The report by the European Commission takes stock of the incentives received by sponsors towards developing orphan medicinal products. The report provides the statistics that possibly endorse the success of these incentives. According to the report, between 2000 and September 2015, the European Medicines Agency (EMA) received 2302 applications for orphan designation, of which the Commission approved 1544 and 1227 are currently active. In the first 15 years of application of the Regulation, 951 protocol assistance procedures were completed, of which 264 involved small and medium enterprises. The EMA receives a special annual contribution from the EU budget to waive fees in part or in full, which till date amounts to €78.4 million.
For EU-funded research on rare diseases and OMPs, Orphan designation has been a requirement for the Framework Program funding since 2009. The report details that there was more than a 50 % increase in both the number of OMP applications submitted and the number of designations granted by Commission during 2009-2015, in comparison with 2000-2008. More than €620 million in funding was awarded by FP7 to over 120 research projects on rare diseases and OMPs. Horizon 2020 maintains strong commitment to fund research on rare diseases and OMPs. Additionally, the Commission launched the International Rare Diseases Research Consortium (IRDiRC) under FP7, in cooperation with its EU and international partners.
The report details the measures taken by individual member states towards encouraging OMP development. In the Netherlands, registration fees can be waived if the OMP is already registered in another Member State and the prevalence of the condition is less than 1:150,000. Orphan medication developers in France are exempt from certain taxes paid by pharmaceutical companies and allow claiming a high price with certain conditions. Last year, Belgium and the Netherlands launched a pilot project on the joint negotiation of OMPs. Spain and Sweden announced specific scientific advice procedures to benefit developers of OMPs. The Committee for Orphan Medicinal Products at the EMA, has established an international liaison in North America and Japan and also holds a monthly teleconference with the U.S Food and Drug Administration.
Finally the report lists all the products that received marketing authorizations as OMPs over the past 15 years, and its distribution per therapeutic area.