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Dutch doctors call for EU evaluation of cost effectiveness of high cost orphan drugs

BMJ 2012; 345 doi: https://doi.org/10.1136/bmj.e5461 (Published 13 August 2012) Cite this as: BMJ 2012;345:e5461
  1. Tony Sheldon
  1. 1Utrecht

Dutch doctors are pressing for an independent European Union assessment of the cost effectiveness of extremely expensive orphan drugs such as enzyme replacement therapies for rare hereditary metabolic disorders.

The move comes after a “storm” of debate in the Netherlands over leaked government advice questioning whether the variable effectiveness of drugs for lysosomal storage disorders always justified their high cost.

Media reports claimed that the draft advice from the government’s Healthcare Insurance Board questioned whether drugs costing €200 000 (£160 000; $245 000) a year for a patient with Fabry’s disease and those costing between €400 000 and €700 000 a year for a patient with Pompe’s disease should be entirely reimbursed.

The Dutch Societies for Clinical Genetics and for Paediatrics expressed alarm at any suggestion that drugs …

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