Marty Makary

New Capabilities

Architect of the CNPV pilot program

Patients with advanced bile duct cancer driven by a rare genetic fusion called NRG1 had nothing approved for them after chemotherapy failed. On May 8, the U.S. Food and Drug Administration (FDA) approved Bizengri (zenocutuzumab-zbco), made by Partner Therapeutics, as the first drug aimed at this small group. It works by blocking the abnormal protein the NRG1 fusion produces, which fuels tumor growth.

Updated 7 days ago

Rule Changes

Co-announcing RAPID pathway

For nearly a decade, medical device makers have faced the same bottleneck: the FDA clears a breakthrough device, and then Medicare spends another year or more deciding whether to pay for it. On April 23, 2026, the two agencies jointly announced the Regulatory Alignment for Predictable and Immediate Device (RAPID) pathway to collapse that gap to as little as two months — and simultaneously paused the existing Transitional Coverage for Emerging Technologies (TCET) program for new applicants, consolidating all breakthrough-device coverage work under the new pathway.

Updated Apr 24

Rule Changes

Announced priority review vouchers for psilocybin-class drugs and first-ever US ibogaine trial clearance

For 55 years, the federal government classified psilocybin, MDMA, LSD, and ibogaine as Schedule I substances — drugs with no accepted medical use. On April 18, 2026, President Trump signed an executive order titled 'Accelerating Medical Treatments for Serious Mental Illness,' directing the Food and Drug Administration (FDA) to expedite clinical trials of those same substances for treating post-traumatic stress disorder (PTSD) in veterans. The order, whose signing ceremony included podcaster Joe Rogan and former Navy SEAL Marcus Luttrell, was driven substantially by Health and Human Services Secretary Robert F. Kennedy Jr. It makes $50 million in federal funding through the Advanced Research Projects for Health (ARPA-H) available for state-level ibogaine research — matched by state funds — and extends the Right to Try law to allow seriously ill patients to access psychedelics still under investigation. Within hours of the signing, FDA Commissioner Marty Makary announced that the agency would issue 'national priority' review vouchers for three psilocybin-class drugs, a first for any psychedelic substance, with decisions possible as early as summer 2026. The FDA also announced steps to clear the way for the first-ever US human trials of ibogaine.

Updated Apr 19

Rule Changes

Walked back earlier claims about leucovorin's potential for autism

In September 2025, White House officials told parents of autistic children that a cheap, generic drug called leucovorin might improve their children's speech and behavior. Prescriptions surged 71% in the following months, pharmacies ran dry, and the Food and Drug Administration (FDA) allowed emergency imports from Canada and Spain. On March 10, 2026, the FDA approved leucovorin — but only for a genetic condition so rare that fewer than 50 cases have ever been identified worldwide, not for autism.

Updated Mar 10

Rule Changes

In office; co-architect of the plausible mechanism pathway

For decades, the Food and Drug Administration (FDA) required the same basic proof for every drug: show it works in a controlled trial with enough patients to be statistically meaningful. That standard made sense for common diseases but created an impossible barrier for conditions affecting a handful of people worldwide. On February 23, 2026, the FDA issued draft guidance creating a fundamentally different standard—called the "plausible mechanism" framework—that would let developers of individualized gene-editing and ribonucleic acid (RNA) therapies win full approval by demonstrating their treatment targets the root genetic cause, successfully edits or engages the target, and improves outcomes compared to the disease's documented natural course.

Updated Feb 23

Rule Changes

Serving as FDA Commissioner; leading onshoring policy efforts

Only 9% of the factories that make active pharmaceutical ingredients for American medicines are located in the United States. China and India account for roughly two-thirds of the rest. For decades, this arrangement kept drug prices low and went largely unchallenged — until the COVID-19 pandemic exposed how quickly a foreign export ban could empty American pharmacy shelves. Now the Food and Drug Administration (FDA) is quietly assembling what amounts to a three-layer incentive stack designed to reverse that dependency: the PreCheck pilot program to accelerate new factory buildouts, a priority review track for generics manufactured entirely on U.S. soil, and a proposed three-year fee waiver for new domestic plants under the next Generic Drug User Fee Amendments (GDUFA) reauthorization.

Updated Feb 20

Rule Changes

In office since March 2025

For 63 years, the Food and Drug Administration required drugmakers to prove their products worked in at least two rigorous clinical trials before Americans could take them. On February 18, 2026, Commissioner Marty Makary formally ended that standard, announcing that one trial will now be the "default position" for all new drugs—not just treatments for rare and fatal diseases, but medications for common conditions affecting millions of patients. In accompanying articles published in the New England Journal of Medicine and JAMA, Makary and Deputy Commissioner Vinay Prasad emphasized that the single-trial standard does not eliminate evidence requirements; instead, sponsors must provide "confirmative evidence" through mechanistic data, findings from related indications, animal models, real-world evidence, or data from drugs in the same class.

Updated Feb 20