Should You Participate in a Clinical Trial?

Are clinical trial participants guinea pigs?

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Among the most challenging decisions faced by cancer patients and their loved ones is whether to participate in a clinical trial.  Unfortunately, the opportunity to become a clinical trial “subject” is often presented when few or no other promising alternatives are available, adding a sense of desperation to the decision for many.

It is important to appreciate that in the United States, a clinical trial cannot administer you only the new drug or treatment being evaluated in place of an approved therapy with known benefit.

  For example, if there is a chemotherapy agent that has proven safe and effective for your type and stage of cancer, every trial subject will receive it, but a subset of subjects (known as a trial “arm”) will also receive the experimental therapy.

Also, agreeing to participate (“enrollment”) does not negate your right and freedom to at any time choose to no longer participate.  And should you choose to no longer participate, no hospital, physician, or other medical provider can deny you any standard cancer treatment moving forward (in other words, there can be no retribution).  I recommend that you first discuss your reasons openly and honestly with your physician, as some issues can be addressed without leaving the trial.  But remember, it is your decision, regardless of whether others understand or agree with it, and such a decision is not allowed to impact your care options moving forward.

  (It is, however, important to recognize that once you have stepped out of a clinical trial, you will not likely be allowed to re-enter later.)

The most important thing when considering participation is the clinical trial phase.  Clinical trials are routinely divided into four phases, each building on the outcome of the earlier phase.

  And the earliest phases offer the least likelihood of directly benefiting the participants.

Phase I trials (routinely the earliest phase studies) usually enroll fewer than 100 subjects and are not focused on the effectiveness of the new treatment in slowing or curing the targeted cancer, but on the safety of different treatment levels or dosages.  Identification of safe treatment levels or doses supports advancement to a Phase II trial.  Given this, you must recognize that there is very little likelihood that enrolling as a Phase I trial subject will benefit you personally in your battle against your cancer.  So why do people participate in Phase I trials?  Because some people are amazingly selfless, willing to participate in research which, while not benefiting themselves, may benefit others battling the same cancer in the future.

Phase II studies evaluate a larger but still small group (often a few hundred) at a selected treatment level proven safe by the selfless Phase I participants.

  Phase II trials seek to demonstrate some effectiveness (as well as further safety) of the novel therapy in treating the targeted cancer.  Thus, participation in a Phase II trial may offer some benefit to you should you participate as well as potentially benefiting similar cancer patients in the future.

If Phase II effectiveness and additional safety is demonstrated, a subsequent Phase III study enrolls thousands of subjects at numerous sites across the country or even across the globe.  Phase III studies aim to demonstrate statistically meaningful effectiveness and safety of the new therapy and are used in seeking U.S. FDA approval to market the treatment with claims (advertising) of effectiveness and safety.  Phase III trials offer you the greatest likelihood of personal benefit, as the new treatment has already demonstrated safety (in both Phase I and II) and at least potential effectiveness (in Phase II) against your cancer.  Given that it may be many months or even years following a Phase III before the new treatment is commercially available, enrollment in a Phase III trial may be the only way that an advanced cancer patient will have access to a new therapy.

Finally, the FDA sometimes requires a Phase IV (also called a “Post Market”) trial to evaluate safety over a much longer period.  However, unlike all previous phases, refusal to participate in a Phase IV trial does not prevent you from receiving the new treatment, as it has been approved for use by the FDA.

In the end, whether or not to participate in a clinical trial depends on where your hopes for both yourself and for future cancer patients fit in relation to the offered clinical trial phase and potential benefits and risks.  Thus the decision is deeply personal, and when decided by you and your loved ones, has no wrong answer.

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