Rebuilding Confidence After Prasad’s Regulatory Debacle

In just two weeks, the U.S. Food and Drug Administration (FDA) managed to do what no slow-moving bureaucracy should ever achieve: it squandered the confidence of patients, investors, and medical innovators in one fell swoop. If only the agency moved as fast to approve life-saving therapies as it did to sabotage its own credibility.  
At the center of the FDA fiasco was Dr. Vinay Prasad, who until recently held three powerful roles—Chief Medical Officer, Chief Scientific Officer, and Director of CBER. This unusual consolidation gave him unilateral control over gene therapy decisions, with no advisory votes or visible multilayered review. Instead, the public got anonymous pronouncements with sweeping consequences. This wasn’t science—it was spin. And it wasn’t just troubling—it was bad governance.   
The chaos began with an anonymous FDA official whispering to STAT News that Sarepta Therapeutics’ new gene therapy for Duchenne muscular dystrophy – Elevidys – was essentially doomed from the start. “How do you show something is safe,” the official mused, “when it’s already proven NOT to be safe?” The effect was immediate: Sarepta’s stock plummeted 36% in a single day, vaporizing billions of dollars in value and sending Duchenne families into panic. These families, already desperate for any hope, suddenly heard that one of their only treatment options might be yanked away in a flash of doubt. 
In a plot twist worthy of satire, the very same FDA reversed course only days later after questions about the decision mounted.  The tragic death of a child that had spurred the initial alarm turned out not to be caused by Elevidys after all. The agency lifted its clinical hold on the therapy for patients who can still walk, issuing a press release to reaffirm Elevidys’ availability. In the span of a week, Elevidys went from “proven not safe” to “safe enough to administer.” One day, the FDA’s actions suggested Sarepta’s entire gene therapy platform should be shuttered; the next, all was apparently well again.  
This dizzying about-face didn’t just confuse the public – it undermined the FDA’s credibility at its core. Such contradictory messaging from the world’s most powerful health regulator is more than just bad optics; it is institutionally destabilizing.  Confidence in the FDA’s consistency and impartiality has been severely shaken. 
It’s little surprise, then, that Prasad has now been fired from the FDA in the wake of this debacle. That is a good start.  Next, if the agency wants to rebuild the trust it has lost, it should take immediate, proactive steps as outlined below: 
Initiate an Independent Investigation: The FDA should launch an Inspector General or similar independent investigation into the Elevidys incident to uncover how confidential information was leaked, who was responsible, and whether any rules were broken.  
 Reaffirm Transparent Communication: The agency needs to overhaul how it communicates critical safety actions to avoid trial-by-media scenarios.  In emergencies or emerging safety issues, timely updates are vital – but they must be accurate and consistent. Going forward, any decision to pause or withdraw a therapy should be accompanied by a clear explanation of the data and rationale, delivered via formal channels. By reducing secrecy and rumor, the FDA can start to rebuild a narrative of honesty and clarity in the eyes of patients and investors. 
 Restore Checks and Balances: Part of the Elevidys fiasco stemmed from putting too much power in Prasad’s hands. The FDA should quickly hire three separate indviduals to fill the positions Prasad controlled. At the same time, it must demonstrate that the new leadership is truly committed to the agency’s strategic plan to encourage new trial designs and novel measures of risk and benefit.  Unfortunately, Prasad promised he would do so only to twist FDA decisionmaking to fit his rationer in chief aspirations.  
Dr. Vinay Prasad’s firing closes a difficult period, but the FDA now faces the challenge of rebuilding public trust. The agency must demonstrate it has learned from recent events by committing to transparency, science, and real reform. Regaining confidence will require accountability and action to ensure the FDA fulfills its duty to safeguard public health. 

Robert Goldberg is Vice President of The Center for Medicine in the Public Interest .

 



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