FDA Programs Offer Advantages for Manufacturers in Rare Disease Space

FDA Programs Offer Advantages for Manufacturers in Rare Disease Space

Introduction   Rare disease drug developers face specific challenges not encountered by developers of drugs for common diseases, such as geographically dispersed and small patient populations, prolonged trial recruitment periods, and a lack of approved biomarkers, among others. Several programs at the Food and Drug Administration (FDA) incentivize the development of drugs for rare diseases and help fill the treatment gap for patients with these …