Marty Makary's resignation as commissioner of the Food and Drug Administration is a positive development for American public health, provided his successor learns from his missteps and changes course. Patients across the country who depend on the FDA to deliver new treatments quickly, predictably, and safely deserve better than what they received over the past 13 months.
When Dr. Makary took office, he said many of the right things. He promised to speed up reviews for rare-disease treatments, shorten the years-long drug development timeline, and advance American innovation. He spoke for patients who feel that bureaucracy stands between them and therapies that could save their lives. His was a vision worth supporting but, as his tenure unfolded, those promises rang hollow.
Perhaps the clearest example was the FDA's treatment of uniQure, a biotech company developing an experimental gene therapy for Huntington's disease – a fatal neurodegenerative condition. The agency blocked the company from submitting a marketing application, concluding that the treatment wasn't helping patients. Yet a clinical trial had shown the therapy slowed disease progression by 75 percent after three years, a result that gave the Huntington’s community real hope.
The FDA had agreed to the developer’s study's design in late 2024. Under Makary, the agency changed course, recommending that uniQure run a new trial comparing the 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. Worse, Makary publicly called the therapy “failed” while it was still under review – a step the company's CEO rightly described as unprecedented.
Alas, UniQure was not an isolated case. Dr. Vinay Prasad, Makary's pick to lead the FDA's Center for Biologics Evaluation and Research, refused to even review a Moderna vaccine application over a dispute about clinical trial design, overriding the advice of career FDA staff. The decision was eventually reversed, but only after needless delay.
The agency rejected an experimental therapy for a rare blood cancer, a gene therapy for Hunter syndrome, and a melanoma immunotherapy from Replimune, which said the FDA had shifted the goalposts on approval requirements. A Senate hearing earlier this year documented the lack of communication and confusing guidance coming out of the agency. One witness asked why the FDA was demanding “another placebo-controlled trial that will take five to eight years,” noting bluntly that “patients on placebo will die.”
The data tell the same story. Only nine drugs were approved last year under one accelerated approval pathway, compared with 20 the year before. The forced exodus of experienced staff did not help review times either. And the loss of expertise will take years to rebuild.
This is not what “right to try,” or advancing innovation looks like. Indeed, it is a painful contradiction for an administration that ran, in part, on the promise of giving terminally ill patients faster access to experimental therapies. While the FDA continued to delay promising treatments, Makary insisted he was doing the opposite. That gap between rhetoric and reality further discredited his leadership.
To be fair, Makary did not get everything wrong. He 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.
The next commissioner has an opportunity to correct course. That means applying reasonable, consistent, and transparent standards to clinical trials, sticking to agreements the agency has already made with sponsors, and refusing to require unethical study designs. It means hiring scientific leaders who share that vision and rebuilding the career workforce. And it means remembering that regulatory unpredictability is not a neutral choice: 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. When the 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 the FDA. Patients across the country are counting on it.
Mr. Pitts is president of the Center for Medicine in the Public Interest and a former associate commissioner of the Food and Drug Administration.
