Nothing destroys confidence in a regulatory agency faster than the perception that it has lost sight of its mission. The Food and Drug Administration exists not merely to keep unsafe drugs off the market, but also to ensure that lifesaving treatments reach patients before it is too late. President Trump’s “Right to Try” initiative reflected a recognition that when Americans are confronting terminal or degenerative illnesses, regulators should lean toward speed, flexibility, and informed patient choice rather than procedural paralysis.
However, it’s not clear that Trump’s appointees at the FDA are even aware of this initiative.
Donald Trump entered the White House in 2017 with an agenda to upset the status quo at the FDA. Hamstrung by an over-cautious bureaucracy with an agenda that seemed to center on self-protection, it kept its foot on the brakes of innovation for decades, leaving politicians of both parties--as well as people suffering from illnesses without any effective treatment--frustrated and more than a little angry.
In his first term the FDA moved with alacrity to get drugs through the evaluation phase and focused on giving people with terminal diseases access to cutting-edge treatments in a timely manner.
However, the current FDA bears scant resemblance to Trump’s first term iteration, as the urgency to get drugs approved has completely dissipated. The White House signaled Friday that it is preparing to replace FDA Commissioner Marty Makary, which was greeted with a chorus of hallelujahs from patients, doctors, and the biotechnology sector alike.
While Trump downplayed the reports over the weekend, the fact that the administration is reconsidering Makary’s leadership suggests that the White House recognizes an increasingly obvious reality: the FDA has drifted back toward the same culture of bureaucratic caution and anti-innovation dogma that Republicans spent years criticizing during the Obama and Biden administrations.
While Makary entered office with considerable goodwill, his tenure became defined by internal turmoil, high-profile drug rejections, and growing skepticism toward accelerated approvals. Makary’s biggest mistake was his decision to empower Vinay Prasad, whose skepticism toward expedited approvals quickly alarmed industry and patient advocates. Under Prasad’s watch, the FDA repeatedly moved goalposts for companies developing therapies for devastating illnesses--including Huntington’s disease, Duchenne muscular dystrophy, Hunter syndrome, and advanced melanoma--with no rationale other than the gains in quality of life or longevity weren’t enough to make it worthwhile. Several therapies that had previously received favorable feedback from agency scientists had senior political appointees intervene to block or delay approvals.
The damage from this approach extends far beyond Trump losing a modicum of political support: When firms spend billions of dollars and years of research to develop novel therapies, only to belatedly discover that FDA leadership has arbitrarily rewritten evidentiary standards, investors will rationally move capital elsewhere. Smaller biotech firms will go out of business and the large pharmaceutical companies will reduce spending in research and development.
Nearly every major breakthrough in oncology, gene therapy, and rare-disease treatment over the past two decades has been produced in the U.S., owing to its relatively flexible approval system. Undermining that ecosystem in pursuit of ever-more stringent evidentiary standards comes with real human costs. And we are seeing China catch up and outpace us in gene therapy treatments.
The FDA’s recent rejection of Replimune’s melanoma therapy RP1 is a case in point. Career FDA reviewers initially supported its approval before senior leadership overruled them. Makary insisted that the agency simply wanted stronger evidence of efficacy, but the stance ignored the practical reality facing patients with late-stage cancers and rare diseases: For this cohort, traditional double-blind randomized trials are either ethically dubious or statistically impossible.
Makary also staffed the agency with officials connected to Arnold Ventures, a billionaire-backed nonprofit whose health-policy network has long argued that the FDA approves too many drugs too quickly. Its preferred model resembles the more restrictive regulatory frameworks common in Europe, where cost containment and procedural caution often take precedence over rapid patient access to therapies.
Even congressional Republicans who broadly support the administration’s healthcare agenda have begun warning publicly about the FDA’s direction. At a House Ways and Means hearing last month with HHS Secretary Robert F. Kennedy Jr., Rep. Darin LaHood (R.-Ill.) questioned whether “bungled drug reviews at the FDA have chilled investments in life-saving innovative cures” while strengthening foreign competitors such as China.
Replacing Makary would remove an unnecessary liability heading into the midterms. The administration gains nothing from headlines portraying the FDA as hostile to medical innovation while simultaneously consumed by layoffs, internal dysfunction, and leadership infighting.
The broader market implications are significant as well. Drug development is one of the few sectors where the United States still enjoys overwhelming global dominance. Biotech supports hundreds of thousands of high-paying jobs and drives enormous amounts of venture capital investment. Every signal that the FDA is becoming more hostile to innovation weakens that competitive advantage.
Trump built his political brand attacking institutions that prioritized bureaucracy over results. Increasingly, the FDA under Marty Makary has come to symbolize exactly that problem. If the White House wants to restore confidence in the agency, a leadership change would be a logical place to begin.
