Changing the lives of patients living with rare diseases requires careful planning that incorporates regulatory, clinical, scientific, and commercial strategies. It also requires a commitment to advancing science to speed up solutions.
Gain guidance for successfully navigating complexities, specifically unique formulation and handling requirements, novel trial designs and the supply chain implications, and regulatory and clinical strategies to support product approvals.
This report provides a framework by outlining some of the processes that stand to gain the most from computational modeling and identifying the in silico capabilities that can be used to accelerate and de-risk each phase of development.
Moving the needle in the fight against rare disease requires an innovative and flexible drug development approach that considers the data limitations of orphan drug research and the inherent vulnerability of biologics.