Development of Orphan Biologics: Challenges and Opportunities скачать в хорошем качестве

Development of Orphan Biologics: Challenges and Opportunities

The development of therapies for rare diseases comes with distinct challenges, including patient recruitment, setting meaningful clinical endpoints, manufacturing and supply and finding the regulatory and market pathway to ensure patients can access therapy. For biologic products (growth factors, monoclonal antibodies, other complex proteins), development and production costs tend to be higher due to the complexity of the drug substance. To manage costs, the clinical development and manufacturing and Chemistry, Manufacturing and Controls (CMC) teams need to be aligned to ensure drug product is delivered on time to meet clinical trial needs. These challenges sit within a wider regulatory and legislative framework for orphan medicinal products (OMPs) that defines whether companies can successfully develop a novel product to market and thus the decisions made for selecting therapeutics and target diseases early in development. During this webinar, speakers covered aspects of clinical trial design for orphan biologics products, key strategies for ensuring clinical trial supply throughout clinical development and the regulatory and legislative framework for delivery to patients. Key Learning Objectives Considerations in identifying meaningful clinical endpoints for orphan diseases Orphan clinical trial design considerations and strategies to ensure sufficient patient recruitment Manufacturing and clinical supply strategies to ensure supply of clinical trial materials while collecting data needed to support NDA submission Regulatory and legislative frameworks that guide decision making for innovation in rare diseases