Regulatory Strategies for Orphan Drugs

The market for orphan drug products continues to expand and developing new treatments for rare diseases is becoming an increasingly important growth area for many pharmaceutical companies. This interest in rare and orphan diseases is linked not only to the incentives offered by the different regulatory authorities but also to the recent development of advanced therapies, which are often developed for the treatment of rare diseases.

The interactive course will cover all the key aspects of orphan drug legislation in both the US and EU, including a review of changes proposed in the revised EU orphan drug legislation, and the regulatory processes designed to expidite development and thereby enable earlier access to innovative medicines. The programme will explain in detail the key concepts critical for a thorough understanding of the orphan legislation in the EU and US and how it is applied. The two-stage process of first obtaining the orphan designation and then submitting the MAA/NDA and maintaining the designation as an orphan drug in order to benefit from incentives will be described. We will also cover clinical development considerations and flexibilities that are relevant for orphan drugs.

Case studies will consolidate and illustrate the learning points with real world examples and ensure that you leave the event with practical skills and knowledge that can be put to use in the workplace. The course will allow you not only to learn from our expert trainer but also to share the experiences of other delegates.