they’re not really involved with insurance, but you can sometimes get money from the sponsor or other third-party support organizations like ACS.

Laura Levaas:             

We have one more. Annie B, “I’m on Medicare. Where do I find a clinical trial in my town?”

Mark Fleury:

Typically, most of the ways that you find clinical trials, again, you can work directly with where you’re seeking care. So, if you have an oncologist, you can ask them about clinical trials. And if they conduct them, they will screen you for the trials that they have open at their site. If they don’t conduct clinical research, then you would either go to one of these public websites like a ClinicalTrials.gov, you could call an advocacy organization. There are several in the lung cancer space, and we can provide a number of different links to different matching engines or third-party organizations that could help match you. But clinical trials typically are not restricted based on insurance types. So, you would use the same search engines as anyone else would.

Laura Levaas:             

Okay. All right. Well, I want to say thank you so much to our esteemed guests for joining us today. We learned so much today about clinical trials, Medicare and Medicaid, the different options. So many takeaways here. We will have a downloadable guide available as well as a replay of the program in case you’d like to dig in a little bit deeper.

Really, I think my takeaway from the whole program is that there are options out there. Clinical trials can be a great solution for your medical care of your disease. I personally am all for it. I know it’s a very personal decision, whether you want to participate or not. But I decided early on that I would definitely enter a clinical trial because I’m willing to sacrifice myself for future generations because there are people that came before me that did the same and I would not be here today if not for that. So, thank you again for joining us Mark, Jeanne, Laura. We very much appreciate you.


We thank AbbVie, Celgene Corporation, Daiichi Sankyo, and Novartis for their support. 

What Is the Value of Diversity in Clinical Trials?

Clinical Trial Mythbusters

Clinical Trial MythBusters: What Is the Value of Diversity in Clinical Trials? from Patient Empowerment Network on Vimeo.

Downloadable Program Guide

In this MythBusters program, Senior Vice President and Chief Medical Officer (CMO) of ASCO, Dr. Robert Schilsky, and 20+ year CML survivor, Mel Mann along with Cecelia Mann, will unpack some of the issues that have led to the lack of diversity in clinical trials and initiatives in place that are changing all of this.


Transcript:

Andrew Schorr:
And greetings from near San Diego, Carlsbad, California. I’m Andrew Schorr from Patient Power. Welcome to this Patient Empowerment Network program, the next in our series Clinical Trial Mythbusters, and this program is so important, discussing what is the value of diversity in clinical trials. And, believe me, you’ll hear it is so critical. We have to do better, and we’re going to discuss that over the next hour.

I want to thank the companies that have provided educational support through grants to the Patient Empowerment Network. They have no editorial control, but their support is welcome. And that is AbbVie Incorporated, Astellas, Celgene and Novartis. All right.

We’ve got a lot to discuss today, and we welcome your questions along the way. I want to first introduce someone who, like me, has greatly benefited from a clinical trial and believes that they are alive today because of their participation. And so joining us from Atlanta is Mel Mann along with his wife and care partner Cecelia Mann. Mel, welcome to the Patient Empowerment Network program.

Mel Mann:
Thank you very much.

Andrew Schorr:
And we’re going to hear more of Mel’s story in just a minute. I want to introduce a very prominent medical expert who joins us. He is the senior vice president and chief medical officer at really the largest cancer organization, the American Society of Clinical Oncology, and that is Dr. Richard Schilsky. Dr. Schilsky, welcome to our program.

Dr. Schilsky:
Thank you, Andrew. Happy to join you.

Andrew Schorr:
Okay. And are you in the Washington, DC, Virginia area?

Dr. Schilsky:
That’s where our organization is based, in Alexandria, Virginia, yes.

Andrew Schorr:
Thank you for being with us. I should mention that Dr. Schilsky has had a lot of experience related to trials. He was the head of hematology/oncology at the University of Chicago, which of course Chicago is a very diverse city, and the University of Chicago does a lot of research. And he also helped run a big research group that doctors from around the world are part of, and he did that for many years. So we’re going hear more from Dr. Schilsky in just a minute. But, first, Mel. So Mel, in the late ’90s you were dying, right, of chronic myeloid leukemia, correct?

Mel Mann:
Yes, I was.

Andrew Schorr:
Losing weight and being told that there wasn’t much to do, right?

Mel Mann:
Correct, yes.

Andrew Schorr:
Maybe a transplant. But you were in Atlanta and you went from doctor to doctor, but somehow you got to MD Anderson, a major research center in Houston. What did they offer you there?

Mel Mann:
When I first went out to MD Anderson they said they were going to put me on a clinical trial after clinical trial. And the first thing they did was increase my dose of interferon, and that was the medication, the standard therapy at the time, and then they tried different combinations of drugs, and eventually I started on different clinical trials.

Andrew Schorr:
Okay. And, Cecelia, you were there in Atlanta and he was scooting over to Houston, it’s not exactly around the corner. Why were you supportive of that?

Cecelia Mann:
I was supportive of that because that was the last chance that he had to a cure and for survival. So from the very beginning, whatever type of treatment he needed when he was flying around, whether he was going looking for bone marrow transplants, doing bone marrow drives, and therefore I was supportive of. We had a five‑year‑old daughter at the time, and so anything that Mel needed I was there to support him.

Andrew Schorr:
So, Mel, this is a happy story because here we are in 2018 as we do this program and you are with us when many people with CML at the time were not with us that long. Hopefully, a transplant could be curative, but a lot of people passed away. You were lucky enough to come back as they were rolling through different trials and there was a new one that opened up for a drug called Gleevec, a pill.

Mel Mann:
Yes.

Andrew Schorr:
What happened?

Mel Mann:
Okay. So in the summer of ’98 the Phase 1 Gleevec study opened up, and I went out to MD Anderson, and I was patient number two, and I started taking it at a low dose, and it was effective for me. And eventually they increased the dose and it started changing my leukemia around to eventually I reached what they call a major molecular response. And that was 20 years ago. This summer I went over 20 years.

Andrew Schorr:
Wow. Well, Dr. Schilsky, is that an example of a patient getting, if you will, tomorrow’s medicine today, what we hope for?

Dr. Schilsky:
Absolutely. And, first of all, it’s such a wonderful story, Mel. It’s great to hear you tell it, and it’s exactly why we do research, exactly why we do clinical trials, to try to discover the new therapies that people need that will give them the kind of long‑term survival and quality of life that you’ve been experiencing. It’s just‑‑it’s wonderful.

Andrew Schorr:
So, Dr. Schilsky, let’s get into the problem. So, generally, there are many clinical trials that take longer than one would hope to fill, and the FDA and I know scientists have been seeing well, gee, how do we know what we’re testing applies to people maybe with different ethnic, gender backgrounds, a variety of situations, and often we can’t find people who fit those categories to be in. What is that‑‑how poorly have we been doing in the past with diversity in trials, and what does that mean for developing new medicines?

Dr. Schilsky:
Well, we don’t do well in almost any dimension. We don’t get enough underrepresented minorities in clinical trials. We don’t get enough older people in clinical trials. You have to remember that 60 percent of cancers occur in people 65 years and older, and yet only about 10 percent of people participating in clinical trials are 65 and older. So we are having to treat the majority of older people, and I would say the majority of minority people, with data derived from participants in clinical trials who are not like them.

We need to change that for a whole host of reasons. It’s historically been very challenging, and the problems really sort of boil down into three big areas that I think we can discuss a little bit further.

First is awareness. Many people are not aware that clinical trials are even an option for them. Many people think that a clinical trial is a last resort, and I want to dispel that myth right out of the box. Clinical trials can be a very good option for patients right from the time of their cancer diagnosis even if it’s their very first treatment. So clinical trials may be a last resort, but they don’t have to be, and there are many clinical trials that are appropriate for people right following the initial diagnosis of their cancer.

So there’s the awareness issue, and sometimes, frankly, not even the doctors are aware of what clinical trial options are for their patients. And the one thing we know for sure is that the most influential person as to whether or not a patient goes into a clinical trial is their doctor. If the doctor does not recommend it, if the doctor is not aware of it, it’s not going to happen.

But then you get into the more technical issues. There are things, there are rules for clinical trials because they are research studies. They are experiments. There are very well defined rules, most of which are in place to protect the people who are participating in the study. Some of these rules are called eligibility criteria, and they specify the characteristics of people who can enroll in the study. Well, historically, they tend to be very rigid and very limiting, and you’ll often hear people talk about how the only people who can get into clinical trials are Olympic athletes. That may be the case, but it’s not Olympic athletes that we’re treating in the clinic every day, so we need to make our clinical trials more representative so that they’re more applicable to the typical person that a doctor sees in their office.

And then there are the logistical or operational issues of the clinical trial. The clinical trial can be very burdensome. Mel just described how he had to travel from his home in Atlanta to Houston to participate in a clinical trial. Not everybody can afford to do that. Not everybody can take time away from work, time away from home. And the clinical trial requires not only that you travel sometimes but that you travel on a specific schedule because of the requirements of the trial.

So all of these are issues that are‑‑can limit participation in trials, and many of them are magnified in minority populations or in populations that don’t have the economic resources to be able to meet the requirements of the trial.

Andrew Schorr:
Right. Let’s talk about that for a minute. So, Cecelia, you go out in the community and speak to people, and you probably meet some people who maybe are diagnosed with a blood cancer, like you’re active with the Leukemia and Lymphoma Society so you may speak to them, and they say even if you brought up about a trial, they say, hey, Cecelia, I’m working two jobs. Or my husband or spouse is working two jobs, and we’ve got two kids, three kids. How could we ever participate? We just can’t get away or we don’t have the family support or whatever. Those are real issues, right?

Cecelia Mann:
True. True, those are real issues, and I try to direct them towards resources that Mel and I found out about along the way. The Leukemia and Lymphoma Society, they have resources to help with travel, and American Cancer Society has resources that help with the hotel and lodging. And there are a number of other different types of funds that can be assessed to make that a possibility.

But you’re right, Andrew. I had one young lady at a health fair and a second cancer had popped up, and she was coming there to get information, and she was saying that they were saying it wasn’t too much they could do, and I brought up MD Anderson. And she immediately said, I can’t go out there, I have to go to work, and she turned around rough. And so when they listen to me then always glad to tell them about it and let them know there is an option and that clinical trials work, and I point to Mel, my husband, as a success story also.

Andrew Schorr:
So, Dr. Schilsky, you talked about physician awareness. It’s also about more physicians participating in trials at I think what you call the community level. So, in other words, MD Anderson and where you work, at the University of Chicago, those are big city centers and where they are in Atlanta there’s Emory and some other mainly centers, but what about out in the hinterland, if you will? Can somebody who lives there diagnosed with a cancer, how do they have access to a trial that their doctor knows about and maybe that’s more close to home, if you will?

Dr. Schilsky:
Right. So of course we know that anybody with cancer prefers to be treated in their community, and most are. So one of the goals is to be sure that oncologists practicing in all sorts of community settings have access to clinical trials. Now, one of the ways that happens is that for more than 50 years now the National Cancer Institute has actually been operating and funding a community‑based clinical trials network. It used to be called the CCOP program. That’s an acronym that we don’t have to go into. They’ve recently changed the name. It’s now called NCORP program, but‑‑that stands for, I think, the National Community Oncology Research Program.

But the point is that the program, which is in most but not all of the states in the United States, funds community oncologists to participate in NCI‑sponsored clinical trials, and there are at least 65 or 70 such clinical facilities around the country right now. So in those medical practices patients can find those clinical trials in their communities without having to travel.

There are also other community‑based networks that are active participants in cancer clinical trials. So I think at the end of the day the critical thing for patients, and this is sometimes easily forgotten because you’re so, you know, your thinking and your time and your emotion are all taken up in dealing with the cancer diagnosis. It’s really important, though, to ask the doctor, do they have access to clinical trials. Do they have a clinical trial that might be appropriate for you? And if not you might want to consider where else you could go, hopefully still relatively nearby to get access to a clinical trial.

Andrew Schorr:
Mel, so for you, you went around to some doctors who were not aware of anything new to do for you, right? And that’s still true in so many areas of cancer. Now, what do you say to patients about speaking up because Dr. Schilsky just referred to it, people are terrified. They really just want the doctor to have the answer. What do you tell people so that they maybe advocate for themselves?

Mel Mann:
Okay. So if you’re looking for a clinical trial and I’m out at, like you say, a health fair, we have a‑‑the Leukemia and Lymphoma Society has something called the clinical trial support center, and they have nurses who work early in the morning till late at night. And you call them up and you tell them about your illness, and they check the availability for what clinical trials are out there, and then they narrow it down to what you actually qualify for, and then they take into consideration your finances and other issues.

And then you’re left with a number of possible clinical trials that you have, and you can take that back to the doctor and you can discuss that with him. So that’s one of the things I talk with them about.

Andrew Schorr:
I could mention, now this is really more broadly across cancer, too. So there are breast cancer groups, there are lung cancer groups, and I would just make a pitch to find out, is there a local chapter or national number for you, for the cancer you or a loved one been diagnosed with, and that’s the question.

Mel Mann:
Yes.

Andrew Schorr:
Say, look, A, I don’t want to feel I’m alone, and, B, how do I get‑‑how do I get connected with what could be lifesaving or life‑extending treatments for me and that I could discuss with my doctor. And understanding‑‑and then, boy, if there are obstacles like financial issues, logistical issues, travel issue, is there support for that.

Mel Mann:
Yes.

Andrew Schorr:
So let’s go back to the inclusion/exclusion or eligibility issue you spoke about, Dr. Schilsky, because, you know, somebody who has cancer may also have heart problems or diabetes or some other issue. Maybe they previously had another cancer, and so for the companies developing new drugs they may be happy with narrow inclusion criteria because they don’t want to have anything get in the way, some previous thing you’ve had, to affect their ability to have a new drug go on the market.

So what kind of work is going on between government and the drug manufacturers so that the criteria, not so tight, but you can still get legitimate scientific answers?

Dr. Schilsky:
Right. So, as you alluded to, Andrew, there are good reasons that there are eligibility criteria. One of them is to protect the patients in the study from circumstances that would increase their risk of participating in the study. Another is because the companies or whoever is sponsoring the study wants to be able to isolate the specific effect of what they’re studying without having a lot of confounding factors that could muddy the water and makes it difficult to interpret the results. But that said, the bad thing about all that is that the results of the study might not be applicable to the majority of people who could benefit from the treatment because they weren’t included in the study to begin with.

So one of the things that my organizations has been working on very hard over the last couple of years now, and we’ve been doing this collaboratively with people from the Food and Drug Administration and the National Cancer Institute and a lot of clinical experts and a patient advocacy group, Friends of Cancer Research, is to try to expand or broaden or simplify some of these eligibility criteria that tend to keep people off of trials and in particular tend to keep minority populations off of clinical trials.

So, for example, it’s not unusual for someone who has a new diagnosis of cancer to have previously had some kind of cancer earlier in their lives. So we might see a patient who has lung cancer who 15 years ago had a diagnosis of prostate cancer. Well, for that lung cancer patient to go on a trial that has the typical inclusion and exclusion criteria that doesn’t allow for this previous malignancy, they would be excluded even if they had been cured of that prostate cancer 15 years ago.

We also see, and you mention what we call in the medical profession comorbidity. So if someone’s got cancer and they also have heart disease, they also have diabetes, high blood pressure, anything that affects the functioning of your normal organs, can also exclude people from participating in trials, and there are certain limits that we feel can be expanded and still allow the treatment to be given safely.

So just about a year ago now we came out with a set of recommendations for how eligibility criteria can be modified to make clinical trials more inclusive. And now just recently, I’m really pleased to say, the National Cancer Institute expanded their sort of template protocol document that many investigators follow to incorporate our recommendations, so now their standard protocol includes these broader inclusion and exclusion criteria. And the FDA now is working on what they call guidance documents to advise commercial companies that are running clinical trials to do just the same thing. So we are very optimistic now that we’ve got this ball rolling. We’re going to be removing these obstacles, and we’re going to be able to have more inclusive and diverse population of patients who participate in cancer clinical trials.

Andrew Schorr:
Great leadership. I hope it works great, and we’ll be happy to support you. So, Mel and Cecelia, let’s talk about the money part of it a little bit. So you were making trips to Houston, Mel, Cecelia was home with a five‑year‑old, and so admittedly there may be hardships, financial hardships, being away from family if you have to go to a trial somewhere else, checking back. What do you say to people when they say, well, I’m just going to go with the traditional stuff. It’s close to home. In other words, if there can be programs that can help them it still takes courage, if you will. So what would you say to people about investing in their life, if you will?

Mel Mann:
Well, you know what the standard, what the current treatment is and the outcome of that, so if you want to have a different outcome then you have to try something new which is probably going to be a new drug. So you have to weigh that with the cost and the travel. Some people may not have the support, the caregiver support to go a long distance, so you have to take that into consideration. As far as the Leukemia and Lymphoma Society they do have certain funds where they can help with travel pay, co‑pay, insurance premiums, that could help alleviate some of it. So there’s a lot of stuff that’s involved, and it’s an individual decision.

Andrew Schorr:
So we’ll just make a comment, though. So, many people have a church or synagogue, friends, neighbors even if they’re living alone, but yet people are sometimes hesitant to ask for help. And I would say speak up. People do want to help you.

Dr. Schilsky, let’s talk about another reality of trials. There’s a history certainly and some fear still in the black community of whether they were tested on, without their knowledge even, going back years and the general thought, you’ve heard it through your career, I’m sure, people say, well, I don’t want to be a guinea pig for a couple reasons. One is we don’t know if it’s going to work. And second of all if there are different arms of a trial I don’t know if I’m going‑‑I’m going to go to all this trouble and expense, I don’t know if I’m going to get the good stuff. So maybe you could speak about that a little bit. First of all, the fears of being experimented on, and then also about whether you will get what could be a breakthrough.

Dr. Schilsky:
Yeah. Well, for sure, you know, there is this sort of sordid history of inappropriate experimentation on people, and clinical trials are a form of experimentation. They are a form of research. There’s no doubt about that. But clinical trials these days are highly regulated, overseen by independent groups that include patients and clinical experts that come together in committees called IRBs, Institutional Review Boards, and they evaluate on both the risks and the benefits to patients who participate in clinical trials.

They make sure that the trial has an appropriate consent process associated with it, that it’s explained in plain language to patients, so I think these days a lot of those concerns no longer exist. And I hope that people can get beyond the history that led to some of those concerns. The‑‑sorry, I lost a train of thought on the rest of your question.

Andrew Schorr:
The issue about are you going to get the good stuff.

Dr. Schilsky:
Oh, yes. So a couple points there. One point I want to make clearly is that in most cases cancer clinical trials do not include a placebo or an inactive treatment. That’s not always the case, but it’s true most of the time. So patients are always going to get at least the standard of care treatment, and of course the standard of care is what is at that time known to be the best available treatment.

The whole point of doing the research is to determine if the new thing is better, and of course we always hope it will be. It’s not always better, but sometimes it is, as in Mel’s experience. And I think this has to be clearly laid out to patients. They have to clearly understand why the research is being done. In many trials nowadays even if the patient is assigned to get the standard of care treatment there still may be an option to get the new treatment at a later point. So if the standard of care doesn’t work many times there’s still the opportunity to get the new treatment following the standard of care treatment.

So the trial really boils down to not standard versus new but new versus standard followed by new. So eventually everybody may have a chance to get the new treatment. That’s not always the case, but I think the key‑‑my key take‑home, in a sense, is that we’re doing the research because we think and we hope the new treatment is better, but we have to do the research to prove that. And everybody in a clinical trial I think can be assured that they’re going to get, at the very least, the best available standard treatment.

Andrew Schorr:
Mel, when you signed the papers to be in a trial, and you probably shared them with Cecelia, especially back in the late ’90s and I participated in one trial in 2000 and another in 2011, there’s a lot of paperwork, things in bold face written by lawyers. I didn’t always understand it. What propelled you beyond that? Was it just that, oh, my god, if I don’t get something I’m going to die? Or how did you two deal with the paperwork and feel comfortable signing on the dotted line?

Mel Mann:
Well, I saw a lot of hope in the paperwork. For example, one trial I was on was peginterferon, and I had been taking interferon every day, injecting myself, and I had to keep it refrigerated and when I travelled it made it difficult. So with peg I can take one shot a week, so that would make the cancer journey easier. It may not make me live longer, but it will improve my quality of life, so I saw my quality of life improving with that clinical trial. And I looked at the paperwork, and I went through it, and I felt comfortable with it.

Andrew Schorr:
And how about you, Cecelia? I mean, your husband says, well, I’m going to be in a trial and I’ve got to sign all these papers. Did you say at any point, wait a minute, that’s scary?

Cecelia Mann:
Well, no, I didn’t. I didn’t because with Mel, he had three years to find a marrow match, and he was at the end of year two and no match in sight. And so when he had the opportunity to go out to MD Anderson and be on a clinical trial or several, I was okay with that. I was okay with that. And I looked at it as actually being a blessing. And it turned out to be, and we’re grateful.

But I would say to anyone else who is contemplating a trial and that person and their caregiver, their spouse, to just educate yourself, and get as much information as you can, ask as many questions as you can, but please don’t just throw it away out of hand. It’s definitely worth considering.

Andrew Schorr:
Dr. Schilsky, so we have more than 50 million people with a Hispanic background in the United States, and even if many people are speaking English they may speak Spanish at home. And then when you are diagnosed with a cancer there’s a whole new language of stuff that comes into play that even if you’re fluent in English it may not be either what you easily understand or even aligns‑‑what’s being asked of you aligns with your cultural background. Okay? So how, beyond, let’s say, the African‑American community, when you look at the Hispanic community, how do we encourage participation there and get over some of these cultural or language nuances, if you will?

Dr. Schilsky:
Yeah. So it’s much the same thing in the sense that the same information has to be conveyed but it may have different meaning and different interpretations in different ethnic and cultural groups. Most clinical trials now will have a consent form that is fully translated into Spanish. But, of course, there are many different languages on the globe. When I was practicing at the University of Chicago for many years on the south side of Chicago, we had Polish‑speaking people, we had Russian‑speaking people, we had people‑‑Chinese‑speaking people.

So the requirements actually are that there must be a consent form, at least some reversion of which is translated into the first language of the patient. So if you’re a native Spanish speaker, a native Chinese speaker, you have to have, be able to see a consent form written in that language, and generally speaking you have to have your native language interpreter present in the room to help you go through the consent form and respond to your questions. And that person has to be someone who is independent from the research team so they can give you the straight answer and not be influenced by any member of the research team. So I think all of that certainly helps.

But, again, what helps a lot more is to have members of the care team who look like the patient. So we have problems with diversity in our profession as well. We have very few African‑American oncologists. We have more Spanish‑speaking oncologists, but again we have few Asian oncologists. So we need to do a better job of improving the diversity of our profession, improving the diversity of the care teams. We need nursing staff and research staff and other people who work with our patients who represent them and gain their trust, who look like them, who talk their language. And I think that will go a long way toward making people feel more comfortable about participating in clinical trials.

Andrew Schorr:
I was at a conference last week and I heard some of the patient experience, people from different drug companies talking about how they were trying to simplify their forms because I know in 2000 when I entered a Phase 2 trial there were all kinds of black boxes, you could die, you could this, everything in the kitchen sink was in it. I’m still here, and I think because of the trial, and most of the side effects I didn’t have or they were definitely handled extremely well.

So right now, where are we, Dr. Schilsky, with participation? And why is it important? In other words, in this age of personalized medicine why do we need more black people in certain trials? Like, I know in multiple myeloma, one of the areas we cover, there’s a higher incidence in the black population, right, but yet few black people are in the trials for myeloma drugs. Or maybe there are differences with Asian populations or other populations. So is it that you can’t really get a clear scientific answer on the differences? Is that it?

Dr. Schilsky:
That’s part of it. First of all, we want anybody who could potentially benefit from being in a trial to be able to be in the trial for their own personal benefit. Secondly, we need to learn about the performance of the drug or the intervention in all the diverse populations in which it might be used. And one of the things we have learned is that not all populations respond the same way. Some treatments are more toxic in certain racial or ethnic groups. Some are more effective in some racial or ethnic groups.

And, you know, since you brought up this whole new world of precision medicine, I’ll give you the example of the lung cancer drugs that are used to treat the specific mutations in a gene called EGFR. So that’s a gene which has mutated in about 15 percent of Caucasian patients with lung cancer, but it’s mutated much more commonly in Asian patients. And in fact one of the clues that there was even a gene mutation that was important in determining whether these drugs worked or not was because it was observed that the drugs worked better in the Asian patients in the clinical trials even before the genetic abnormality had been discovered. And the clue was what’s different about the Asian patients than the other patients in the trial.

So the diversity is critical to our learning and critical to our application of the therapy in all the diverse populations that we serve.

Andrew Schorr:
If you’re in our viewing community and you have a question, send your questions into questions@patientpower.info, questions@patientpower.info. We’ll continue our discussion of course, but we invite you to join in.

So, Mel, when you get to talk to people, what do you say? Somebody is sick, diagnosed with a cancer, what do you say? Dr. Schilsky was talking about not seeing clinical trials as a last resort, and you weren’t seeing it that way (?) Inaudible, but today what would you say to people when you talk to them about it?

Mel Mann:
Well, I will say explore your possibilities because there are all different opportunities at each phase. You may not go into Phase 1 but you could do a Phase 2, 3, 4‑‑or Phase 3, and you don’t know what’s going to happen in each of those phases. So you just have to see what’s out there. And I’m exhibit A, so they look at me and they say, well, I can work, and then not as suspicious, you know. We have Tuskegee, and that was 1972, and it was that dark period of cancer history so that kind of rolls around in their mind, but you can’t let that jeopardize the opportunity such as Gleevec that I took advantage of. So we know that Gleevec worked, and there are other drugs that have improved the quality of life and the lifespan of cancer patients. So definitely research those drugs.

Andrew Schorr:
Did you lose heart when you were first in one trial and the medicine wasn’t working for very long? Some would say, well, all right, I tried a trial, forget about it, you know. But you then pursued other trials. What propelled you to do that?

Mel Mann:
Well, I was still in the game, so I saw that these trials took‑‑well, first of all, I could not find a bone marrow donor, but a bone marrow transplant was pretty drastic in itself so I was looking at these other opportunities as maybe not even having to take part in‑‑have a bone marrow transplant. So that was another incentive. So‑‑and I knew that if I didn’t find one‑‑there was a very small chance, there was only about 5 percent of Americas who are on the marrow registry, so basically I was helping to build a list, maybe not for myself but for people in the future who needed a transplant.

Andrew Schorr:
Dr. Schilsky, let’s talk about the pace of research. So, first of all, if we don’t get enough participation in trials how does that slow drug development?

Dr. Schilsky:
Well, it slows it down enormously because we have to have a certain number of people in each trial to be able to get a reliable answer. And these days it’s becoming even more challenging because as we’re developing drugs that only target a specific genetic abnormality in the tumor which sometimes is very rare so we may be looking for a genetic abnormality that only occurs in 2 or 3 percent of all people with a certain kind of cancer. First you have to find the people who have that genetic abnormalities, then you have to be able to enroll them in a clinical trial. They have to be willing. They have to meet the enrollment criteria. So it can take a long time, and even a global effort to find enough people to fill out a clinical trial.

And most clinical trials in order to produce a reliable result are going to require a minimum of 50 to 100 patients. Some require many hundreds of patients or even many thousands of patients depending on the question being asked. So you can see if people are not participating it’s going to take long time to get those answers.

Andrew Schorr:
Now, Mel, you got Gleevec in a trial at least three years before it was approved, and it was approved fast because it was such a breakthrough, right? So you literally got tomorrow’s medicine today, and it saved your life in the process, right?

Mel Mann:
Yes, because I was past the three years. I was about three years and eight months in my diagnosis, so you add another three years onto that and I would not have been here.

Andrew Schorr:
Right. In my case I was in a trial related to chronic lymphocytic leukemia, three‑drug combination, and I received that in a Phase 2 trial 10 years before that was approved. So it was a long time.

So I have a question for you about personalized medicine, Dr. Schilsky. So for instance in chronic lymphocytic leukemia I’m not‑‑I’m Caucasian but I’m Ashkenazic Jewish, okay? So where we’re going with personalized medicine, are we beginning to find subsets among Caucasians, among African‑Americans, among Asians, where there are even more narrow slices to help us understand targeting of medicines and what’s effective for whom? Is that where we’re headed?

Dr. Schilsky:
Absolutely. And, as I said earlier, we’re seeing that all the time. So almost every common cancer now is being broken down into a whole basketful of rare cancers under the broad umbrella of whatever the cancer type. So lung cancer, there’s probably six or eight different kinds of lung cancer now that each have a specific genetic abnormality, that each requires a specific treatment. And many of those treatments now are FDA approved, but the first thing you have to know is does the cancer have the genetic abnormality and then what is the appropriate treatment to use. We’re seeing that in breast cancer, in melanoma, in many other kinds of cancer types.

There’s another‑‑there’s a related issue here, though, of course, which is that not everybody metabolizes drugs the same way, and so another reason to have diverse populations in a clinical trial is to learn about side effect profile of the drug, learn about the right dose of the drug to use. And we know full well, for example, that African‑Americans metabolize some drugs differently from white people, and so, depending upon how the drug is working in the body, an African‑American person might require a higher dose or a lower dose of the same drug that a white person would require in order to get the same therapeutic effect.

So it just speaks to the point again where if you don’t have diverse populations in the trials you can’t learn this stuff so that doctors then have the information they need to be able to prescribe the drug in the best way for their particular patient.

Andrew Schorr:
Okay. Here’s some questions we’ve been getting in. Kaitlin wrote in, Mel, she wants to know, do you still participate in follow‑up activities related to the trial you were in? So tell us about your participation and sort of follow‑up.

Mel Mann:
My follow‑up is I go out to MD Anderson twice a year, and it’s just a one‑day, one‑hour doctor visit where they take the blood work and they check and see if everything’s stable. And then when I’m back home, twice a year I have my blood work checked back at home, and that’s the extent of the follow‑up. I still have to take medication, one pill a day.

Andrew Schorr:
Right. And is that still covered by the trial?

Mel Mann:
Well, it’s covered by the trial, but my insurance also covers it. I did Gleevec for life because of the trial.

Andrew Schorr:
Okay. Dr. Schilsky, let me just ask you, is that a benefit typically of trials? Like with these oral cancer medicines which you know can be so expensive, if you’re in a trial for one do you get it for life or an extended time or how does that work?

Dr. Schilsky:
Depends a little bit on the trial and the sponsor for the trial, but the one thing for sure is when you’re on a trial whatever the investigational drug is, whatever is being researched, that’s provided for free. And any testing that would be considered to be for research purposes is provided by free‑‑for free. So that’s a benefit of participating in the trial.

Typically the drugs continue to be provided for free for as long as the patient continues to benefit. Now, sometimes if the drug ultimately gets FDA approved then it may be necessary at some time in the future for a patient to switch over from the research drug to the commercial drug, but of course at that time the drug is FDA approved and if the person has insurance it will generally be covered by their insurance.

Andrew Schorr:
Okay. We got a question in though for you, Dr. Schilsky, from Darrell. We were talking about genomic testing to understand what version of a disease we have either because of our ethnic background or some other thing that’s going on with us. As you know, insurance companies for a while have been balking at some of these more sophisticated tests, yet we and our doctor need that for us to get what may be right on target for us. So maybe you could talk about work that ASCO’s doing at all related to that. We want the testing done, but we also want to get it paid for so we can get that right, precise care.

Dr. Schilsky:
Yeah. It’s a complicated question because the testing is done at different points in the person’s illness. And so typically a test on a tumor specimen that’s necessary to determine a standard of care treatment, and many of these tests are referred to as companion diagnostic tests. Those tests typically are covered by insurance because the treatments themselves are also covered by insurance and the only way to know if you can get the treatment is to have the test done.

Now, where it gets a little bit uncertain is when you get into this sort of large‑scale genomic testing where a patient’s tumor might be tested for many hundreds of genes not really knowing what you’re looking for and not really knowing what you’re going to do when you find it. And that is where you’re beginning to bump up a little bit against, research and that’s where the insurance companies, sometimes some are reluctant to pay for that kind of testing.

Now, at least one of those large genomic profiling tests earlier this year was approved by both the FDA and Medicare and now will be reimbursed. So that’s the good news, and I think that’s the direction that most insurance companies are heading in.

One of the things that my organization is doing to try to understand how best to use these tests and how best to use targeted cancer drugs is we’re doing our own clinical trial that’s available in 20 states around the country, so not the entire country yet, but has already enrolled more than 1200 people on the study over the last two and a half years. And we’re doing this study to understand how this kind of genomic testing is done, what kind of treatment is recommended based on the results of the genomic test and whether or not that treatment actually works.

Andrew Schorr:
Cecelia, you mentioned earlier about the lady walked into the health fair and when you started talking to her about trials she said I got to go to work, and she walked out. And Dr. Schilsky was talking about eligibility requirements, but there are other issues where the study may be asking you to come back to some place or have multiple tests with some frequency so it’s not just leaving work one time. It may be leaving work 20 times. Have you had people voice that concern to you, that it’s just‑‑what’s being requested is just too much?

Cecelia Mann:
Yes. I think the lack of convenience for people who don’t have the funds or don’t search out the funds would definitely hinder them being on a clinical trial or being open to hear about the clinical trial. So, yeah, convenience and awareness. We try to spread awareness that, yes, after we talk about the disease, the myeloma and the symptoms then we go into the resources. And, you know, I make sure I tell them about calling the information line and talking to the masters level oncologist professional there and finding out about the latest trials, the latest treatments in addition to what they’re doing currently with their doctors or their family members or friend that is diagnosed with one of the blood cancers.

Andrew Schorr:
Dr. Schilsky, she’s getting at helping people sort out what trials are available. So medical science is a moving forward, and I’m sure you as an oncologist say, hallelujah, there are more trials than ever, but it’s often not only daunting to understand any one of them but to go through a bunch of them to understand what might be right for you. So how can ASCO help that? Is it just simply educating your doctor, or what can we do for families affected by cancer so they can get at what could be a match for them?

Dr. Schilsky:
Yeah, it’s a great question. So there are a lot of resources available, as Cecelia has mentioned. We can help patients understand and even begin to sort of, you know, wade through and winnow down the potential clinical trial options for them. One things that we’re working on and very interested in doing is sort of flipping the current paradigm by which trials are done. So right now, typically speaking, the patient has to travel to whatever site has the trial available. If they’re fortunate enough that that’s their own doctor’s office, that’s great, but, as in Mel’s case he had to travel to MD Anderson to get the trial.

The technology these days is at a point where we think we can flip that. Instead of making the patient go to the trial we’re going to work toward making the trial come to the patient. So if your doctor is aware that a trial exists somewhere in the world it should be possible for the doctor to basically just go to a website, find the research study, find the consent form, find the other documents that are necessary and present that to the patient. And if the patient qualifies just sign them up instead of making the patient travel hundreds of miles away to the one place that has the trial available.

Some trials have already gone down this road, and they’ve actually been recruiting very successfully, but it’s still not the usual way in which things are done, and we think we need to try to flip things around a little bit to make it easier for the trial to come to the patients. Let the trial travel. Let’s not make the patient travel.

Andrew Schorr:
That sounds great.

Mel Mann:
Can I add something to that, Andrew?

Andrew Schorr:
Sure.

Mel Mann:
Okay. As a veteran, I was part of the VA system, and I had to go out to MD Anderson, but this summer they started something called Navigate, the VA had started something called Navigate, which partners with the NCI. And it’s at 12 sites around the country, and it’s to bring the clinical trials to the VA. It’s right there. So if it’s an NCI clinical trial then the veteran can get on that clinical trial. And there’s a lot of African‑American veterans who can take advantage of that.

Andrew Schorr:
For sure. That’s terrific. So we’re going in the right direction. One other thing I think that needs to change is we talked about the scientists, whether they’re federal government scientists or drug company scientists, and they want to get answers to a whole bunch of scientific questions. So they may say, as you write the protocol I think it is, well, you have to get so many CT scans and you have to get so many blood tests and stuff like that. And it can become onerous, Dr. Schilsky.

What’s happening in trial design so that, A, we talked about eligibility, you can get into the trial, but the things you’re asking of me may have logistical hurdles as well that you’re kind the lightening up on it to get to the key scientific question without all these other bells and whistles that make it tough on me.

Dr. Schilsky:
Yes. I like to think of it as the need to know and the nice to know, right? There are certain things you need to know in the trial to be sure that the treatment is working, the patient is safe and not having any severe side effects and things of that sort. A lot of that stuff is the same stuff that doctors order every day on their patients as part of routine clinical care, and so much of what needs to be collected in clinical trials really aligns pretty well with standard of care.

Now, that said, because clinical trials are research and because there’s always new frontiers to explore, sometimes testing in a clinical trial extends beyond what the standard of care is. Sometimes patients are asked to give extra specimens of their blood, of their normal tissues, of their tumor tissues. Extra biopsies might be required, things of that sort. Patients need to understand why they’re being asked to do that, what those specimens are going to be used for, how is it going to advance research.

And, frankly, they’re very important to expanding the scope of the research. So, for example, oftentimes those specimens are used. If the treatment doesn’t work in a patient having those specimens can help the scientists understand why the treatment didn’t work, and that opens up a whole new horizon to explore to potentially make the treatment better in other patients.

Andrew Schorr:
Mel, do you recognize that by being in a trial and the work that you and Cecelia have been doing that you’ve probably helped thousands of patients by first being in a trial and then you and Cecelia talking about it?

Mel Mann:
Yeah. Yeah. I guess that’s kind of hard sometimes. You don’t see yourself in that role, but as I look back on it, yes.

Andrew Schorr:
Cecelia, you’ve probably talked to a lot of people. Have you seen a change where‑‑you’ve been doing this for a number of years where earlier on people said no, no, no. Are people more receptive? Do you see a change going on? Let’s say in the African‑American community, do you think people are a little more receptive?

Cecelia Mann:
Yes, I think so. I think they are more receptive, and this has a lot to do with education and awareness, and that’s what we are out there doing when we are out there in the community. And the more they hear about it and the more they read about it and the more they can see examples like Melvin, and we know one or two other people that we’ve met that were also on a clinical trial. One is in our church, and he had a type of leukemia, and we didn’t know why he was sick. But he is doing very well.

And so the more we can get those examples out there in the community of successful clinical trial patients, it really helps and goes a long way toward helping people of color relax and come aboard. And I just say, please, do your research, educate yourself and ask questions and please stay open and don’t dismiss clinical trials out of hand.

Dr. Schilsky:
And, Andrew, if I could just add to that. I just want to make the point that it’s people like Mel who are creating the future. Everything we know about how to treat cancer we learned from the people who participated in the clinical trials. We’ve been doing clinical trials in cancer for at least 70 years, and all of the standard of care treatments that we have today came from the participation of people in clinical trials. And that’s how we make progress. That’s how we’ll continue to make progress.

So it’s the clinical trial participants who, sure, they’re in it for themselves. We understand that. They’re looking for a new treatment, a better outcome, but they are the heroes of oncology because they are paving the way, trying the course and ultimately making a better future for every cancer patient who follows them.

Andrew Schorr:
Amen. Let me just recap a couple of things, and correct me if I get anything wrong, either of you. So, first of all, Dr. Schilsky, I know there are more trials now than ever before, and they’re now looking at these rare subtypes as well, and so if we participate we may get the benefit of tomorrow’s benefit today. Cecelia was talking about assistance programs, people to help you sort it out, that you are noticing how there are difference among us about the ways that drugs are effective or not, and that’s so important to learn.

If we partner with you, Dr. Schilsky, and the many thousands of oncologists and researchers that you represent, can we get to the goal line faster? In other words, are you hopeful that if we really consider trials and participate in trials and stay in trials and the different groups that we can get closer to cancer cures?

Dr. Schilsky:
Absolutely. I mean, we have more and better cancer treatments today than we’ve ever had before. We have all sorts of new and hopeful treatments on the horizon. We have to prove that they are safe and effective treatments to get them out there into routine clinical practice, and that’s where the clinical trials come in. So the more people who participate in trials the more quickly those trials can be completed and give us an answer, then the more quickly those drugs will make their way into standard clinical practice where everybody can benefit from them.

Andrew Schorr:
Okay. So a couple of to‑dos for our audience if you’re a patient or a loved one or however you hear this. Ask your doctor about whether trials apply to you or your loved one who is diagnosed with cancer even if it’s on day one. You don’t have to be at death’s door. You’ve been diagnosed or a diagnosis is suspected, what tests can we do, how do we know what we’re dealing with, and when we look at the treatment options is a trial a possibility if that makes sense, right?

Dr. Schilsky:
Absolutely.

Andrew Schorr:
Okay. Step two, are there resources to help me overcome any obstacles I may have to participation, first understanding the trial, understanding it in my first language, sorting out is it right for me, getting to where it is and then staying in the trial because many people, unfortunately, don’t stay in the trial and so how do be help the trial get to the goal line?

And then lastly, Dr. Schilsky, it sounds like you’re doing a lot at the community level to have more doctors have an easier time of the bureaucracy that we’ve had with trials before and the understanding of this flood of trials that’s happening, right?

Dr. Schilsky:
Absolutely. And, you know, to be perfectly honest, the clinical trial community has‑‑we ourselves have created some of the bureaucracy, some of the excess regulation, some of the barriers to participation. It’s up to us to strip those away and solve the problem and make clinical trials more broadly available. We are working very hard now to make that happen.

Andrew Schorr:
Okay. So whatever community you’re in. I’m in the Ashkenazic Jewish community. Mel and Cecelia are in the African‑American community. We have people watching who are in the Hispanic community, the Asian community. If you have benefited from a trial, talk it up, right? Cecelia, people should talk it up, correct?

Cecelia Mann:
Exactly, yes. Please, talk it up.

Mel Mann:
Yes.

Andrew Schorr:
Mel, thank you. I wish you really continued good health. How many years has it been since you were diagnosed?

Mel Mann:
Well, in about two months it will be 24 years.

Andrew Schorr:
Twenty‑four years, and for me it’s 22 years. And had there not been trials either that we were in or somebody else was in we wouldn’t be here.

Mel Mann:
Yes.

Andrew Schorr:
So, thank you. And also, Cecelia, thank you for being a community activist when it comes to trials and being supportive of Mel as he’s been in a trial because some other people would say, you can’t go there, you can’t do this, and you’ve been supportive every step of the way. Thank you for that.

Cecelia Mann:
Oh, you’re so welcome. It was a pleasure to do it.

Andrew Schorr:
Mel and Cecelia Mann from Atlanta. And Dr. Richard Schilsky, you’ve devoted your life to this, Dr. Schilsky, and I just want to say on behalf of the cancer patient community thank you and thank you for the leadership that ASCO is trying to do, both with changing research requirements, working with government, working with industry, and you thank you personally for your devotion to us. I really appreciate you being with us.

Dr. Schilsky:
It was my great pleasure. And, again, congratulations to Mel and Cecelia.

Andrew Schorr:
Okay. Thank you all. So this is what we do with our Clinical Trials Mythbusters program. Please tell others about it. The replay is available very shortly and all kinds of little highlights that we’ve done today. But what’s important is wherever you are is remember we can’t develop new medicines unless all of us work together to participate to get the scientific answers that apply to us, whatever our unique situation is, and then we can work with government to approve new medicines, get them on the market and so many people can benefit in the US and worldwide.

Thank you so much for being with us on this Patient Empowerment Network program. I’m gratified to be part of it. Thanks too to our financial supporters AbbVie, Astellas, Celgene and Novartis and their dedication to drug development and supporting and sponsoring clinical trials. I’m Andrew Schorr near San Diego. Remember, knowledge can be the best medicine of all.


Please remember the opinions expressed on Patient Empowerment Network (PEN) are not necessarily the views of our sponsors, contributors, partners or PEN. Our discussions are not a substitute for seeking medical advice or care from your own doctor. That’s how you’ll get care that’s most appropriate for you.