FDA’s polarizing and high-profile biologics chief Vinay Prasad is leaving the agency for the second time since his tenure began a year ago, and this time his departure may be permanent, after yet another controversy over rare disease approvals spilled into public view and cast new doubts on Prasad and Commissioner Marty Makary’s leadership. Makary’s next pick to run the biologics center is viewed as a make-or-break decision for his commissionership.
A House Energy & Commerce Committee Democrat who is vocal on FDA issues told Inside Health Policy the next goal for the agency’s critics could be ousting the commissioner. “Prasad is Makary’s man and Makary’s failure. Makary’s seat is hot and now we need a hearing to make it hotter,” Rep. Jake Auchincloss (MA) said.
In a social media post, Makary spun the departure as a planned return to Prasad’s family and academic committees after a one-year leave of absence from the University of California San Francisco, saying he has accomplished lasting reforms at the agency. Prasad had not previously mentioned plans to spend only a year at FDA.
The latest controversy for the Center for Biologics Evaluation and Research was its decision not to approve a Huntington’s disease gene therapy from UniQure, followed by anonymous statements from FDA officials to the media that left industry fuming. But that was one in a string of rare disease decisions under Prasad that angered industry and other FDA stakeholders. He also made several decisions on vaccines that were unpopular with regulated industry and public health stakeholders, including refusing to review Moderna’s mRNA influenza vaccine. He made headlines for claiming FDA had linked COVID-19 vaccines to multiple pediatric deaths, then never publicized promised data.
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Many members of Congress have already criticized the agency under Makary’s leadership for what they see as inconsistent or inappropriate demands placed on rare disease drugmakers. Auchincloss said at an event earlier this week that Makary and Prasad should both be fired.
The dispute with UniQure centers around FDA asking the company to do a follow-up trial of its gene therapy that would require placebo surgery. FDA officials say the surgery isn’t risky or invasive; but UniQure and many Huntington’s disease patient advocates say it is. The anonymous FDA official said on the call with press UniQure’s original trial offered a “distorted or manipulated comparison,” and an HHS spokesperson told the Washington Post UniQure “lied” about what FDA wants from a second trial.
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The dispute was viewed as not boding well for Prasad’s leadership at the biologics center. He had already left FDA for a short period once, amid controversy over a different gene therapy, before rejoining the agency. Another Makary appointee, George Tidmarsh, was accused of using his position to pursue a “bizarre personal vendetta” against a regulated company and left the agency permanently.
The backlash to the UniQure situation from rare disease stakeholders also isn’t a good sign for buy-in to FDA’s plausible mechanism pathway, which Makary has touted as evidence of a flexible regulatory flexibility but appears to offer more flexibility in theory than the agency is offering in practice.
Diana Zuckerman, president of the National Center for Health Research, told IHP Prasad’s departure from CBER represents a setback for efforts to strengthen scientific rigor in the agency’s regulatory decisions. She said Prasad brought a focus on scientific evidence that many researchers and policy experts have long argued has been missing at senior levels of FDA.
Zuckerman said that across several administrations, the agency has increasingly emphasized “customer service,” with industry too often treated as the primary customer rather than patients and public health. While she acknowledged that Prasad’s decisions and outspoken criticism of the agency drew opposition from industry groups, some rare disease advocates and even FDA staff, she said independent researchers have repeatedly raised concerns that FDA has approved costly medical products that are ineffective or unsafe.
In her view, the agency’s reliance on more “flexible” approval standards for products that lack clear evidence of benefit is contributing to rising health care costs and straining federal programs such as Medicare and Medicaid. “I think Dr. Prasad leaving is a loss for the FDA, for patients, and for Medicare and Medicaid,” Zuckerman said.
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