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 FDA approved Bizengri (zenocutuzumab-zbco) from Partner Therapeutics as the first drug targeting this NRG1 fusion, blocking the abnormal protein it produces, which fuels tumor growth.

Updated May 31

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 — it cuts that timeline to as little as two months. They simultaneously paused the existing Transitional Coverage for Emerging Technologies (TCET) program for new applicants, consolidating all breakthrough-device coverage work under RAPID.

Updated May 31

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. On April 18, 2026, President Trump signed an executive order 'Accelerating Medical Treatments for Serious Mental Illness' directing the FDA to expedite clinical trials of those substances for PTSD in veterans.

Updated May 31

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 May 30

Rule Changes

In office; co-architect of the plausible mechanism pathway

For decades, the FDA required all drugs to prove effectiveness through controlled trials with sufficient sample sizes, a standard that made sense for common diseases but created an impossible barrier for ultra-rare conditions. On February 23, 2026, the FDA issued draft guidance for a new standard—the 'plausible mechanism' framework. Developers of gene-editing and ribonucleic acid (RNA) therapies could win full approval by showing that their treatment targets the root genetic cause, engages it, and improves outcomes compared to the disease's natural course.

Updated May 29

Rule Changes

Serving as FDA Commissioner; leading onshoring policy efforts

Only 9% of the factories making active pharmaceutical ingredients for American medicines are in the U.S.; China and India account for roughly two-thirds. 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. The FDA is assembling three programs to reverse this dependency: PreCheck to accelerate new factory buildouts, priority reviews for domestically made generics, and a proposed three-year fee waiver for new domestic plants under the next Generic Drug User Fee Amendments (GDUFA) reauthorization.

Updated May 29

Rule Changes

In office since March 2025

For 63 years, the FDA required drugmakers to prove their products worked in at least two rigorous clinical trials. On February 18, 2026, Commissioner Marty Makary formally ended that standard. Now one trial is the 'default position' for all new drugs—from rare diseases to 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 doesn't 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 May 29