NICE Once Again Rejects Vertex’s Cystic Fibrosis Drug

Despite appeals, the UK’s drug pricing watchdog has held firm against reimbursing Vertex Pharmaceuticals’ cystic fibrosis drug Orkambi, once again noting its modest short-term benefits to lung function and unknown long-term improvements.

In a final appraisal, the UK’s National Institute for Health and Care Excellence found the cost of a one-year course of treatment — $146,751 — was considerably higher than the standard of care given the drug’s limited short-term benefits.

The committee acknowledged that the drug reduced instances of people with cystic fibrosis being hospitalized, but that did not justify the cost.