Vertex Pharma’s cystic fibrosis (CF) drug Orkambi will not be made available on the NHS in England because the cost of treatment is too high for the benefit offered, says NICE.
While Orkambi (lumacaftor/ivacaftor) has been shown to reduced hospitalisations in CF patients, the drug’s benefits on lung function “appeared modest in the short term while the long term benefits were uncertain,” said the agency.
Orkambi costs £104,000 per patient for every year of treatment and is licensed to treat CF patients who have a genetic defect known as the F508del mutation. NICE says there are around 2,750 patients in England who would be eligible for the drug.
The verdict was immediately criticised by the Cystic Fibrosis Trust charity, which said it “demonstrates the weakness of the current NICE assessment process”.
The charity had asked NICE to recommend Orkambi so that real-world evidence for long-term benefits could be collected whilst patients have access to the drug, but this request was denied with NICE saying this would require Orkambi to be supplied “in a cost-effective way”.
“If the company is able to put forward a proposal that provides Orkambi at a cost-effective price, we would welcome it,” said Prof Carole Longson, director of the NICE Centre for Health Technology Evaluation.
The CF Trust has now called on Vertex and NHS England to reach an agreement before the end of July that will allow access to the drug, particularly as new data on Orkambi – not reviewed by NICE – suggests Orkambi almost halved the rate of decline in lung function in people with CF over a two-year period.
The charity said it is working to develop a way to collate real-world evidence using its clinical registry, which holds health data for 99% of people living with CF in the UK.
“This issue is about people’s lives and there is no time to lose,” said CF Trust chief executive Ed Owen.
“We want to see meaningful discussions underway by the end of July to design an access solution for Orkambi and we will not sit idly by if this does not materialise.”
That sentiment was echoed by Simon Bedson, senior vice president and international general manager at Vertex, who said: “The single technology appraisal process is not appropriate for assessing medicines, such as Orkambi, for rare diseases like CF.”
“Vertex has submitted an access scheme to the Department of Health, and we urge the NHS and the government to consider this proposal,” he added.
NICE is also in the process of reviewing evidence PTC Therapeutics’ Translarna (ataluren), which according to the CF Trust could be of use in the treatment of the 500 or so patients in England with CF caused by ‘nonsense’ mutations.
Translarna is approved to treat Duchenne muscular dystrophy (and recommended by NICE) but to date has not been submitted for approval in CF.