FDA Drops Two-Trial Drug Approval Rule as Staff Exodus Hits 40% in 2026

The FDA will now approve drugs based on a single clinical trial instead of two. That's the headline. Here's what the headline doesn't tell you: the agency making this promise has lost 40% of its cancer drug reviewers, burned through five department heads in one year, and just rejected at least five rare disease treatments that patients were counting on.

It's a contradiction sharp enough to cut: faster approvals, fewer people to do them, and the treatments already in the pipeline getting blocked.

The Policy That Wasn't New

FDA Commissioner Marty Makary and biologics chief Vinay Prasad published the policy shift in The New England Journal of Medicine in February. "In 2026 there are powerful alternative ways to feel assured that our products help people live longer or better than requiring manufacturers to test them yet again," they wrote.

It sounded bold. It wasn't.

In 2024, 66% of all new molecular entities approved by the FDA's Center for Drug Evaluation and Research already relied on evidence from just one clinical trial. Oncology has used single-trial approvals since the accelerated approval pathway launched in 1992.

"This is not a new paradigm," Harpreet Singh, a former FDA oncology division director, told BioSpace. Kristen Hege, a former Bristol Myers Squibb executive, was blunter: "I have never run two trials for approval."

The real change isn't what it allows. It's what it signals.

Steven Grossman, a regulatory consultant, identified the shift precisely: the FDA moved from "two studies, but we'll listen if one is enough" to "one study, unless we tell you otherwise." That's a power transfer from the agency to the companies it regulates.

The Paradox at the Centre

Here's where the story breaks. At the same moment Makary promised faster, leaner approvals, his agency was doing the opposite for the patients who need speed most.

Prasad's Center for Biologics Evaluation and Research rejected at least five cell and gene therapies that experts believe would have been approved under previous leadership. One of them was UniQure's experimental gene therapy for Huntington's disease — a condition affecting 41,000 Americans, with 200,000 more at risk.

UniQure's CEO said the company "did not reach alignment on a submission pathway." Translation: the FDA said no, despite UniQure believing its data was strong enough.

The day after the Huntington's rejection, HHS held a press call in which a senior official publicly trashed the experimental treatment. That's not standard procedure. Regulators evaluate drugs; they don't campaign against them.

The New York Times reported that patients with rare diseases "view recent decisions as signs that the doors are closing on their options." The FDA talks about flexibility. Patients experience rigidity.

Forty Percent Gone

Former FDA Commissioner Scott Gottlieb put numbers to the damage at the CNBC Cures Summit in early March. The FDA's oncology review staff — the team that evaluates cancer treatments — once had about 100 people. It's now headed below 60.

The Center for Drug Evaluation and Research had five different directors in 2025. Richard Pazdur, a longtime cancer drug regulator, retired in December because he believed political officials were corrupting the agency's scientific process. George Tidmarsh, who held the role before Pazdur, left amid allegations of using his position to pursue a vendetta against a former colleague. Prasad himself — co-author of the one-trial policy — was fired in July 2025, rehired in August, and resigned again in March 2026.

"When you lose the folks who have been doing this a long time, it starts to impact review decisions," Gottlieb said. "The people who were always engaged in that kind of engagement are now gone."

Former Republican Senator Rick Santorum, who initially supported Makary's appointment, didn't mince words: "The agency is in a bit of chaos."

The Staff Who Actually Approve Drugs

A single-trial policy sounds efficient until you think about who's reading the trial. Drug approval isn't a rubber stamp. It's hundreds of hours of reviewing patient-level data, checking statistical methods, flagging safety signals, and making judgement calls on ambiguous evidence.

With fewer reviewers, each remaining staffer handles more submissions. With more submissions based on single trials, there's less redundant data to cross-check. With less experienced personnel — the senior people who've "seen it before" — there's less institutional memory to catch the signals that only show up in the fine print.

Grossman warned that the policy "changes the balance of power between FDA and industry." When the agency was at full strength, that might have been manageable. At 60% staffing with a revolving door at the top, the balance tilts further.

What This Means for the Drug Pipeline

The one-trial default will matter most outside oncology — in neuropsychiatry, metabolic diseases, immunology. These are the areas where two trials were still the norm, where diseases affect millions instead of thousands, and where bad data can mean bad drugs reaching enormous patient populations.

A single well-designed trial, supported by real-world evidence and strong biological rationale, can be enough. That's always been true. But "can be" is different from "should be the default," especially when the reviewers tasked with distinguishing good single trials from bad ones are disappearing.

The FDA approved the first at-home brain stimulation device for major depression this month — a genuinely new kind of treatment reaching patients faster. That's the version of this story the administration wants you to see. The Huntington's patients who were turned away? That's the version they'd rather you didn't.

The Perception No One's Measuring

This story is almost entirely American. No other major regulatory agency has followed the FDA's shift. The European Medicines Agency hasn't commented. Japan, South Korea, India — all silent.

That matters because drug approvals travel. When the FDA approves a drug, other countries often follow. If the standard drops in Washington, the ripple reaches pharmacies in Lagos, São Paulo, and Jakarta.

The people who'll be most affected by a thinner FDA review process are the last people being asked about it. The CNBC Cures Summit panellists were former commissioners, senators, and pharma executives. Not the Huntington's patient whose treatment just got rejected. Not the oncologist whose review team lost 40 of its 100 members.

The FDA says one trial is enough. Its own building says otherwise.