EMA Committee for Orphan Medicinal Products - strengthened interactions with patients and international partners

"The European Medicines Agency’s Committee for Orphan Medicinal Products (COMP) is stepping into another stage of its role in the lifecycle of orphan medicines and in the field of rare diseases, after the first 12 years of the implementation of the Orphan-Medicinal-Product Regulation in the European Union," explains Bruno Sepodes, chairman of the COMP. Integrating the views of patients, expanding international cooperation and collaborating with health-technology-assessment (HTA) bodies for a better understanding of orphan designation are some of the topics the COMP will focus on this year.

The Orphan-Medicinal-Product Regulation was introduced in the European Union (EU) to incentivise the development of medicines for rare diseases. Orphan designation can be granted at any stage of the development process to medicines that are intended for diagnosis, prevention or treatment of life-threatening or very serious conditions that affect not more than five in 10,000 people in the European Union or for which development costs would not be covered by the marketing return without incentives. If methods of treatment, prevention or diagnosis for the rare disease exist, designation is possible only where there is significant benefit brought about to patients by the medicine. Sponsors of medicines designated as orphan medicines by the European Commission benefit from a range of incentives, including market exclusivity for 10 years, fee reductions for some of the Agency’s services, protocol assistance and access to the centralised authorisation procedure.

The success of the orphan incentives is underlined by the steady increase in the number of designations for orphan designation: 107 designations were granted in 2011, 148 in 2012, and more than 150 are expected in 2013. In line with this trend, in 2012, 19 applications for marketing authorisation concerned designated orphan medicines, compared with 14 in 2011.