On April 7, Simeon Bennett from Bloomberg reported that individual members of the European Union are attempting to control the cost of medical care by containing the reimbursement of drugs intended for smaller patient populations, generally called orphan drugs. In Europe, orphan drugs are defined as a medicine to treat no more than 5 in 10,000 inhabitants. Many of these drugs undergo a centralized approval process via the European Medicines Agency (see related blog resource page here.) In practice, however, these drugs may only reach the market when each member state decides that its national health system will reimburse for the drug. For example, 35 orphan drugs reached the market in Belgium, 44 in the Netherlands, and 28 in Sweden in 2008. 35 such drugs reached the market in France and 23 in Italy in 2007.
According to Yann Le Cam, CEO of Eurodis, a French patient advocacy group for patients with rare diseases, "The price of orphan medicinal products is under much more debate. We have seen countries which were providing good access to orphan medicinal products now questioning the continuation of reimbursement."
Some examples provided in the Bloomberg report included the Netherlands demanding price reductions for certain therapies such as Sanofi's Myozyme® (alglucosidase alfa), an enzyme replacement therapy for patients with Pompe disease, which costs 700,000 euros ($909,000). As we previously reported from MassBio's Annual Meeting, Myozyme® was the largest research and development effort in the history of Genzyme, which was later acquired by Sanofi, and the result of a concerned father of two children with Pompe's disease pushing the promising therapy along to help it reach the public. Another example mentioned in the report included Ireland recommending against the government paying for Vertex Pharmaceuticals, Inc.'s Kalydeco® (ivacaftor) for cystic fibrosis until the company significantly reduced the price for the drug product. Yet another example was the rejection by the United Kingdom ("UK") to expand use of Alexion Pharmaceuticals, Inc.'s drug Soliris® (eculizumab) for two blood disorders, despite the recommendation for this use by an advisory panel. Instead, the government referred the matter to its National Institute for Health and Care Excellence, which we recently blogged on here, as an instrument to encourage value-based medicine in the UK.