Obstacles & Opportunities in Orphan Biologics- From Development to Production and Supply
Summary: The pressure to get orphan drug development right on the first attempt, and so to maintain the program’s momentum, can be intense and stressful, especially when so many orphan therapies in development are lifesaving or the first of their kind. Developing therapies for rare diseases has always come with a unique set of challenges such as difficulty in assessing clinical relevance and logistical and manufacturing obstacles related to the production of treatments. For biologics, R&D and production costs also tend to be higher. This panel of experts will discuss how to overcome obstacles that span from discovery to launch of orphan drugs as well as discuss the needs of patients and drug developers in orphan biologics development.