Volume 80, Number 4

October 2005

Facilitating Informed Medical Treatment Through Productions and Disclosure of Research into Off-Label Uses of Pharmaceuticals

Mitchell Oates

Pharmaceutical manufacturers must conduct extensive research to prove the safety and efficacy of a new drug before it can be sold to the public. However, once the Food and Drug Administration (FDA) approves a drug for one use, doctors may prescribe it to patients for any purpose for which they believe it may be beneficial. Because manufacturers are not required to prove the efficacy of a product for these “off-label” uses, research upon which physicians might base treatment decisions involving novel uses of approved drugs is likely to be lacking. In this Note, Mitchell Oates addresses two interrelated problems: a lack of research into off-label uses of pharmaceutical products and a failure, when such research is undertaken in the first place, to ensure that the findings are made public. He argues that there are limited incentives for pharmaceutical manufacturers to conduct research into the efficacy of off-label uses of their approved products. Furthermore, even when a manufacturer does conduct such research, the public benefit that results is uncertain because the manufacturer is under no obligation to publish or otherwise disseminate the data, and it is unlikely to voluntarily release research findings that might be damaging to sales. While manufacturers must submit summaries of post-approval research to the FDA, public access to these data is blocked by various legal provisions that protect against the release of trade secrets and confidential commercial information. Oates argues that the application of such provisions to data pertaining to off-label uses is inappropriate because the release of such data is unlikely to cause competitive harm to the manufacturers whose research is disclosed. In light of the problems identified and the lack of legitimate objections based on competitive harm, manufacturers should be required to conduct research into some off-label uses of their products and to disclose the data in a form useful to practitioners. Oates concludes by outlining a potential solution, modeled after an existing legislative scheme, the Best Pharmaceuticals for Children Act.