The OMPR has now been in place for 15 years It has resulted in o

The OMPR has now been in place for 15 years. It has resulted in orphan drug designation being granted to 1247 therapies and marketing exclusivity being

granted to 85 therapies [3]. It has benefited patients who suffer from serious, rare conditions for which there has been no satisfactory treatment. The current estimate of number of rare disorders is 6000 to 8000, many of which are of genetic origin, and affect children at a very early age. The EHC, EAHAD and the WFH believe in the importance and utility of the OMPR particularly for rare bleeding disorders such as FII, FV, FX and FXIII deficiencies, which completely lack or have very limited access to factor-specific treatment products. However, the case ALK mutation is completely different for haemophilia. The OMPR defines orphan drugs as treatments for patient populations having an incidence of less than five in 10 000 people affected by life-threatening or seriously debilitating conditions. Certainly, Ulixertinib this is the case for both haemophilia A and haemophilia B. However, the OMPR also specifically targets ‘…conditions [that] occur so infrequently

that the cost of developing and bringing to the market a medicinal product to diagnose, prevent or treat the condition would not be recovered by the expected sales of the medicinal product; the pharmaceutical industry would be unwilling to develop the medicinal product under normal market conditions’ [4]. Haemophilia A has a total of 40 plasma-derived and recombinant treatment products available worldwide (of which

22 are available in Europe) and haemophilia B HSP90 has a total of 30 plasma-derived and recombinant treatment products available worldwide (of which 13 are available in Europe). The current global market for haemophilia products is worth in excess of US $ 7 billion and the market is expected to be worth US $11 billion by 2016 [5]. Clearly, haemophilia does not require the OMPR to be profitable. On the contrary, and in an ironic twist of fate, the OMPR may in fact create a barrier to patients accessing longer acting treatment products by giving 10-year marketing exclusivity to the first product that receives marketing authorization. The OMPR automatically grants an orphan designated product 10-year marketing exclusivity once it has received marketing authorization in the EU.

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