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 worldâs 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 âright to tryâ laws seeking to give terminally ill patients greater access to medications that have passed Phase 1 testing for safety. Over the FDAâs objections, judges have been interceding to provide patientsâ 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, âItâs like dĂ©jĂ vu all over again.â Todayâs FDA skeptics are hardly the first to question the agencyâs role. But they are the most resistant to evidence that the FDAâs expertise is essential to clinical care. [...]
Now, in the era of personalized medicine, the FDAâs 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 agencyâs expertise is especially needed.
Given the agencyâs history, the latest attacks on the FDA cannot be called surprising. After all, as early as 1894, Dr. Harvey Wiley, the agencyâs first leader, noted: âTo 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.â5
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