Pharmacovigilance in Rare and Orphan Drugs
Rare and orphan drug development presents unique pharmacovigilance challenges due to limited patient populations, heterogeneous data, and accelerated approval pathways. This session examines strategies to ensure effective safety monitoring in these contexts. Topics include leveraging patient registries, using surrogate endpoints, and applying innovative methodologies to detect and assess adverse events. Collaborative approaches involving regulators, patient advocacy groups, and academia are emphasized to enhance data quality and interpretation. The role of real-world evidence and adaptive safety surveillance plans will be discussed. Attendees will learn how tailored pharmacovigilance frameworks can ensure the safe and ethical use of therapies for rare diseases while addressing unmet medical needs.