FDA Commissioner Outlines Vision for Accelerated Reviews, Flexible Trials, and AI Integration at BIO 2025
At the 2025 BIO International Convention, FDA Commissioner Dr. Marty Makary outlined his regulatory agenda, emphasizing expedited drug review processes, enhanced clinical trial flexibility, and the integration of artificial intelligence (AI). Addressing concerns over recent FDA staff reductions, Makary clarified that the cuts did not affect scientific reviewers or inspectors, but targeted administrative redundancies, accounting for approximately 10% of agency personnel.
Makary introduced the Commissioner’s National Priority Voucher (CNPV) program, a new pilot initiative designed to shorten review times for select drugs to 1–2 months. This approach separates the chemistry, manufacturing, and controls (CMC) section from final clinical trial data, allowing earlier partial submissions and leveraging clinical trial time for review preparation.
Importantly for the gene and cell therapy (GCT) sector, Makary emphasized the need for customized regulatory approaches based on disease context and therapeutic modality, especially for rare diseases. He supported the use of synthetic control arms and large real-world datasets (e.g., national electronic health records) to supplement or replace traditional randomized controlled trials when appropriate—an approach aligned with GCT developers’ calls for earlier trial entry and flexible evidence requirements.
In addition, Makary affirmed support for vaccines and clarified the FDA’s distinct regulatory role from the CDC, particularly in the context of COVID-19 booster recommendations.
He also highlighted the rollout of ELSA, an internal AI tool aimed at streamlining reviewer workflows, noting positive feedback from early users. Furthermore, the FDA plans to collaborate more closely with companies on domestic manufacturing infrastructure to improve regulatory clarity and inspection preparedness.
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