FAQ: FDA’s Shifts in Pharma Regulation and Strategy in 2025

1. What major leadership changes have affected FDA's drug and biologics centers?

George Tidmarsh, MD, PhD, was appointed as the new director of the Center for Drug Evaluation and Research (CDER) in July 2025, succeeding Patrizia Cavazzoni, who retired in January 2025 (1,2). After a brief departure, Vinay Prasad, MD, MPH, resumed leadership of the Center for Biologics Evaluation and Research (CBER) in August 2025, having been initially appointed in May 2025 (3–5).

2. What new mechanisms are available for expediting drug approval review times?

FDA launched the Commissioner’s National Priority Voucher (CNPV) pilot program in June 2025, offering an opportunity to shorten final drug application review times from the traditional 10–12 months to just one to two months (6,7). This program uses a common-sense, team-based evaluation often described as a "tumor board style" discussion to deliver timely decisions (6,8).

3. What specific criteria must a drug application meet to qualify for the CNPV pilot?

To qualify for the CNPV program, applicants must address a health crisis in the United States, deliver innovative cures, address unmet public health needs, and increase domestic drug manufacturing as a national security issue (6,9). USAntibiotics received the first approval through the CNPV pilot program for an amoxicillin-clavulanate potassium antibiotic in just two months’ time (10).

4. How is FDA working to enhance transparency in the drug review process for sponsors and the public?

FDA is implementing a policy to publish Complete Response Letters (CRLs) promptly after they are issued to sponsors, providing early insight into regulatory deficiencies (11). Previously, FDA released an initial batch of more than 200 previously confidential CRLs issued between 2020 and 2024 for applications that were not approved during their initial review cycle (12).

5. Is FDA still relying on external expert advisory committees for critical drug reviews?

FDA is reportedly considering reducing its reliance on external expert advisory committees for new drug applications, based on the rationale from CDER head George Tidmarsh that these panels are redundant and time-consuming (13). Critics argue that abandoning these expert reviews could reduce public scrutiny and transparency in the drug approval processes, especially since advisory committees offer public input before a decision is made, unlike disclosed CRLs (13).

6. How is FDA integrating artificial intelligence (AI) into its own operational workflows?

The agency is deploying agentic AI capabilities for staff to create complex AI workflows and assist with multi-step tasks, including pre-market reviews and compliance (14). FDA also launched Elsa, a generative AI tool, which is used to accelerate clinical protocol reviews and scientific evaluations by securely accessing internal agency documents (14).

7. Are there defined pathways for qualifying innovative drug development tools, like AI algorithms or wearables?

Yes, FDA’s Innovative Science and Technology Approaches for New Drugs (ISTAND) pilot program is transitioning to a permanent drug development tool (DDT) qualification program, creating a clear pathway for tools that do not fit into existing agency qualification parameters (15). Qualified DDTs include AI-based algorithms, digital health technologies like fitness trackers, and animal-free assays, all intended to accelerate drug discovery and development (15).

8. How is FDA addressing the risk of relying too heavily on foreign drug manufacturing?

FDA introduced the PreCheck program, which features a Facility Readiness Phase designed to increase regulatory predictability and streamline the review of domestic manufacturing facilities, helping to reverse the reliance on foreign drug manufacturing (16). To receive the CNPV, a program that speeds review time, companies must demonstrate that they are increasing domestic drug manufacturing as a national security issue (6,16).

9. How is FDA adjusting regulatory standards for novel cell and gene therapies?

FDA issued new draft guidance establishing the Regenerative Medicine Advanced Therapy designation, which provides expedited development programs for regenerative therapies intended to treat serious conditions. This guidance encourages flexibility in clinical development, allowing the use of adaptive designs and real-world evidence, while still maintaining stringent chemistry, manufacturing, and controls standards that necessitate early and frequent interaction with CBER (17).

References

1. FDA. Stanford Faculty Member George Tidmarsh, MD, PhD Named Director of Center for Drug Evaluation and Research. Press Release. July 21, 2025.
2. FDA. Patrizia Cavazzoni, MD, Director–Center for Drug Evaluation and Research. FDA.gov, last updated Jan. 18, 2025 (accessed Dec. 15, 2025).
3. Armstrong, D. Vinay Prasad Is Out at FDA, Following Sarepta Decision and Vaccine Controversies. EndpointNews.com, July 29, 2025.
4. Bayer, M. Prasad Returns to FDA, Resuming Head of CBER Role. EndpointsNews.com, August 9, 2025.
5. Makary, M. Post on X. May 6, 2025. https://x.com/DrMakaryFDA/status/1919802781466079264 (accessed May 7, 2025).
6. FDA. FDA to Issue New Commissioner’s National Priority Vouchers to Companies Supporting US National Interests. Press Release. June 17, 2025.
7. Cole, C. Industry Expert Panel Weighs CNPV Benefits, Risks, and Readiness: Part I. PharmTech.com, Nov. 21, 2025.
8. Cole, C. Ready or Not: Biopharma Alignment for CNPV’s Rapid Review. PharmTech.com, Nov. 20, 2025.
9. Cole, C. The Regulatory Crucible: Risk, Resource Drain, and the Hidden Trade-Offs of the CNPV Pilot Program. PharmTech.com, Nov. 19, 2025.
10. FDA. First Approval in Commissioner's National Priority Voucher Pilot Program Strengthens Domestic Antibiotic Manufacturing Capacity. Press Release. Last updated Dec. 10, 2025.
11. FDA. FDA Announces Real-Time Release of Complete Response Letters, Posts Previously Unpublished Batch of 89. Press Release. Sept. 4, 2025.
12. FDA. FDA Embraces Radical Transparency by Publishing Complete Response Letters. Press Release. July 10, 2025.
13. Allen, A. Under Trump, FDA Seeks to Abandon Expert Reviews of New Drugs. KFFHealthNews.org, Sept. 12, 2025.
14. FDA. FDA Expands Artificial Intelligence Capabilities with Agentic AI Deployment. Press Release. Dec. 1, 2025.
15. Buckman-Garner, S. and Thanh Hai, M. FDA Advances Drug Development Innovation by Establishing ISTAND as Permanent Qualification Program. FDA Voices. FDA.gov, July 31, 2025 (accessed Aug. 4, 2025).
16. FDA. FDA Announces New FDA PreCheck Program to Boost US Drug Manufacturing. Press Release. Aug. 7, 2025.
17. FDA, Draft Guidance for Industry, Expedited Programs for Regenerative Medicine Therapies for Serious Conditions (CBER/OTP, September 2025).