NICE rejects Vertex's cystic fibrosis therapy Orkambi over cost

The National Institute for Health and Care Excellence on Friday declined to recommend Vertex Pharmaceuticals' cystic fibrosis treatment Orkambi (lumacaftor/ivacaftor). In its decision, the agency determined that the therapy's benefits versus standard of care do not justify its cost of 104 000 pounds ($148 755) per patient per year. 

"We know how important a new treatment option would be for people with cystic fibrosis; but for the benefits it offers, the cost of Orkambi is too high," commented Carole Longson, director of the NICE Centre for Health Technology Evaluation. Longson continued "if the company is able to put forward a proposal that provides Orkambi at a cost-effective price, we would welcome it." 

NICE noted that after its previously issued guidance, recommending against NHS reimbursement of the therapy, the Cystic Fibrosis Trust stated that data collected in the real world would demonstrate long-term benefits for Orkambi. However, the agency stated that Vertex would need to provide the therapy on a cost-effective basis for it to be made available in this situation, adding that the company failed to provide such a proposal.  

Commenting on the news, Ed Owen, chief executive of the Cystic Fibrosis Trust, who remarked that the group was "very disappointed" by the guidance, said the decision "demonstrates the weakness of the current system, and NICE has been unable to confidently predict the long-term benefits of the drug." Owen added "however, new data published last month shows the positive impact of Orkambi over a two-year period to slow the decline in lung health." 

Meanwhile, Vertex senior vice president Simon Bedson disclosed that the company is in negotiations with the UK Department of Health to identify a price that would make Orkambi accessible. Bedson remarked "as we've said for many months, the single technology appraisal process is not appropriate for assessing medicines, such as Orkambi, for rare diseases like cystic fibrosis, adding "Vertex has submitted an access scheme to the Department of Health, and we urge the NHS and the Government to consider this proposal." 

Orkambi ​received EU approval​ in November last year and​ ​has been authorised for use in ​other ​markets including the US, Canada and Australia for the treatment of cystic fibrosis.

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