Monthly Archives: December 2017

Cost-effectiveness of ERT in adult patients with Pompe disease

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The number of orphan drugs, i.e. drugs for rare diseases, is vastly growing. These drugs are often associated with high drug prices. Cost-effectiveness studies can be used to examine the ratio of incremental cost and effects of a new intervention. Tim Kanters and colleagues recently published a cost-effectiveness study of enzyme replacement therapy for adult patients with Pompe disease, an orphan disease, in Orphanet Journal of Rare Diseases.

Medicine