Health regulators in the United Kingdom on Wednesday recommended against covering a pricey cystic fibrosis drug developed in San Diego.
A National Institute for Health and Care Excellence committee said costs associated with the drug, sold under the brand name Orkambi, "were considerably higher than what is normally considered a cost-effective use of NHS resources."
Orkambi is the first drug many patients with cystic fibrosis can take to treat their underlying condition. It can reduce the occurrence of lung flare-ups that lead to frequent hospitalization.
But the drug, developed in San Diego by the Boston-based company Vertex Pharmaceuticals, does not come cheap.
Orkambi's price tag in the United States is $259,000 per patient per year. That high cost has set off debate among patients and scientists in the cystic fibrosis community.
The U.K.'s national health care system would have paid less for the drug. But regulators determined its £104,000 annual per-patient price was not justified by its clinical effectiveness.
In a statement, Vertex Senior Vice President Simon Bedson said the committee’s evaluation process was "not appropriate for assessing medicines, such as Orkambi, for rare diseases like cystic fibrosis."
"We are concerned that application of the wrong appraisal process may lead to no access to Orkambi for eligible patients in England, who currently do not have a medicine that treats the underlying cause of their disease," Bedson said.
Vertex has maintained the drug's price is based on years of development. The company says it will channel part of the revenue from Orkambi toward research on new cystic fibrosis drugs.
To see previous KPBS coverage of the debate over high cystic fibrosis drug pricing, watch the video below:
