Measuring the Value of Therapies for Ultra-Rare Diseases

The pricing of drugs is characterized by a tension between providing incentives to drug companies to invest in the development of innovative therapies and ensuring affordability so patients have access to needed medicines. The Institute for Clinical and Economic Review, an independent non-profit research institute that analyzes the evidence on the effectiveness and value of drugs and other medical services, recently issued final modifications to its framework for assessing the value of treatments for serious, ultra-rare diseases. The framework applies to therapies that are eligible to treat no more than 10,000 U.S. patients including certain treatments for inherited eye disease, hemophilia A, and cystic fibrosis. We spoke to Steven Pearson, president of ICER, about the framework, why modifications were needed for drugs to treat ultra-rare diseases, and what the implications are for a pipeline or therapies with the potential not just to treat, but to cure progressive and deadly rare diseases.