NICE Says Cost of Orkambi for Cystic Fibrosis Is Too High for Low Benefit

In March, the U.K. National Institute for Health and Care Excellence (NICE) issued a draft guidance against recommending Orkambi (lumacaftor-ivacaftor marketed by Vertex Pharmaceuticals) for treating cystic fibrosis (CF).

The agency’s independent appraisal committee concluded that the cost of Orkambi was considerably higher than the current standard of care, and it could not be considered a cost effective use of National Health Service (NHS) resources.

For every year of treatment, Orkambi costs 104,000 euros per patient. The drug is licensed for patients carrying the F508del mutation, a specific mutation within the CFTR gene, the genetic defect that causes CF.

In England alone, approximately 2,750 people would have been eligible for treatment with Orkambi. The Scottish Medicines Consortium issued guidance in May which also does not recommend Orkambi

According to NICE, while data shows that Orkambi can reduce hospital stays for CF patients, the benefits to lung function are modest in the short-term and undetermined in the long-term.

As NICE opts not to recommend Orkambi to the NHS England, the Cystic Fibrosis Trust has suggested that Orkambi be provided to all who need it while further evidence is collected on its long-term clinical impact using the UK Cystic Fibrosis Data Registry.

If NICE allows Orkambi to patients on that basis, the company would have to provide the drug to the NHS in a cost effective way. But Vertex Pharmaceuticals did not advance that proposal for consideration by the NICE committee.

According to a press release, Carole Longson, director of the NICE Centre for Health Technology Evaluation said that the availability of a new CF treatment is important but the high price of Orkambi outweighs the drug’s benefit.

“We can only recommend treatments when we are certain they are both clinically effective and represent good value for money. If the company is able to put forward a proposal that provides Orkambi at a cost-effective price, we would welcome it,” Longson said.

The draft guidance has been issued to consultees, healthcare professionals and members of the public, who can comment on the evaluation. If there are no appeals, a final decision will be handed down in July.

After a final decision, companies are allowed to submit a new proposal demonstrating the cost-effectiveness of the drug, which could be considered by NICE under a rapid review process.

CF is an inherited condition caused by a gene defect in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Patients experience serious  and progressive problems with breathing and digestion that begin at a very young age.