Concept to Dosing: Challenges in New Orphan Drugs
Summary: With significant market opportunity available and dedicated regulatory pathways defined, the number of orphan drug programs are growing globally. As drug developers look to address unmet medical needs for orphan diseases, working with experienced partners can help in navigating key challenges such as a limited active pharmaceutical ingredient supply, efficient small-scale manufacturing for small patient populations and distribution logistics. Read this article that shares insights from industry experts on key strategies for managing an orphan drug program.