Ratio Working Papers
Do Off-Label Drug Practices Argue Against FDA Efficacy Requirements? Testing an Argument by Structured Conversations with Experts
() and Alexander Tabarrok
Abstract: The Food, Drug and Cosmetics Act of 1938 with amendments
in 1962 is inconsistent regarding FDA certification of a drug’s efficacy.
The act requires efficacy certification for the drug’s initial (“on-label”)
uses, but does not require certification before physicians may prescribe
for subsequent (“off-label”) uses. Are there good reasons for this
inconsistency? Using a sequential online survey we carried on a “virtual
conversation” with some 500 physicians. The survey asked whether efficacy
requirements should be imposed on off-label uses, and almost all physicians
said no. It asked whether the efficacy requirements for initial uses should
be dropped, and most said no. We then gently challenged respondents asking
them whether opposing efficacy requirements in one case but not the other
involved an inconsistency. In response to this challenge we received
hundreds of written commentaries. This investigation taps the specialized
knowledge of hundreds of physicians and organizes their insights into
challenges to the consistency argument. Thus, it employs a method of
structured conversations with experts to test the merit of an argument. Is
the consistency argument a case of “foolish consistency,” or does it hold
up even under scrutiny?
Keywords: Food and Drug Administration; drug approval; efficacy requirements; off-label uses; on-label uses; certification; liberalization; (follow links to similar papers)
JEL-Codes: I18; (follow links to similar papers)
45 pages, December 20, 2003
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