Dr. Mitchell Seymour, October 24th, 2013

Dr. Mitchell SeymourWhen current medical options are limited or insufficient, doctors often leverage their knowledge and professional experiences to think and act outside the box. Off-label use is a critical tool in this innovative clinical care. 

A recent article in the Washington Post provokes interesting considerations regarding drug repurposing. doctor prescription The article suggests that off-label prescribing is inherently suspect or dangerous, and further supports this through commentary from prominent bioethicist Alexander Capron.  Capron believes that when physician finds a helpful off-label use for a drug, he/she is ethically obliged to publish a research article on the findings.

In response, author Ford Vox wrote a critical commentary on the Washington Post article. When questioned by Vox, Capron proposed a threshold for when clinical care ends and the practice of unethical, non-institutional review board approved research begins. He stated that once a physician finds a new off-label use helpful in more than two patients, he/she is engaging in unethical conduct by treating further patients without establishing a research study.

As a researcher in an academic medical center and a regulatory professional, I strongly disagree with Capron. First, a critical element of clinical research is randomization to treatment(s) and control arms(s). A doctor engaged in innovative clinical care may be unwilling to assign patients to a placebo group for the sake of scholarly activity; he/she may find this unethical.  Second, ethical medical practice should always include a frank discussion of drug risks and benefits, including the disclosure that the drug would be prescribed off-label. Capron implied that patients are uninformed about such risks by their doctors, which is an assumption and cannot be broadly applied. Third, the Caprons’ views suggest that informed consent in clinical trials adequately educates study participants of risks and benefits, which is not uniformly true. Informed consent is a process, not just a document, and that process can be flawed despite the clinicians’ best intentions.

My perspective is that the realm of innovative clinical care is much more complex than Capron asserts. While clinical trials and scholarly publishing of off-label experiences are certainly ideal, the success of repurposing is already communicated within the medical community in many ways, including professional meetings and word-of-mouth. By Caprons’ guidelines and constraints, innovative medicine with off-label uses could become an impractical and limited endeavor. Repurposing is valuable for both research and clinical care, and I do not feel that one approach is ethically superior to the other, but are instead are two sides of the same valuable coin of medical innovation.

Read all Regulatory Roundup posts by Dr. Mitchell Seymour


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