Will the Next Commissioner Learn from Makary’s Mistakes?
Former FDA associate commissioner Peter Pitts, now president of the Center for Medicine in the Public Interest, says the recent resignation of FDA commissioner Marty Makary can be a positive development for American public health if whoever is appointed as his successor can learn from Makary’s mistakes and change the agency’s direction. “Patients across the country who depend on FDA to deliver new treatments quickly, predictably, and safely deserve better than what they received over the past 13 months,” Pitts declares in a Real Clear Health online post.
While Makary said all the right things when he took office, Pitts says, his promises to speed approvals, shorten development timelines, and advance American innovation ended up ringing hollow.
He reviews the agency’s treatment of uniQure and its experimental gene therapy for Huntington’s disease, noting that FDA blocked the company from submitting a marketing application, concluding the treatment wasn’t helping patients, despite a clinical trial showing the therapy slowed disease progression by 76% after three years, a result that gave hope to patients. He notes that FDA recommended a new trial that compared the company’s treatment, which involves brain surgery, to a sham surgical procedure.
“Asking dying patients to undergo placebo brain surgery is not a reasonable scientific request, and it’s certainly not ethical,” Pitts says. “Worse, Makary publicly called the therapy ‘failed’ while it was still under review, a step the company’s CEO rightly described as unprecedented.”
Noting that uniQure was not an isolated case, Pitts also discusses the initial refusal of then-CBER director Vinay Prasad, a Makary pick, to review a Moderna vaccine application, overriding the advice of career FDAers. The refusal was reversed, but caused needless delay, Pitts says.
After citing other examples and discussing the data on drug approvals and the loss of experienced staff, Pitts says that to be fair, Makary did not get everything wrong. He says the former commissioner provided useful public clarification on the safety and efficacy of measles vaccination and acetaminophen. “But this cannot offset the broader pattern of delay, mixed signals, and shifting standards that defined his tenure,” Pitts writes.
To correct course, the article concludes, the next commissioner must apply reasonable, consistent, and transparent standards to clinical trials, stick to agreements the agency has already made with sponsors, and refuse to require unethical study designs. The new hire also needs to hire scientific leaders who share that vision and rebuild the career workforce. And the commissioner must remember, Pitt says, that regulatory unpredictability is not a neutral choice since every delayed approval has a human cost, measured in patients who run out of time waiting and billions of dollars in abandoned clinical research.
“The American pharmaceutical sector is defined by innovation,” Pitts writes. “When FDA hobbles new and improved treatments, it diminishes the best feature of American life sciences and erodes public trust in the agency itself. The administration now has a chance to restore reliability at FDA. Patients across the country are counting on it.”