Rare disease clinical trials in the European Union: navigating regulatory and clinical challenges

Mishra, S., Venkatesh, M. Rare disease clinical trials in the European Union: navigating regulatory and clinical challenges. Orphanet J Rare Dis 19, 285 (2024). doi:10.1186/s13023-024-03146-5

Collaboration among academic institutions, pharmaceutical companies (both small and major), patient groups, and health authorities is crucial in overcoming obstacles related to clinical trials and providing assistance and creative ideas. The ultimate objective of granting rare disease patients timely and affordable access to medications with a positive balance between benefits and risks is to be met.

Rare disease clinical trials in the European Union: navigating regulatory and clinical challenges