Monthly Archives: December 2017

Cost-effectiveness of ERT in adult patients with Pompe disease


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.