Once again, Vertex Pharmaceuticals and a U.K. government agency are at odds over the value of life-saving cystic fibrosis medicines.
In draft guidance published late last week, the National Institute for Health and Care Excellence stated that a groundbreaking treatment called Trikafta and two other cystic fibrosis medications sold by the company offer notable clinical benefits, but are not cost-effective. A final recommendation, though, will not be released until the agency has digested public comments and holds further deliberations.
The preliminary recommendation does not alter reimbursement for people who have been treated with the Vertex medicines since the company reached a four-year deal with the government in 2019. But if the current agency recommendation is finalized, anyone with the disease — an inherited disorder that causes severe damage to the lungs and other organs — will not have access going forward.