The Institute for Clinical and Economic Review (ICER) said the prices of two of three cystic fibrosis drugs from Vertex Pharmaceuticals Inc. would need to be discounted by about half in order for the drugs to be cost-effective. Despite the higher threshold for orphan and ultra-orphan drugs, the committee deemed the incremental cost-effectiveness ratios of about $1 million per QALY vs standard of care to be too high to be approved without a significant discount.
European authorities have struggled with the cost-effectiveness of Vertex’s drug Orkambi’s. French authorities requested an 80% discount from the price offered by Vertex; in response, Vertex withdrew plans to launch Phase III studies of its next-generation CFTR modulators in France (see BioCentury, Feb. 23).
In 2016, the U.K.’s NICE recommended against Orkambi’s approval based on high cost-effectiveness estimates. Today (19 March 2018), the Parliament’s House of Commons will debate a petition to make the drug available on the NHS (see BioCentury Extra, March 5).