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最近发表了一项成本效益研究of enzyme replacement therapy for adult patients with Pompe disease, an orphan disease, inOrphanet Journal of Rare Diseases.