Exclusive Orphan Drugs Industry Survey Results Desire to Treat Rare Diseases Tempered by a Lack of Internal Expertise and Capability
Summary: Orphan drugs face obstacles that span from discovery to launch, largely because of small patient populations and limited markets for rare disease therapies. In an August 2020 survey of nearly 600 qualified respondents from the orphan drugs space, Catalent explored the driving forces and challenges across the orphan drug development and commercialization phases. Though burdened by expertise and capability shortfalls, the innovators that rise to the challenge are often motivated to help patients who are currently without any other recourse.