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  • Joshua M. Sharfstein

Déjà Vu at the FDA

As the Donald J. Trump administration takes over in Washington, DC, the US Food and Drug Administration (FDA) finds itself in a strange predicament. On the one hand, the agency remains the world’s gold standard for drug approval, conducting reviews more thoroughly and faster than any other regulatory agency in the world. The recent approval of a breakthrough genetic treatment for spinal muscular atrophy, for example, occurred just several years after the basic science discoveries and only about 3 months after the application was filed.

On the other hand, at the same time, a growing number of Americans seem to be questioning why we have an FDA that evaluates medications in depth at all. More than half the states have passed “right to try” laws seeking to give terminally ill patients greater access to medications that have passed Phase 1 testing for safety. Over the FDA’s objections, judges have been interceding to provide patients’ access to dubious treatment. The belief that the FDA is standing between patients and miraculous cures also appears to be gaining traction in the White House.

For the FDA, in the immortal words of Yogi Berra, “It’s like déjà vu all over again.” Today’s FDA skeptics are hardly the first to question the agency’s role. But they are the most resistant to evidence that the FDA’s expertise is essential to clinical care. [...]

Now, in the era of personalized medicine, the FDA’s old is new again. Charismatic clinicians are promising breakthroughs from stem cells and other regenerative treatments; while many are entranced by individual stories, the FDA is asking to see the data. There is an American tendency to equate scientific review with bureaucratic delay. But it is this review that distinguishes what works from what does not and that orients the pharmaceutical market toward meaningful benefits for patients instead of hype. Ironically, the growing threats to the FDA are happening at a time when the agency’s expertise is especially needed.

Given the agency’s history, the latest attacks on the FDA cannot be called surprising. After all, as early as 1894, Dr. Harvey Wiley, the agency’s first leader, noted: “To be cheated, fooled, bamboozled, cajoled, deceived, pettifogged, hypnotized, manicured, and chiropidized are privileges dear to us all. Woe be to that paternalism in government which shall attempt to deprive us of these inalienable rights.”5

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