Maladies rares et médicaments orphelins en Belgique et dans l’Union Européenne : où en est-on ?
(2015) Louvain médical — Vol. 134, n° 10, p. 527-534 (2015)
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- (en) [Rare diseases and orphan drugs in Belgium and in the european Union: what is the current situation?] Rare diseases, which affect less than 5 persons in 10,000, have become a public health priority in Belgium and the European Union (EU). Towards the end of 1999, the European Parliament and Council of EU published the regulation (EC) No 141/2000 on orphan medicinal products that are intended for the diagnosis, prevention, or treatment of rare diseases. This regulation allows pharmaceutical companies to benefit from different incentives from the EU to develop these orphan drugs, which are considered as having little or no profitability for manufacturers, given the low number of patients concerned. Since 2000, 1,586 applications to receive the orphan designation have been submitted, of which 98.5% were granted. Among the 1,563 granted designations, more than one-third were granted for rare tumors (37.4%, n=584/1563), and one-quarter for rare endocrine, nutritional, or metabolic diseases (16.1%, n=251/1563). All in all, 96 different orphan drugs have received a marketing authorisation since 2000, half of them between 2011 and October 2015, illustrating the boom in the development of orphan medicinal products. However, the high costs associated with these products pose new challenges for our health system’s sustainability. In April 2015, M. de Block, the Belgian Minister of Social Affairs and Public Health, announced collaborating with her Dutch counterpart in order to negotiate fair pricing of orphan medicinal products with the pharmaceutical companies. This pilot project, likely to start in 2016, reveals the relevance of EU countries’ active collaboration in the field of rare diseases and orphan drugs.
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Henrard, S., & Hermans, C. (2015). Maladies rares et médicaments orphelins en Belgique et dans l’Union Européenne : où en est-on ? Louvain médical, 134(10), 527-534. https://hdl.handle.net/2078.5/216192 (Original work published 2015)