Makary Outlines Sweeping Reforms to Speed Drug Development

FDA commissioner Marty Makary is signaling a broad push to overhaul how the agency reviews new drugs, aiming to reduce early-stage regulatory friction, accelerate clinical trials, and expand the use of novel programs designed to shorten approval timelines.

In remarks at a 5/4 Milken Institute conference detailing his agenda, Makary acknowledged persistent industry concerns that unclear regulatory expectations — particularly in investigational new drug (IND) applications and Phase 1 trials — led companies to over-submit data, slowing development in the U.S. “Companies are doing guesswork,” he said, describing a system where sponsors often provide more information than necessary out of caution. The result, he argued, is delayed early-stage trials and a growing shift of first-in-human studies to countries such as China, the U.K., and Australia.

Makary said FDA is preparing a “giant set of reforms” to streamline IND requirements and the Phase 1 process, with a focus on clarifying what data are, and are not, needed. The changes will include deferring certain requirements and narrowing early review to core safety considerations.

The effort comes amid intensifying global competition for clinical research. Makary noted that China now initiates significantly more early-stage trials than the U.S., a reversal from prior years. “We should be the leader in biotech in the world,” he said, adding that reforms are intended to restore U.S. competitiveness and reduce administrative bottlenecks such as institutional review board delays and contract negotiations.

Makary also defended the agency’s move toward greater transparency, including making complete response letters (CRLs) public, amid criticism that regulatory decisions can appear inconsistent. He cited an internal FDA analysis showing that roughly 80% of drug rejections were previously “spun” by companies in public disclosures, often minimizing deficiencies identified by regulators.

Under his leadership, Makary said approval and rejection decisions strictly reflect the conclusions of FDA review teams. “If your drug works, it’s going to get approved,” he said, provided safety and manufacturing standards are met.

A centerpiece of Makary’s agenda is the national priority review voucher program aimed at cutting “idle time” in the review process. The initiative targets high-impact therapies and has already produced markedly shorter review timelines, he said.

According to Makary, the FDA has granted 21 vouchers and completed several reviews in a matter of weeks rather than the typical 10–12 months. Some approvals have occurred in as little as 42–55 days. The program focuses on therapies addressing serious conditions, including cancer and rare diseases, and relies on close coordination between FDA staff and sponsors. Makary said review teams retain discretion to extend timelines if needed but are meeting aggressive targets.

Selection of voucher candidates is decentralized, with FDA’s more than two dozen review divisions nominating products they view as high priority.

Makary also highlighted a newly launched “real-time” clinical trial initiative, including two pilot programs in collaboration with Amgen and AstraZeneca (see earlier story). The model allows FDA reviewers to monitor trial endpoints in real time via cloud-based systems, rather than waiting for trial completion and formal submission. The approach is intended to reduce what Makary described as “dead time,” which can account for roughly 45% of the drug development timeline.

The agency ultimately aims to enable continuous clinical development with minimal gaps between trial phases, while also improving transparency around negative trial results, which are often not publicly reported.

Beyond clinical trials, Makary outlined several additional priorities:

• Expanding over-the-counter drug availability where safety permits
• Increasing use of artificial intelligence to streamline administrative review steps, including reducing a 60-day application completeness check to minutes
• Advancing women’s health initiatives, including research on hormone replacement therapy
• Continuing efforts to accelerate treatments for conditions such as cancer, Alzheimer’s disease, ALS, and PTSD

Makary framed the reforms as part of a broader effort to compress drug development timelines from a decade or more to “a couple years,” without compromising safety.

“We want more cures and meaningful treatments,” he said.