PC Treatments and Clinical Trials Archives

When it comes to treatment, prostate cancer patients and their care partners have much to consider. There are often many options available, each with advantages and disadvantages. Some people may seek 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.

More resources for Prostate Cancer Treatments and Clinical Trials from Patient Empowerment Network.

What Do Prostate Cancer Patients Need to Know About COVID-19?

What Do Prostate Cancer Patients Need to Know About COVID-19? from Patient Empowerment Network on Vimeo

Due to COVID-19, many patients with prostate cancer must follow new guidelines to receive care. Dr. Alicia Morgans, a hematology and oncology specialist, explains precautions patients should take and the role telemedicine plays in prostate cancer care.

Dr. Alicia Morgans is an Assistant Professor of Medicine at the Robert H. Lurie Comprehensive Cancer Center of Northwestern University.

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Prostate Cancer Research News

Prostate Cancer Treatment Decisions: Which Path is Best for YOU?

 


Transcript:

Dr. Alicia Morgans:

Men with prostate cancer, like every patient with cancer, do need to take precautions because of COVID, but the degree of caution that they need to take really depends on a couple of factors. One is probably that individual’s age, with older people being more susceptible to having severe complications related to COVID, especially if they have other medical conditions like COPD or lung disease or heart disease with a history of things like heart attack or stents in the heart. Things like diabetes can even increase the risk of having complications, according to some studies, for people with cancer. So, these are things to think about. Comorbid illness and certainly advancing age.

The other thing that I always think about is what kind of therapy are you getting as a man with prostate cancer? Are you getting something that really is only affecting hormones, like lowering testosterone levels or blocking testosterone signaling? That’s the male hormone. Hormonal treatments don’t suppress a person’s immune system. So, they don’t change the way that an individual’s immune system can attack the COVID virus and protect them from that illness. And those kinds of treatments are not as dangerous to use in a pandemic like we’re experiencing now, because they don’t affect a person’s ability – their innate and normal ability – to fight off the disease.

Things like chemotherapy, on the other hand, do suppress the immune system. They make it difficult for the immune system to fight things like that SARS virus, SARS-CoV-2, that causes COVID-19, because it suppresses the immune system such that a patient can’t mount the normal response that he would have against that virus if it came into his body.

When we don’t have an immune system, we can be more susceptible to things like that SARS-CoV-2 virus that causes COVID-19, but we can be susceptible to things that we would find in our normal environment and sometimes even to infections from bacteria that live in our body all the time. So, things like chemotherapy can be challenging whenever you take them. They can be incredibly effective against cancer. And so, it’s always this trade-off.

And if it’s recommended to you, you can get it safely, but taking extra precautions with, of course, washing hands, wearing masks, but also, probably, really still socially distancing even though some of the restrictions in most of the United States have lessened. If you’re on chemotherapy, I would still recommend social distancing and staying out of public places, because you do not necessarily have the immune system that you would normally have to protect yourself from the virus.

Telemedicine has been great for men with prostate cancer when they don’t necessarily need to come in to be seen. This can be really helpful, especially between visits where people are getting injections that they get to lower testosterone as androgen deprivation therapy. If that injection is due every three months or four months, but your doctor wants to check in on you every six weeks or eight weeks, having a telemedicine visit at that interim visit can be really useful so you don’t have to come all the way into the clinic to see the provider.

They can even be useful if you do need to get the injection or you do need to get lab work, because you can get those procedures and then go home and still be safe not sitting in a waiting room, not sitting in a doctor room. And the doctor can usually call and have that telemedicine visit.

For men who have been treated and are simply having their PSA followed because they’ve had a prostate surgery or have had radiation to the prostate and are believed to be cured, as long as they can get that lab work done, the telemedicine visit gives them the opportunity to get the guidance of their doctor who has looked at their lab work, without actually going in to see that doctor in person and potentially put themselves at risk of getting an infection in the in the clinic or the hospital setting.

So, telemedicine is a way for us to really protect our patients and stay engaged while we’re not seeing them in person. But it is still important to do the telemedicine and not just say I’m not going to do anything. And it will be important at some points for many men with prostate cancer to come in at least to do lab work or to get their injections if that’s part of their treatment plan to make sure that they are still being monitored despite the pandemic.

Prostate Cancer Research News

Prostate Cancer Research News from Patient Empowerment Network on Vimeo

Are there developments in prostate cancer research that patients should know about? Dr. Alicia Morgans discusses highlights from the 2020 American Society of Clinical Oncology (ASCO) meeting.

Dr. Alicia Morgans is an Assistant Professor of Medicine at the Robert H. Lurie Comprehensive Cancer Center of Northwestern University.

See more from The Pro-Active Prostate Cancer Patient Toolkit

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What Do Prostate Cancer Patients Need to Know About COVID-19?

Prostate Cancer Treatment Decisions: Which Path is Best for YOU?

 


Transcript:

Dr. Alicia Morgans:

Just recently in June, ASCO, which is our American Society of Clinical Oncology, meeting was held here in Chicago, and it was a virtual meeting. It was actually very exciting for people who take care of prostate cancer and for men who have prostate cancer in several advances. Some of those advances were around imaging and new strategies that we’re going to have, I think, in the relatively near future using PSMA-targeted imaging for men who have prostate cancer that is high-risk before they go through things like surgery or radiation, or for men who have a rising PSA after they’ve had their initial treatment for prostate cancer.

We also learned the survival data that was associated with three agents that we now have to treat non-metastatic castration-resistant prostate cancer. And this is prostate cancer where we have had a group of men who have already had treatment of their prostate, but now have a rising PSA blood level despite having imaging that doesn’t really show any areas of cancer on the scans. And there are three drugs that we have to use for men with this particular stage of prostate cancer, or state of prostate cancer, and we learned that those drugs not only prolong the time until men develop metastatic disease or disease that we can see on those scans, but they also help men live longer.

And this tells us that if we move those therapies earlier on in the stage of treating prostate cancer, we can actually, probably bend the curve of that man’s survival for the rest of his life. Intervening early, at our earliest opportunity, in this particular situation may be so helpful for men over the rest of their journey, no matter what their next treatments might be.

And finally, we learned information about a drug called lutetium, which is not yet approved for the treatment of prostate cancer, but was tested in a clinical trial for men with more advanced prostate cancer and called metastatic castration-resistant prostate cancer. And we learned that this drug can be both tolerable and potentially as effective, or perhaps more effective, than the chemotherapy that we have traditionally used in this state. So, lutetium is a drug that we expect will eventually be approved for the treatment of prostate cancer, pending some clinical trial data that we are still waiting for. And that was real exciting, to learn about the upcoming advances with this particular drug.      

Why You Should Consider a Prostate Cancer Clinical Trial

Why You Should Consider a Prostate Cancer Clinical Trial from Patient Empowerment Network on Vimeo

Dr. Alicia Morgans, a hematology and oncology specialist, explains the importance of prostate cancer patients of different geographic locations participating in clinical trials and the role trials plays in clinical care.

Dr. Alicia Morgans is an Assistant Professor of Medicine at the Robert H. Lurie Comprehensive Cancer Center of Northwestern University.

See more from The Pro-Active Prostate Cancer Patient Toolkit

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Are You Prepared for Your Prostate Cancer Appointment? Expert Tips

Prostate Cancer Research News

Prostate Cancer Treatment Decisions: Which Path is Best for YOU?

 


Transcript:

Dr. Alicia Morgans:

From my perspective, I think any time in a prostate cancer journey is a great time to think about a clinical trial if that trial is available where you live or is available at a place where you would be willing to travel. We have so much to learn about prostate cancer, about how to continue to provide options to patients, and about how to support men as they go through their treatment. And the only way we can learn those things is if men participate in clinical trials. So importantly, also, we need to have men of diverse backgrounds of diverse races from geographic diversity.

Because if we only study certain people from the city of Chicago, for example, where I live, we’ll really only know what we know about those men. And we won’t necessarily know if we can apply our findings to men who live in Atlanta and are Black. It’s going to be the kind of thing where we have the data, but we don’t necessarily know if it’s going to be the right data for you.

So, the more men of color, the more men from different geographic locations that we can encourage to participate in clinical trials, the more we learn for every patient and the more we are able to take care of the specific and unique needs of you as an individual, which is really a critical part of what we do and why we do what we do. So, participating in clinical trials, no matter who you are, if you’re able, and if you’re willing, is really a great service to you to get, hopefully, better outcomes for you, but also a great service to your community of men with prostate cancer.  

Prostate Cancer Treatment Decisions: Which Path is Best for YOU?

Prostate Cancer Treatment Decisions: Which Path is Best for YOU? from Patient Empowerment Network on Vimeo.

Which prostate cancer treatment path is best for you? Dr. Alicia Morgans discusses how multiple factors, including disease progression and patient goals, determine which treatment path is best to help improve a patient’s outcome and overall quality of life. 

Dr. Alicia Morgans is an Assistant Professor of Medicine at the Robert H. Lurie Comprehensive Cancer Center of Northwestern University.

See more from The Pro-Active Prostate Cancer Patient Toolkit

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What Do Prostate Cancer Patients Need to Know About COVID-19?

Prostate Cancer Research News

How Does Prostate Cancer Staging Affect Treatment Approaches?

 


Transcript:

Dr. Alicia Morgans:

The main factors I think about when approaching a treatment plan for a patient is to understand is this treatment for cure. Are we able to cure this patient? Is that our goal? Or are we in a situation where we know that the cancer is going to be incurable, but we can prolong that individual’s life and improve the quality of life that he has?

That is a major breakdown or separation point in how we approach treatment. Once we figure that out, we can try to sort through among all the choices. If we’re going to use curative treatment to the prostate itself, what do we think is best for you as an individual man? And what do we think is possible from a medical perspective? Whether that’s radiation or surgery or even just watching and waiting with an active surveillance plan, there may be choices.

And similarly, with metastatic prostate cancer or advanced prostate cancer that’s incurable or not able to be cured, what are the medical treatments that we can use? And what are the choices that you as a man with prostate cancer want to make to really maximize your benefit – thinking through what’s important to you? What barriers do you have? And how do you want to go through your treatment sequence?

We’re actually really fortunate in prostate cancer care to have many choices, whether it’s in treating localized curable prostate cancer or in treating metastatic prostate cancer that we’re really trying to treat to prolong life and improve quality of life. In each setting, in most cases, there are multiple choices to make along the journey. Sometimes these choices would exclude other choices in the future, but sometimes they don’t. Sometimes you can choose A or B, because in a few months, you’re going to have the opposite option available to you. So, exactly what your choices are going to be are going to be important for you to speak with your doctor about.

But having those choices really empowers men to get engaged in each of these treatment decisions to explain this is my preference for that side effect or this particular toxicity, and I’m going to choose this treatment, because it works best for me because I can get to work or because it doesn’t lead to incontinence or because it doesn’t cause me to lose my hair or whatever the reason is. Men’s preferences can be so importantly incorporated into the treatment decision, because we have all the choices we have in treating prostate cancer.

How Does Prostate Cancer Staging Affect Treatment Approaches?

How Does Prostate Cancer Staging Affect Treatment Approaches? from Patient Empowerment Network on Vimeo

Every stage of prostate cancer stage requires different treatment approaches. Dr. Alicia Morgans explains prostate cancer staging and how it impacts treat options.

Dr. Alicia Morgans is an Assistant Professor of Medicine at the Robert H. Lurie Comprehensive Cancer Center of Northwestern University.

See more from The Pro-Active Prostate Cancer Patient Toolkit

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What Do Prostate Cancer Patients Need to Know About COVID-19?

Prostate Cancer Research News

Prostate Cancer Treatment Decisions: Which Path is Best for YOU?

 


Transcript:

Dr. Alicia Morgans:

Staging in prostate cancer is a way for people to understand how to best approach the treatment of the disease. To say this a different way, low stages – things like Stage I, II, and usually Stage III – can be treated with local therapies to the prostate itself with a goal of trying to cure the prostate cancer. And some patients who have Stage I disease may not even need active treatment, but could be followed on active surveillance as a way to monitor the cancer and prevent side-effects by simply monitoring until it would actually need treatment. Higher stage, like Stage IV, means that the cancer has spread outside of the prostate.

And it’s still prostate cancer. It just is cancer cells from the prostate that now live in the bones, or live in distant lymph nodes, or live in another organ or place in the body. Those cancer cells are still treated the exact same way we treat prostate cancer in terms of the medical therapies – the injections, the pills, the chemo agents potentially – that we would use to treat those cancer cells, whether they’re in the bones or in the prostate. But when they have spread outside of prostate, that typically means that there’s no longer an opportunity for us to cure that cancer. And we wouldn’t necessarily use things like surgery or radiation to the prostate if the cancer had spread.

I say “wouldn’t necessarily,” because that is certainly an area that’s evolving. And now even men with metastatic prostate cancer or Stage IV prostate cancer can be treated with radiation, in particular, to the prostate, and we know that can be beneficial. So, staging helps us understand how far the cancer has spread or not spread.

And it helps us understand if we can treat that patient with local treatments to the prostate to try to cure them, or if we need to use medical therapies as a major backbone of treatment rather than things like radiation or surgery to treat them for prolonging their life and improving quality of life but knowing that we can’t cure their disease.

How Can You Access Personalized Prostate Cancer Treatment?

How Can You Access Personalized Prostate Cancer Treatment? from Patient Empowerment Network on Vimeo.

How could genetic testing results affect your prostate cancer treatment plan? In this INSIST! Prostate Cancer webinar, Dr. Sumit Subudhi will discuss key prostate cancer tests, the latest targeted therapies and tools to help you advocate for a personalized treatment approach and insist on better care.

Dr. Sumit Subudhi is a Medical Oncologist at The University of Texas MD Anderson Cancer Center.

Download Program Resource Guide

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Three Key Steps to Take Following a Prostate Cancer Diagnosis

 

 


Transcript:

Katherine:                  

Welcome to Insist Prostate Cancer, a program focused on empowering patients to insist on better care. Today we’ll discuss the latest advances in prostate 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. Sumit Subudhi. Welcome, Dr. Subudhi. Would you please introduce yourself?

Dr. Subudhi:        

Hi, I’m Sumit Subudhi. I’m a medical oncologist at MD Anderson Cancer Center, and I specifically focus on prostate cancer.

Katherine:    

Excellent, thank you. Before we start, a reminder that this program is not a substitute for seeking medical advice. Please refer to your own healthcare team. Well, Dr. Subudhi, I’d like you to begin with a brief explanation of the stages of prostate cancer.

Dr. Subudhi:               

Yeah, that’s a great question. So, we use stages, and there’s four – Stage I, II, III, and IV. And we use it to help us determine what treatments the patients need for their prostate cancer. In general, Stage I is localized prostate cancer, and it’s localized only to the prostate. And when we do a digital rectal exam, we cannot feel or palpate the prostate.

And the treatment for Stage I prostate cancer is either active surveillance, where you’re not trying to cure the cancer, you’re just actively watching it, and you’re using a PSA imaging studies, prostate biopsies, and digital rectal exams at regular intervals to follow the patients. But other patients with Stage I prostate cancer can actually get definitive treatment for curative intent with radiation therapy or surgery. Stage 2 prostate cancer is also localized, but on physical exam, we can actually palpate or feel the prostate cancer. And this also can receive definitive treatment for the prostate to cure it, and that, also, you can use radiation therapy and surgery.

Stage III is what I consider locally advance. This is where the prostate cancer is now starting to leave the prostate. And it still can be cured by radiation and surgery, but most likely needs a multidisciplinary approach, where you might need both or maybe even in addition of a systemic therapy. Stage IV is the last stage that I’ll talk about, and it has distant metastases. And here we’re not looking for a curative approach; we’re actually looking for palliation, which means that we’re trying to treat the prostate cancer as a chronic disease.

Katherine:                  

I understand that there are many types of prostate cancer that have been identified. How can patients advocate for a precise diagnosis?

Dr. Subudhi:    

Yes, you’re absolutely right. There are many types. So, we have historically used histological classification. And when I say histological, that means when we look at the cancer under the microscope, we can look at the different structures within the prostate cancer and classify them.

And there are multiple types such as adenocarcinoma, neuroendocrine, small-cell, mucinous, etc. But more recently, with the advances in genetic and molecular testing, we now can look at the genes inside the prostate cancer, and that has also helped us better classify the cancer. Now many of these types of approaches are best done at major cancer centers, where they have experienced pathologists who actually evaluate both histologically and molecularly the cancer.

So, I recommend to my patients, or family and friends, that have been diagnosed with prostate cancer that they don’t necessarily have to go to the major cancer centers. They can have their local doctor send the tissue from the biopsy to the advanced cancer centers to get a second opinion.

Katherine:    

But this would be an initial visit to a doctor. Is that right?

Dr. Subudhi:    

Good question. So, I’m presuming that the patient is actually being seen at a local center where they have a local doctor, and so they don’t have to come, for example, to MD Anderson Cancer Center to see me. They could actually have their tissue sent to our pathologists and get it reviewed, and they can still be at home. And especially in this era of COVID, that’s important.

Katherine:      

What is the role of genetic testing in prostate cancer?

Dr. Subudhi:     

That’s a great question, because this is something that wasn’t really available when I was training and understanding prostate cancer. But over the last few years, this has actually hit the mainstream, and it’s very important. And I see it having three roles. The first role is whether or not you can receive a certain type of targeted therapy or systemic therapy known as PARP inhibitors. So, if your genetic test is positive for certain markers – that I think we’ll cover later – then it can help give you more treatment options. The second is that generate testing can give you also risk of other cancers besides prostate cancer. For example, if you have the BRCA mutation, you’re 15% to 20% more likely to get breast cancer in men.

The third is that because the genetic testing is looking for inheritable mutations in your genes, that means you can pass it along to your kids. And this could have a tremendous impact on the screening strategies your children want to use in the future.

Katherine: 

Would you mind going into that a little bit?

Dr. Subudhi:  

Yeah.

Katherine:  

For instance, my ex-husband had early prostate cancer. My 22-year-old son is worried now about also getting prostate cancer. His grandfather had prostate cancer.

Dr. Subudhi:               

Yeah, great question. So, it’s not just about prostate cancer. So, prostate cancer, genetically, is linked to other cancers, as well.

So, in your case, you’re turning by your son. But if you have daughters or any female members in the family, consideration needs to be given to breast and ovarian cancer. And for both men and women, we also have to think about melanoma and pancreatic cancer. So, it’s not just prostate cancer that we’re thinking about when you have these genetic risks. And that’s very important, because each of these different cancers can have different screening modalities.

Katherine:                  

Oh. Well, how is the testing administered then?

Dr. Subudhi:               

The testing is actually a blood test, so very simple.

Katherine:                  

Have there been any major advances in testing?

Dr. Subudhi:               

Yeah, so when we’re talking about the inheritable testing, that’s just a simple blood test. And the reason why it can be done simply through the blood is because every cell in your body has it. So, when they collect the blood, they can just take any cell from there and do genetic analysis. And if that gene is mutated or missing, it will be captured.

Now, there’s another type of testing where they test your tumor tissue itself – so, your cancer tissue – whether you got it by biopsy or surgically removed. And so, that’s a different type of testing. That’s looking for what we call somatic mutations. These are not inherited mutations. These are mutations that are specific for your prostate cancer. Again, in contrast, the inheritable mutations are in every cell in your body – not just your prostate cancer cells, but every cell in your body. And the somatic, it’s just in your prostate tissue itself.

And so, sometimes with prostate cancer, it’s difficult to get the tissue. And what’s happened more recently – and to answer your question – is that the advances have been in what we call liquid biopsies, where they are able to use your blood and get the DNA from the tumors and actually genetically test the cancers that way. And so, that’s where the future is going.

Katherine:      

Oh, that’s amazing. Are there specific tests that patients should ask their doctor for following the diagnosis?

Dr. Subudhi:   

Yeah. So, if inpatients with high risk or metastatic prostate cancer, they should definitely be considering tests to see if they have mutations in what we call the DNA damage repair pathway or homologous recombination DNA pathway. And I know they’re fancy terms. What these genes are, they’re genes that help the body repair their DNA, and DNA is very important. And so, when there’s defects in the DNA repair pathway, then mutations occur. And these mutations can actually help the cancer grow.

Now what’s happening is that what they’re looking for in these genetic tests – whether it’s the inheritable test or the somatic mutation test that’s looking just within the tumor itself – they’re looking to see if there’s any DNA damage machinery that’s defective. And if it is, then you’re more likely to benefit from PARP inhibitors, which are oral drugs that specifically target the DNA repair pathway.

Katherine:    

All right. Dr. Subudhi, what is the link between inherited mutations and prostate cancer?

Dr. Subudhi:               

Yeah, so in approximately 10% to 15% of patients with prostate cancer, they have an inheritable cause for their cancer. And so, this predisposes them to not just having prostate cancer, but potentially to other cancers, but also their family members.

Katherine:                  

Would you give us an overview of common mutations in prostate cancer?

Dr. Subudhi:               

Yeah. So, in regards to the inheritable causes, the BRCA mutations – BRCA2 and BRCA1 – are very common. In fact, BRCA2 is more common than prostate cancer than BRCA1. In addition, there’s CHEK2 and ATM which are common inheritable mutations. And the other ones are the mismatch repair genes. Again, all these play an important role in repairing DNA. So, if you’re mutated in these genes, then your ability to repair DNA has been significantly diminished, and you’re more likely to gain more mutations.

Katherine:                  

How do these mutations affect disease progression?

Dr. Subudhi:               

Yeah. So, what they can do is they can lead to mutations that make the cancer grow more. And there’s two ways to do it. You can have a mutation in what we call an oncogene, a gene that when it’s active, it’s going to just promote the cancer.

And then we have other genes called tumor suppressor genes. Their normal function is to prevent the cancer from growing. But if the tumor suppressor gene gets mutated so it’s no longer functional, then the cancer can then take off, because it’s no longer suppressed. So, those are how these genes can actually affect the prostate cancer.

Katherine:                  

What about treatment options, what’s available?

Dr. Subudhi:    

Yeah. So, if you have either an inheritable mutation in these genes or a somatic mutation, then there’s a chance that the PARP inhibitors could actually work for you. And the PARP inhibitors, they actually target cancers where there’s a defect in the DNA repair pathway.

Now, there’s one thing that I want to point out that a lot of people sort of are missing, and it’s not a subtle point. Not all inheritable mutations are made the same – or even somatic mutations. Meaning, what we’re learning is the PARP inhibitors seem to be more active with the “Braca,” or BRCA, mutations and the ATM mutations. Whereas, they’re less active with other types of DNA repair mutations. So, the point is not all mutations are made the same.

Katherine:                  

Let’s turn to targeted therapies. How exactly do they work?

Dr. Subudhi:      

Yeah. So, this is a form of personalized medicine. So, what you’re doing is you’re looking at the patient’s cancer, either their inheritable cause of genetic causes or the somatic. And then you’re saying, oh, wait, they have a genetic defect in a DNA machine. So, let’s use the PARP inhibitor, which also targets the DNA machinery.

And these are the cancer cells that are most likely to be susceptible to PARP inhibition. And actually, the cancer cells will die from it. Whereas if a patient has a normal DNA machinery, the PARP inhibitors will actually not have any effect on the cancer.

Katherine:                  

Oh, I see. Just as a follow-up, how are these targeted therapies administered?

Dr. Subudhi:               

They’re given, actually, orally twice a day. The two drugs are rucaparib and Olaparib that have been FDA approved for this indication.

Katherine:                  

How do these newer treatments differ from traditional chemotherapy?

Dr. Subudhi: 

That’s a great question. So, with chemotherapies, at least in prostate cancer, they’re given intravenously every three weeks. And the goal of the chemotherapies, they are actually designed to kill any actively dividing cell in the body.

And the problem is it’s not just cancer cells that are actively dividing in our body. For example, with the chemotherapy such as docetaxel or cabazitaxel, that’s used in prostate cancer – their brand names are Taxotere and Jevtana – these chemotherapies will also affect hair loss. Why? Because hair grows really fast. And in fact, I need a haircut every three to four weeks, which my wife has been helping me with.

So, the chemotherapies are targeting all actively dividing cells, and that’s why you also get nausea vomiting, because the cells of our GI tract are also affected by that. So, chemotherapies are not personalized. They’re there to kill actively dividing cells. Luckily prostate cancer divides a lot more quickly than any other cell in our body, and that’s why they’re susceptible to chemotherapy.

Katherine:   

And as far as the targeted therapies, Dr. Subudhi, are there side effects with those?

Dr. Subudhi:     

Yeah, there are. One of the most predominant side effect is actually anemia. And so, that’s when the red blood cells in our body are lower than usual. And so, that’s one of the major side effects for PARP inhibitors. But in addition, you can have nausea, vomiting, and diarrhea as other side effects with the PARP inhibitors.

Katherine:  

What are you excited about in prostate cancer research right now?

Dr. Subudhi:  

So, to me, it’s the combination of treatments. So, not just treating with one PARP inhibitor or just one hormonal therapy, it’s combining these approaches, and especially with immunotherapies, so that we can potentially cure what’s considered incurable cancer. To me, that’s the most exciting.

Katherine:   

What would you say to patients who are nervous about participating in a clinical trial?

Dr. Subudhi:    

Yeah. This is a common question that I deal with in clinic, because we tend to have a lot of trials at MD Anderson. And the first thing, for me, is to understand why they’re nervous, because there’s different reasons why people are nervous.

Some people have heard of placebo trials, where the experimental drug that they’re hoping to get is only given to a portion of the patients and not all. And so, patients are worried what if they get on the placebo arm. And so, what I tell patients in that case is that please note that you’re going to be monitored very closely – more than usual, and so I’ll be seeing you in clinic more often. And if there’s any signs of progression, I will take you off the study. But I also always have a back-up plan. So, I tell them this is the next drug I’m going to give you if you progress, so don’t worry, I’ve got a plan for you. So, that’s one thing.

The other thing that people get concerned about are experimental drugs – just the fact that they are experimental. And I have to remind them that all these standard therapies that we have for prostate cancer were all experimental at one point. And it was the courage of the other patients that went through clinical trials that helped bring it as standard of care. And then sometimes some people have issues with travel, and those are more logistical issues. And especially now with the COVID era, we have to think about that. And so, we’re also trying to find and use networks to see if there’s other trials that are more amenable for patients so they don’t have to travel far.

Katherine:     

How can patients find out about clinical trials that may be right for them?

Dr. Subudhi:               

Yeah. So, one way is using clinicaltrials.gov. And that’s a website that allows you to search for specific trials either by drug name or by disease type – so, for example, prostate cancer. So, that’s one resource. And the others are cancer societies like the American Cancer Society or ASCO or Prostate Cancer Foundation. They also have links to clinical trials that are exciting.  

Katherine:                  

Do you recommend having patients see a specialist?

Dr. Subudhi:               

Absolutely. I think that if you have a metastatic disease, you need to have a medical oncologist on board that can still work with your urologist, who’s more surgically trained.

Katherine:                  

Right. Well, we’ve talked about COVID a couple of times, and I’d be remiss if we didn’t touch upon it now. What should prostate cancer patients be considering at this time, especially those with advanced disease?

Dr. Subudhi:               

Yeah, so what I don’t want are people to say, oh, I can wait a little bit longer to contact my physician, whether it’s primary care or a prostate cancer doctor, because of COVID. I think it’s very important that the medical team is up to date on a patient’s symptoms and what’s going on medically, whether it’s related to prostate cancer or not. And so, that’s one of the messages we’ve been trying to pass on to our patients.

And with every single patient, we try our best to see if we can provide medical care as well as expertise without having physically see them through telehealth, whether it’s through video or whether it’s just through a simple phone call. So, we’re trying to look at that with each individual patient. Now, sometimes when there’s treatment decisions that have to be made – especially like do we start chemotherapy? – it’s harder to do that over the phone. But sometimes what I’ll do for my patients that are in areas where COVID is really worrisome, I’ll work with the local medical oncologist and talk to them and basically see if we can develop a plan together so that the patient can be served best.

Katherine:                  

Is there a time when telemedicine is more appropriate than others?

Dr. Subudhi:               

I think that has to be a case-by-case basis. And that’s how we do it in clinic.

Every week, a week before hand, I go through my entire clinic list – I go through each patient’s case – and I say, okay, this is a patient that would be better served with telemedicine, this is a case that I really need to see the patient to get a better sense of what to do next.

Katherine:                  

If a patient has to go into clinic, what safety measures are in place for them?

Dr. Subudhi:               

Yeah, so I can only speak for our hospital and how we’re doing it. But there’s actually like a thermal scan where when you walk in the building, they actually measure your temperature without you even knowing it. But they delete it, so it’s not something that’s kept. And this is still kept private, so you don’t have to worry about public disclosure of your temperature. And so, they’re monitoring both the staff and the patients – their temperatures. The staff themselves have to go through screening questions that they have to answer every time, and they’re actually handed a mask that’s required to be used at all times.

As far as patients go, they are also getting their temperature measured. But in addition, they are asking questions about their exposures, whether it’s family members that are asymptomatic, or not symptomatic. And in addition, the new patients will get a COVID test done prior to seeing the medical team. And for the follow-up patients, they’re not required to get a COVID test unless there’s concerns of symptoms.

Katherine:                  

Unless they’ve been exposed to somebody?

Dr. Subudhi:               

Correct, that’s right. Thanks for pointing that out. In addition, all patients are asked to wear their masks at all times, especially if they’re going to be within 6 feet of a healthcare provider or a patient or anyone else. And so, these are the measures that we’re taking to keep our patients safe.

Katherine:                  

Oh, good. Dr. Subudhi, what advice do you have for patients who may be hesitant to speak up an advocate for themselves when it comes to their own care and treatment?

Dr. Subudhi:               

Yeah, I’d say that it’s interesting because we all do this ourselves. And when it comes to our car – let’s say a car breaks down, or if we’re trying to buy furniture – we’ll get three, four different opinions. But for ourselves, for our own body, we don’t do that. And when you watch – we were talking earlier about Major League Baseball – and these players, when they get injured, they get the three best specialists in the world to evaluate them. And they’re seeing the best of the best.

And so, we owe it to ourselves and the patients owe it to themselves to actually get second opinions. I encourage it. I encourage my patients to get second opinions, even if I’m the first doctor they see, because I want them to feel comfortable with their decision.

And it’s important to understand that just because you’re seeing a doctor doesn’t mean that it’s a one-size-fits-all. You will get different opinions from different doctors, and you have to go with the one that makes you feel most comfortable.

Katherine:    

We have a question from the audience, Dr. Subudhi. Amy is saying she’s the daughter of a prostate cancer patient. And she’s curious to know how she goes about getting genetic testing, and if her children should be tested, as well.

Dr. Subudhi:               

Yeah. So, one of the things is that family history is very important in determining who should get genetically tested. So, if you’re a prostate cancer patient and you have metastatic disease, you should get genetically tested. And the reason for that is because we have a new set of drugs, the PARP inhibitors. But if you’re a family member that’s wanting to know whether you have a loved one has inheritable cancer that you may end up inheriting, that requires more understanding of the family history.

For example, did the grandfather have prostate cancer? Did the uncle have prostate cancer? And as I mentioned earlier, it’s not just prostate cancer. Is there a family member with breast cancer or ovarian cancer? These things play out in the decision-making of who should be genetically tested.

Katherine:                  

Absolutely. As a researcher in the field doctor, Dr. Subudhi, what would you like to leave patients with? Are you hopeful?

Dr. Subudhi:               

Yeah, I’m very hopeful. It’s a really interesting time, because with the technological advances and scientific advances – Traditionally, prostate cancer has always been treated just with hormonal therapies from the 1930s all the way to early 2000. Then in 2004, chemotherapy became the next thing. And then after chemotherapy, we’ve now got a dendritic cell vaccine; we’ve also got a radiopharmaceutical agent. And so, what the point is now we have a lot more different FDA-approved agents. And now, experimentally, the PARP inhibitors have now become a standard of care for those patients with mutations in the BRCA1, BRCA2, or ATM.

And then, in addition, we have many other types of technologies, such as BiTE and CAR T cells, that are coming out that are showing in early studies to be exciting. And so, I feel like that these therapies in combination may actually lead us to cure the cancer.

Katherine:                  

ASCO happened in June. Was there any news that patients should know about?

Dr. Subudhi:   

Yeah, so the PARP inhibitors got a lot of press during ASCO, as they should, because this is a new class of drugs that is the first personalized version of medicine that we have in prostate cancer. Now, personalized medicine has been around for a long time in cancers such as breast and lung cancer. But for first time, we actually have it in prostate cancer.

Katherine:

Dr. Subudhi, I want to thank you so much for joining us today.

Dr. Subudhi:

Thank you for your time. I really appreciate it.

Katherine:

And thank you to all of our partners. To learn more about prostate cancer and to access tools to help you become a more 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.

New Website Educates Prostate Cancer Community about Chemotherapy

This article was originally published by Yahoo! Finance on January 13, 2020 here.


Three non-profit organizations have joined together to help prostate cancer patients better understand chemotherapy and when its right for them

DENVER, Jan. 13, 2020 /PRNewswire/ — Prostate cancer advocacy organizations ZERO, Us TOO International and Prostate Conditions Education Council announce the launch of an educational website specific to chemotherapy for treating prostate cancer.

It is estimated that more than 191,000 men in the U.S. will be diagnosed with prostate cancer in 2020. While most prostate cancer is diagnosed at an early stage, prostate cancer can recur or advance after an initial treatment; and some men have an initial diagnosis of advanced prostate cancer. Once prostate cancer is advanced, it can be managed but not cured. Chemotherapy is a common treatment for managing advanced prostate cancer but not routinely considered as a treatment option at other stages of the disease.

The prostate cancer chemotherapy website provides objective, balanced information about how chemotherapy works and when it should be considered as a treatment option. It addresses using chemotherapy in combination with other treatments and the potential for sequencing it in a treatment regimen. Along with the benefits of chemotherapy, probable treatment side effects are outlined as well as information for managing side effects.

“The purpose of the website is to provide an enduring educational platform on prostate cancer chemotherapy with current, accurate, and unbiased information,” noted Wendy Poage, President of the Prostate Conditions Education Council. “Content will help to educate patients and caregivers about how chemotherapy fits into the evolving prostate cancer disease journey. It features questions to ask your doctor and debunks some common myths about chemotherapy.”

The idea for the website was the result of a brainstorming session at an industry meeting attended by all of the participants. A discussion about current challenges for finding comprehensive information about chemotherapy for prostate cancer led to the idea of developing the website.

“Patients are finding it increasingly difficult to understand various prostate cancer treatment options throughout the disease journey,” stated Jamie Bearse, CEO of ZERO. “This website will serve as an important online destination for the prostate cancer community to find reliable facts about chemotherapy.”

A common deliverable of the three prostate cancer nonprofit organizations collaborating on this project is educational content to help men living with prostate cancer make informed treatment decisions at all phases of the disease. In addition to comprehensive educational written content and videos, the website will feature links to other resources including support groups.

“Beyond the facts and stats, it’s important to provide opportunities for men with prostate cancer to learn from each other and share their personal experiences with treatment decisions and side effect management,” said Us TOO International CEO Chuck Strand. “This website includes connections to support groups for men to exchange peer to peer information in person, online, or on a conference call.”

Collaboration on the chemotherapy prostate cancer website will extend beyond the launch to include future content updates.

The chemotherapy website can be found at any of the web addresses below:
https://www.prostateconditions.org/about-prostate-conditions/prostate-cancer/chemotherapy 
https://www.ustoo.org/chemotherapy 
https://zerocancer.org/learn/current-patients/types-of-treatment/chemotherapy/

About Prostate Conditions Education Council
A national organization committed to men’s health, the PCEC is the nation’s leading resource for information on prostate health. The PCEC is dedicated to saving lives through awareness and the education of men, the women in their lives and the medical community about prostate cancer prevalence, the importance of early detection and available treatment options, as well as other men’s health issues. The Council, comprised of a consortium of leading physicians, health educators, scientists and prostate cancer advocates, aims to conduct nationwide screenings for men and perform research that will aid in the detection and treatment of prostate conditions. More information is available at prostateconditions.org.

PCRI: Managing Side Effects of Chemotherapy

This video was published by the Prostate Cancer Research Institute on June 25, 2019 here.

 

Transcript

Hi, I’m Dr. Scholz. Let’s talk about prostate cancer.

In the video today we want to cover how to minimize side effects from chemotherapy. Fortunately, chemotherapy is not often required with prostate cancer; hormonal therapy is very effective. We reserve chemotherapy for more advanced situations. Usually, men that have some sort of metastatic disease. We’re covering this particular section in Indigo, that is men that have enlarged lymph nodes. In particular, in High-Indigo studies are now showing that a short course of chemotherapy can improve cure rates and improve survival.

People enter into giving chemotherapy with more trepidation. Everyone hears that word and you think side effects: Hair loss, nausea, fatigue, and these are certainly issues. Fortunately, there are a number of measures that can be implemented that will reduce those side effects, and that’s what we want to cover in this video. We’ll be taking the various side effects one-by-one. In some cases, there isn’t a lot you can do, and in other cases, you can make a big difference.

So let’s jump in first and talk about the side effect of fatigue. This can already be an issue in men that are on hormonal therapy and adding chemotherapy such as Taxotere can make it worse. Over and over we stress when men are on hormone therapy that they should be exercising. This same fact is true for men on any kind of chemotherapy, and it also goes for radiation as well. The men who exercise and stay strong are going to do much better and have much less fatigue. So consider that a non-negotiable reality, although men on chemotherapy may be so tired they may feel that they’re incapable of exercising. If that’s the case, then men should simply go through the motions. Move their body around even without the weights. Continue to remain active and mobile. This will help people get through the fatigue that’s caused from treatment.

When we’re talking about reducing side effects there are a number of different ideas regarding reducing the dosing of the medicine, the frequency of the medicine, and I’ll just share a few thoughts along those lines. Taxotere, the most commonly used type of chemotherapy is given every three weeks. It is possible to give a smaller dose on a weekly basis, and studies show that the side effects are reduced. Of course, that’s greater inconvenience, more doctor visits, but the weekly dosing regimen is milder, so that’s one consideration. Another possible alteration is to reduce the number of cycles. We frequently ascribe to the standard study results would suggest that six cycles is optimal for getting the maximum benefit from giving Taxotere; however, most of the side effects occur in the fourth and fifth cycle, and probably most of the benefit occurs in the first four cycles. So men that are contemplating this type of treatment but want to try and minimize side effects can consider going with only four cycles rather than six. This is a bit unorthodox and perhaps the cure rates won’t be quite as good, but it certainly will cut back on side effects.

So what other things can be done to combat fatigue? Well, many doctors use daily cortisone with prednisone in conjunction with the Taxotere. That has some benefit. It also has some potential additional side effects. Many doctors consider that sort of standard. We don’t in our practice, but men that are having excess fatigue, the addition of some prednisone five-ten milligrams a day may be helpful.

Another thing to consider is a medicine that’s approved for the treatment of narcolepsy. It’s called modafinil or Provigil. It’s sort of an upper type medicine that may help counteract fatigue in a dose from 100-200 milligrams a day.

Another idea is to consider switching Taxotere to another type of chemotherapy called Jevtana (or cabazitaxel). Studies aren’t clear on this but in my own experience and the experience of other experts, it seems like Jevtana has fewer side effects than Taxotere. So men that are running into excess fatigue or other problems with Taxotere can consider switching over to Jevtana. The medicine is given a similar three-week type protocol just like Taxotere.

So fatigue is sort of the big issue with Taxotere. In the old days, we used to be worried about nausea, which is common with all types of chemotherapy, it turns out that modern anti-nausea medicines are incredibly efficacious now. Medicines such as Zofran—an oral agent or can be given intravenously at the time of the chemotherapy administration—usually eliminate nausea altogether. There are other new agents that can be used as a backup if the Zofran is not doing the job, and so these days, although nausea used to be a very prominent issue, we don’t encounter it much anymore.

What about hair loss? Hair loss is occurring in up to 50% of people that take these agents, Taxotere or Jevtana, and the hair loss is reversible but hair loss can be very bothersome cosmetically to people and there are new ice caps that can be worn during treatment. They aren’t FDA approved yet and so it may involve a significant out-of-pocket expense. The ice caps are applied an hour or two before the chemotherapy and because of the cold blood flow is shunted away from the scalp and hair loss, therefore, is avoided.

So hair loss, fatigue, nausea and vomiting, these historically have been the big problems with any kind of chemotherapy. There’s a list of some other issues we’ll quickly and shortly address because they’re mostly correctable.

Low blood counts from chemotherapy which can place people at risk for infection can now be counteracted with an injectable medicine called Neulasta or neupogen. Our policy is to give this type of medicine in everyone who gets chemotherapy. You might want to discuss this with your doctor because some centers wait until people get a serious infection before they initiate these medications. So policies vary from office to office, but waiting around for an infection to develop seems like a rather poor plan and my recommendation would be to talk to your doctors and ask them to administer these prophylactics at the time of chemotherapy.

Chemotherapy can cause an effect on the taste buds and making food taste bad or metallic. If people keep some ice in their mouth during the infusion and for about an hour after the chemotherapy infusion, the blood, again, is shunted away from that part of the body and the incidence of problems with the taste buds is dramatically reduced.

The same thing can be said for preserving fingernails. Especially with the weekly administration of Taxotere, damage to the fingernail growth can occur. The placement of the fingers on some ice—some blue ice for example—during the Taxotere infusion and shortly thereafter shunting blood away from that area and thus the exposure of the fingernails to the Taxotere in the blood will reduce the incidence of fingernail damage and in our experience eliminate the risk altogether.

Last but not least, and this is a more common side effect with the weekly Taxotere, and that is that the Taxotere can get into the tears and then irritate the lining of the tear ducts. In serious cases the irritation becomes bad enough it can cause some scarring or closure of the tear ducts. This is a situation patients will complain and say the tears are coming out of my eyes, obviously the tears aren’t draining through the tear ducts anymore. This is a sign that you need to visit the eye doctor and they can place a small little tube in the tear ducts, open it up again, and the drainage—normal drainage—will ensue. The problem is that if you don’t do that the scarring theoretically can become permanent. So anyone on Taxotere that starts to notice increased tearing should visit the eye doctor and have their tear ducts checked out so that a stent can be placed which can be later removed after the Taxotere treatments are over.

So the use of Taxotere or its backup Jevtana is becoming more common now that studies are coming out showing that there’s a survival advantage for men in the Indigo stage who have pelvic lymph node involvement. Better cure rates, better survival all justify implementing a medicine that does have some downsides, some side effects, but if these side effects are handled wisely many of them can be bypassed or at least minimized and made tolerable so that people can get through their protocol and get on with their lives.

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

Robotic Surgery for Complex Prostate Cancer

This podcast was originally published by City of Hope Radio here.

  • Topic Info: Cancer of the prostate gland is a serious health risk for men. In fact, nearly 240,000 American men yearly will be diagnosed with it. The good news is that prostate cancer is survivable, especially if it is detected early, before it can spread.

    City of Hope is a leader in the use of robotic-assisted prostatectomy to treat prostate cancer; our experienced team have performed thousands of robotic prostatectomies since the program’s start in 2003, more than any other medical center in the Western U.S. and second in the nation.

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 Toolkit

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.