Increasing Pressure on Orphan Medicinal Product Designations in the EU

In the EU, a sponsor can obtain an “orphan designation” for a medicinal product pursuant to Regulation 141/2000. Adopted in December 1999, the regime aims to encourage investment in R&D for treatments for rare diseases. The most important incentive is the 10-year market exclusivity for designated products. Other incentives include protocol assistance and fee reductions. At the national level, there may be benefits under pricing and reimbursement procedures and, for instance, also tax incentives.