In a series of high-impact moves, the FDA is reshaping how it approves and regulates drugs, including accelerating reviews, revising vaccine standards and simplifying biosimilar development. Leadership driving these changes is also in flux: Richard Pazdur, MD, who was named director of the agency’s Center for Drug Evaluation and Research in November, has filed for retirement effective at the end of December, according to a Dec. 2 report from STAT.
Here are five key updates:
1. 15 drugs selected for FDA’s fast-track program
The agency has awarded national priority vouchers to 15 therapies under a pilot program aimed at cutting review timelines to as fast as two months. The initial batch of nine included Cytisinicline for vaping cessation, Teplizumab for Type 1 diabetes, and gene therapies for congenital deafness and pancreatic cancer. Six more were added Nov. 6, including Wegovy and Orforglipron for obesity, and Casgevy for sickle cell disease.
2. Biosimilar guidance updated to cut costs, speed approvals
On Oct. 29, the FDA eliminated routine comparative efficacy studies and eased requirements for biosimilars to gain interchangeable status. The goal is to reduce US drug costs, especially for biologics that account for 51% of spending despite making up just 5% of prescriptions.
3. New pricing rules tie U.S. costs to global benchmarks
The biosimilar guidance supports a broader strategy led by HHS to enforce “most favored nation” pricing. Pfizer and AstraZeneca have already signed compliance agreements involving $120 billion in domestic investments and significant price cuts on branded and consumer drugs.
4. Path introduced for ‘plausible mechanism’ approval
A new regulatory framework outlined Nov. 12 would allow bespoke therapies for rare diseases to be approved without traditional randomized trials. Treatments must act on a known biological mechanism and show clinical improvement in patients. Post-approval, drugmakers must collect real-world safety and efficacy data.
5. FDA official proposes vaccine approval reforms after internal review
In a Nov. 30 internal memo, FDA Vaccine Chief Vinay Prasad, MD, attributed 10 pediatric deaths to myocarditis from COVID-19 vaccines and proposed stricter approval standards for all vaccines. The claims were criticized by the Infectious Diseases Society of America as lacking transparency and risking public trust.
