Affordability problem with orphan drugs

A potential problem for HSPers



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With plans underway to implement clinical trials to test a drug treatment for SPG4 HSP, the big question is “will the drug work in people?” If it does, another question arises “can people afford the drug?”.


The cost of making many drugs contributes little to market pricing. What can make drugs prohibitively expensive is in recouping costs of research and clinical trials, a commercial profit margin, and small market size where rare diseases are concerned.


The International Rare Diseases Research Consortium (IRDiRC) recently commented:


In the World Health Organization Bulletin, an article addresses the future of funding access to orphan drugs. According to the article, European Medicines Agency recommended 89 different orphan medicinal products for marketing authorisation in Europe, by the end of 2015. However, many of these drugs are heavily priced to compensate for the research cost and lower market size.


According to the authors, while pricing and reimbursement is negotiated individually with pharmaceutical companies in each Member State in Europe, this is changing. Two European countries – Belgium and the Netherlands – have teamed up to negotiate the pricing of orphan drugs with pharmaceutical companies. A pilot project was scheduled to begin in 2016. Since the agreement was signed, several pharmaceutical companies have declared their willingness to cooperate in the pilot project. In September 2015, the Grand Duchy of Luxemburg joined the Belgium–Netherlands project and the health ministers of other EU countries have demonstrated their interest in the project.


The authors note that “this collaboration has a potential triple benefit: (i) for health system sustainability; (ii) for pharmaceutical companies; and (iii) for patients having a rare disease.”


SOURCE: 20 November 2016

European Member States collaborate to negotiate prices for orphan drugs

Posted by Anneliene Jonker

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