EMA approach to fee reduction for orphan medicinal products: one step forward two steps back according to EBE and EuropaBio
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Since the adoption of the Orphan regulation in 2000 the European Medicines Agency has continually provided companies with incentives to encourage the development of orphan medicinal products (OMP). These include market exclusivity, as well as fee exemptions and reductions for several other services that have been critical in bringing orphan medicinal products to the market. However, the announcement of changes in the fee structure by the EMA, with changes effective from 1 January 2013, has been of extreme disquiet to rare disease stakeholders. In a letter to the EMA, members of the Joint Task Force on OMP, jointly organised by EuropaBio and European Biopharmaceutical Enterprises (EBE), which is comprised of companies that are involved in bringing orphan medicinal products to the market, expressed deep concern on the gradual changes of the fee structure that has been taking place. The fee reduction package for non-SME’s producing OMP’s included a 75% fee reduction for protocol assistance as well as a 10% and 100% fee reduction for initial marketing-authorisation applications and pre-authorisation inspections respectively. From 1 January 2013 non-SME’s will have fewer incentives with only 40% fee reduction protocol assistance (scientific assistance) and a complete withdrawal of fee reductions for initial marketing-authorisation applications and pre-authorisation inspections for non-paediatric products. The incentives for OMP's were introduced for the explicit purpose of “encouraging the development of medicinal products for the diagnosis, prevention and treatment of rare diseases”. Without these incentives bringing the orphan products to the market is not cost-effective for these companies. In the letter the task force quotes the study conducted by the Office of Health Economics which notes that “incentives provided in the legislation greatly fostered innovation and entry into the market of therapies addressing hitherto unmet medical needs”. The letters also emphasises that “the fee waivers and fee reductions were identified by the surveyed companies as the second-most-important incentive, second only to the Market Exclusivity provisions of the Regulation”. There are several policy initiatives that are currently being encouraged by the European Union such as the setting up of International Rare Disease Research Consortium (IRDiRC) and development of the National Plans for Rare Diseases before the end of 2013, both of which will be highly influential in furthering the cause of rare disease patients. However, equally important are the incentives provided by the EMA in the form of fee reductions which make it viable for companies to develop OMP’s. The cutback of fee reductions is thwarting the efforts of the rare disease community in Europe who have fought very hard to obtain these incentives in the first place. Additionally, the task force fears that these changes by the EMA also increases the attractiveness of the United States as a base for rare disease research. Finally, members of the joint Task Force on OMP acknowledge the strict budgetary constraints that the EMA operates within and are therefore willing to work with the EMA in this regard.
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