regulatory guidance
FDA Launches New Submission Program for Rare Disease Gene Therapies via CDER and CBER
CDER; CBER; rare disease; gene therapy; FDA; submission program; accelerated approval; RDEP; regulatory guidance; rare genetic disorders
Rethinking Clinical Trial Data in the Era of Decentralization: Key Trends and Challenges (2025)
decentralized clinical trials; DCT; data quality; patient diversity; remote monitoring; AI in trials; data integration; risk-based oversight; clinical trial data; regulatory guidance