Small Molecule Orphan Drugs: Status Quo, Challenges, and Perspectives
Summary: The development of orphan drugs in the past year has continued to trend upwards in the US, the European Union, and Japan. This is due to the financial incentives offered through regulation, with orphan designation appearing to be successful at generating opportunities and positive value for drug sponsors and patients. Despite that, many rare diseases remain without effective treatments despite the rising number of approved orphan drug products. In addition, the decision to invest in the orphan drug space is more complex than simply responding to regulatory incentives.
In this webinar, experts discuss the key drivers that motivate drug sponsors in developing orphan drugs. In addition, experts explore the challenges of orphan drug development and manufacturing and will discuss how the industry is leveraging strategic partnerships to help mitigate these challenges to create a viable orphan drug business model.