Development of orphan biologics: challenges and opportunities

Abstract

The development of therapies for rare diseases comes with distinct challenges such as patient recruitment, difficulty in setting meaningful clinical endpoints, as well manufacturing and supply challenges. 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. During this session, speakers cover aspects of clinical trial design for orphan biologics products as well as key strategies for ensuring clinical trial supply throughout clinical development.

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.