EU orphan regulation--ten years of application

In April 2000, European Regulation (EC) No 141/2000 on Orphan Medicinal Products, which, following the U.S. example, had been adopted to boost the research, development, and marketing of medicinal products for rare diseases, became effective. Ten years later, figures prove that, with an average of more than 70 orphan designations per year, the European orphan regulation is a success. To date, the key issue is no longer research and development but effective market access. Less than 10% of the orphan designated products are approved for marketing and even less products are actually placed on the European national markets due to pricing and reimbursement obstacles. The article examines the European orphan regime, focusing on its two cornerstones--orphan designation and exclusivity--and highlighting the concepts that are still unclear and the issues that have not yet been addressed. The European Orphan Regulation has been proved to work well, but it would be even more successful if orphan designation was easier and orphan incentives were more attractive. The article concludes on the changes to be made to the European orphan legal regime that would encourage even more the research and development of orphan products.