Posted by Michael Wonder on 13 Nov 2017
Institute for Clinical and Economic Review announces final modified framework for assessing value of treatments for ultra rare diseases
13 November 2017 - Broader societal considerations and expanded cost-effectiveness ranges will be included for treatments targeting fewer than 10,000 patients.
Following a nine-month public process, the ICER today posted the final modifications to its value assessment framework for reviews of certain treatments for serious, ultra-rare diseases. The modified framework applies to therapies that, based on approved indications and planned clinical trials, will be eligible to treat no more than approximately 10,000 US patients.
These modifications are effective immediately for all relevant ICER assessments, including the ongoing reviews of new treatments for biallelic RPE65-mediated retinal disease, haemophilia A, and cystic fibrosis.
