The FDA’s approach to precision medicine changed substantially in 2025. Scientific progress continued in cell and gene therapy and the agency advanced frameworks for individualized and rare disease therapies.
At the same time, sponsors faced significant disruption. Operational challenges placed pressure on the FDA’s review capacity. Several major programs experienced unexpected reversals in evidence expectations, and long-standing incentives such as the Rare Pediatric Priority Review Voucher (PRV) program temporarily lapsed before gaining momentum for reinstatement. The result was a year characterized by shifting standards, mixed signals, and evolving interpretations of evidence.
This whitepaper explains how these scientific, regulatory, and operational forces interacted and what they mean for teams working in advanced therapeutics, rare disease, and data-driven precision medicine.
Key insights from the report include:
- How staffing reductions, leadership turnover, and the shutdown affected FDA operations
- New FDA guidance shaping development paths for advanced and genetically targeted therapies
- What late-stage reversals in programs reveal about shifting evidence expectations
- How the expiration and potential revival of the Rare Pediatric PRV program changed incentives
- Growing emphasis on lifecycle evidence and real-world data across precision medicine
Download now to learn more.