The benefits of orphan drug designation in the EU are vast. If marketing authorization is granted pursuant to the EU’s centralized procedure or by all Member States, the Community and the Member States will not, for 10 years, accept another application for a marketing authorization, grant a marketing authorization or accept an application to extend an existing marketing authorization, for the same therapeutic indication with respect to a similar medicinal product.43 Regulation No. 847/2000 and recent guidance interpret the term “similar medicinal products” as those products with the same active substance or the same principal molecular structural features (but not necessarily all of the same molecular structural features), which acts via the same mechanism as the substances in the currently authorized orphan product, and which has the same therapeutic indication.44 Although market exclusivity for orphan drugs is generally granted for a period of 10 years, it may be extended to 12 years for pediatric products, or may be reduced to six years if, at the end of the fifth year of exclusivity, the drug no longer satisfies the original designation criteria (e.g., there is sufficient evidence that the product is profitable enough to not justify maintenance of product exclusivity).45 In addition, the sponsor may lose its exclusivity if it consents to a second orphan drug application or is unable to supply enough of the drug in question. In addition, a second applicant can obtain marketing approval if it can demonstrate that its drug, “although similar ... is safer, more effective or otherwise clinically superior” as compared to the initial orphan drug.46 Additional benefits of orphan drug designation include access to advice from the EMEA on what is needed to achieve marketing approval, a centralized mechanism under which sponsors may obtain marketing approval, and a reduction or waiver of fees for marketing authorization. 47
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