About 30 million people living in the European Union (EU) suffer from a rare disease. In Europe a disease is considered rare when it has prevalence of 5 in 10,000 people. Treatments of rare diseases have long represented a huge unmet medical need for patients affected by them, as well as serious challenges for clinical development. Knowing the importance of developing medicines for rare diseases, EU regulators have offered pharmaceutical companies incentives to support the development of orphan drugs: medicines for rare diseases. Incentives relate to prolonged marketing exclusivity, reduction of fees for applications to regulatory authorities, as well as free advice on protocol design and research grants for the development of orphan drugs. Despite the market niche laid out by the incentives on orphan drugs, challenges on clinical development for the pharmaceutical companies striving to develop them persist. Since 2001, 63 orphan medicines have been licensed in EU. There are lessons to be learnt from those examples that could provide the basis for future development of new approaches to diagnose, treat and prevent rare diseases.
This webcast presents an overview of EMA regulation on Orphan Drugs:
- Orphan Designation; the COMP and criteria for orphan designation
- Incentives for orphan medicines in Europe.
- Challenges of clinical development for orphan drugs.
- Studies supporting marketing authorisation of orphan medicines.