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For What Reasons Would You as a Patient Enter a Randomized Clinical Drug Trial?

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Pharmaceutical companies as well as other research laboratories are regularly developing new drugs for treatment of symptoms and diseases. At some later stage of the drug development it is necessary to determine in human subjects whether the new drug is effective for treatment. This requires a study called a clinical drug trial and will require patients who have the symptoms or disease to be the subjects of the trial. The goal of the trial is to determine if the new drug has some beneficial effect over no drug and if so is the new drug equal to or superior to an established drug for the condition. Patients are divided into groups in which one group will get the new drug treatment and one group will get the established drug treatment, if one is available, and the third group will get a placebo ("sugar pill"), which may represent no drug treatment. In order to provide a scientifically significant answer, it is necessary to randomize the trial so that no patient and no investigator who is doing the study is aware of who got what. This is called a "double blind randomized controlled study". A scientist who is not involved making a conclusion of the results is aware of the randomization and can stop the study for a patient who is having a reaction or for a group that may be harmed.

The details of the scientific and therapeutic value of the study, potential benefits and risks should be made available to the subject as part of what is called informed consent. The ethical use of a placebo is controversial but is considered by some ethical if the risk for harm is small and potential harm to the patient by no treatment is mild.

What would be of interest is whether you as a patient with a condition, which is being studied, would volunteer for a study where you would not know whether you got a new or old medicine or no medicine. And if you would enter the study, for what reasons would you do so?

(Moderator's note: For more on the topic of the ethics of placebo-controlled drug trials, you may wish to read the discussion in The American Journal of Bioethics Vol. 2 No. 2 (2002).)

Here is the question:

For What Reasons Would You as a Patient Enter a Randomized Clinical Drug Trial?

As a physician I would enter a randomized clinical trial if I thought that the drug or procedure might benefit me. I would accept the risk of receiving placebo or no treatment since the benefit of if the new drug or treatment is not yet established, It is not immoral to use clinical trials if the benefit of a new treatment is not established because there iw no more effective way to establish the effectiveness or lack thereof of an new drug or treatment than a randomized double blinded trial, We cannot simply assume that any new drug or treatment is effective or that because a group of patients does better than previous patients it is the effect of a new treatment; there are simply too many confounding variables, If a treatment is sown to be superior then further randomized treatments are no longer necessary or ethical.

As a patient, I would enter a randomized clinical trial only to receive a new drug or treatment that is not otherwise available. But I would always ask whether my physician could give me the experimental treatment outside of a randomized trial so that I would be assured of receiving the desired treatment.

As a physician, if I am able to provide an experimental treatment outside of a randomized trial, I would feel obligated to provide it at the patient's request.

If every patient refused to be randomized then there would be no randomized trials and that would be fine with me.

In my opinion, randomization of patients with life-threatening diseases is clearly immoral. Randomization of patients with non-life-threatening diseases is also immoral, albeit lesser so. No one enters a clinical trial to receive a placebo, and most, if not all, patients would be more comfortable knowing what treatment they are receiving than not knowing. If I were a patient from whom a potentially effective treatment was witheld (even if temporarily), I would find no solace and much resentment in knowing that my suffering served only to increase the statistical validity of a study by a fraction of a percentage point.

I doubt whether randomization, in iteslf, contributes enough to the statistical validity of a study to justify its use. Where are the randomized trials comparing randomized and non-randomized trials? If randomized trials were honestly designed to address issues in dispute, shouldn't there be enough patients who choose of their own free will to either receive or not receive the experimental treatment over the conventional treatment?

It seems to me that most large clinical trials are not designed to provide answers to controversial issues of treatment, but to unequivocably demonstrate the efficacy of "experimental" treatments that are on the verge of becoming the standard treatment. Large randomized clinical trials are so expensive and labor-intensive that they are rarely undertaken without a reasonable likelihood of demonstrating the desired outcome, which more often than not, can be predicted by previous non-randomized trials if not by common sense alone. Therefore, at this late stage of large clinical trials, randomization is often employed purely for its own sake - that is, to imprint the "seal of randomization" on a study to increase its legitimacy in the eyes of the medical community.

I support the increased introduction of scientific integrity to clincal research, but I believe the practice of randomization goes too far by dehumanizing patients and taking away their right to choose their treatment.