FDA Expedited Drug Approval Pathways

By February 4, 2019 Commentary

Researchers commissioned a survey of physicians to ascertain attitudes about two aspects of FDA drug approval and marketing processes that some find controversial:  the increasing use of expedited approval processes, which means that the agency is making decisions on less data and relying more on post-approval information gathering; and the limits courts have imposed on the agency’s ability to regulate direct-to-consumer advertising, especially in regard to off-label indications.  They published the results in a research letter in JAMA Internal Medicine.   (JAMA Article)   The respondents were 686 endocrinologists, cardiologists and internists.  80% agreed that the FDA’s approval process was effective in protecting the public against dangerous medications.  78% felt that therapies should be tested in at least two prospective randomized clinical trials, while 21% felt one such trial was adequate and only a few would accept other kinds of studies.  Since about one-third of recent new drug approvals have relied on a single trial, physicians approval of FDA processes and confidence in its decisions is somewhat at odds with their preference for better evidence.  But physicians are probably unaware of the evidence behind specific approved drugs.  Manufacturers and the FDA should probably be required to highlight the exact nature of the evidence used for an approval.  The FDA is torn between trying to get potentially beneficial therapies to market quickly, especially for diseases with no or few current effective treatments, and ensuring that these medications are in fact safe and effective.

60% believed that the FDA should not allow off-label promotion of medications to physicians, with 70% believing that would be a bad idea if it occurred in physicians offices and 68% if in medical journals.  60% that such activities might increase prescribing without any real benefit to patients, and were particularly concerned about prescribing for issues not previously considered to be medical problems.  Many more respondents believed off-label promotion would worsen than better clinical decision-making.  My opinion for a long-time has been that the Supreme Court erred when it said that corporate entities have first-amendment rights; it is that decision that inhibits FDA regulation of drug manufacturer marketing activities.  I think that all direct-to-consumer advertising should be banned.   It serves no value.  And direct-to-physician marketing should be strictly controlled, with only very clear factual information permitted and no promotion of off-label uses.  Researchers who wish to study off-label uses should be required to follow the usual FDA requirements and those uses should not be permitted in general clinical practice unless and until the FDA approves them.  All of this would be one more way to help control spending, as well as protecting patients.

Kevin Roche

Author Kevin Roche

The Healthy Skeptic is a website about the health care system, and is written by Kevin Roche, who has many years of experience working in the health industry through Roche Consulting, LLC. Mr. Roche is available to assist health care companies through consulting arrangements and may be reached at khroche@healthy-skeptic.com.

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