Patient-Selected Controlled Clinical Trials: A Valid Proposal

Lagging patient accrual numbers for cancer clinical trials has always been an issue. In a previous post, I mentioned several reports that showed that the current system of enrollment for clinical trials just isn’t working for patients, researchers or sponsors from pharmaceutical companies.

In the current issue of the ASCO Post, Jim Omel, MD and Karl Schwartz, MFA have written a great article proposing a new type of clinical trial: A Patient-Selected Controlled Trial.

The authors explain that this approach is not meant to replace the randomized controlled trial design “when it’s feasible and ethical to use it”. They suggest that it be used as “an additional tool to consider when comparing treatments for cancer, such as (but not limited to) when the compared interventions have very different risks, or when both treatment protocols can be used off-study.”

CityofHope0897The patient-selected controlled trial would let the patient decide whether to be randomly assigned to treatment or to choose the treatment arm they want to be in. Their decision would be based on their personal situation and, most often a detailed discussion with their physician.

This type of trial would most likely result in out of balance study arms. This shortcoming could be mitigated by factors such as the following:

  • This patient-selected design would be more attractive to patients, resulting in larger studies and faster accrual.
  • Statisticians may be able to apply methods to achieve balance – such as limiting accrual in the rapidly enrolling arms
  • Propensity scoring which can anticipate and account for confounding variables in order to adjust for bias may be used in this type of trial.
  • Study doctors can provide or capture the reason for choosing one arm or the other, thereby helping to interpret outcomes and to determine whether a larger study is needed.

and others.

The authors argue their case in part by saying, “We should not let the perfect be ‘the enemy of the good’ – that is, insisting on perfection can result in no improvement at all” In other words, isn’t it better to have a good, complete patient-selected controlled trial with a bias that can be mitigated than wasting time, energy and finances on a randomized controlled trial that has to be terminated because no one shows up.

The authors conclude,

We submit that the patient-selected controlled trial is clearly superior to any randomized clinical trial that is never started because it’s judged to be unfeasible, or to any randomized controlled trial that is terminated because of poor enrollment. What good is a statistically perfect well-designed randomized controlled trial if no one signs up? We hope and expect that the patient-selected controlled trial provides another way to do good science while practicing good medicine.”

Please read the full article here and comment!