Accessing the accelerated approval pathway for rare disease therapeutics

Emil D Kakkis, Sara Kowalcyk & Max G Bronstein, Nature Biotechnology 34, 380–383 (2016) doi:10.1038/nbt.3530

Improvements must be made to the qualification process for biomarkers as primary endpoints in pivotal clinical studies of treatments for the rarest of diseases.

 Accessing the accelerated approval pathway for rare disease therapeutics