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March 22, 2018

MPs call on government to make cystic fibrosis drug available on NHS

Members of parliament have urged NHS England and drug manufacturer Vertex to come to a deal to make a ‘life-changing’ cystic fibrosis (CF) drug available on the NHS in a parliamentary debate.

By Ellen Daniel

Members of parliament have urged NHS England and drug manufacturer Vertex to come to a deal to make a ‘life-changing’ cystic fibrosis (CF) drug available on the NHS in a parliamentary debate.

The debate, which took place in the House of Commons on 19 March, was prompted by a public petition calling for CF drug Orkambi to be made available on the NHS, which has gained 115,419 signatures to date.

The petition, calling for the British Government to help resolve ongoing drug pricing negotiations between Vertex, NHS England and the National Institute for Health and Care Excellence (NICE) ‘as a matter of the utmost urgency’, was launched after NICE’s decision not to approve funding for the drug.

Orkambi is approved by the European Medicines Agency for people who have a F508del mutation that affects the gene for a protein called cystic fibrosis transmembrane conductance regulator (CFTR). This protein is involved in regulating the production of mucus and digestive juices.

The drug is recommended for patients who have inherited the mutation from both parents and therefore have the mutation in both copies of the CFTR gene. 50% of people with CF in the UK have this mutation and the drug has proved effective in improving lung function and quality of life.

NICE, the cost regulator for the NHS, did not approve funding for Orkambi due to cost-effectiveness issues and a lack of long-term data.

In NICE guidance published in July 2016, a committee concluded that the estimated incremental cost-effectiveness ratios for the drug were considerably higher than what is considered a cost-effective use of NHS resources. The drug costs around £104,000 per patient per year of treatment.

Orkambi is currently available in Republic of Ireland, Austria, Germany and the US. Vertex’s other CF drug Kalydeco has been recommended for NHS funding, but it is only effective in around 5% of patients.

Around 500 of the 10,500 CF patients in the UK could benefit from the drug. Some patients are able to access the drug through Vertex’s compassionate use programme, but the majority are not.

During the debate, which was attended by 60 MPs, 22 MPs shared the experiences of constituents with cystic fibrosis. MP Paul Scully, who opened the debate, said: “It is important that [the government] plays the role of middleman to encourage negotiations between Vertex and NHS England to ensure that sufferers can get the cutting-edge treatment they deserve.”

MP Edward Davey called the system used by NICE to evaluate cost-effectiveness into question, saying: “Is the problem that the current system of NICE analysis, using its quality-adjusted life year measures, is unable to take account of negotiated discounts through pipeline agreements?”

CF is an inherited disease that affects the cells that produce mucus and digestive juices in the lungs and the digestive system respectively. The cells produce a thick secretion that causes blockages, inflammation and long-term infection. The condition affects around 10,000 people in the UK, the second highest number of CF cases in the world.

NHS England and Vertex have been involved in pricing negotiations since 2016, with NHS England refusing to make room in its budget to cover the expensive drug. In February 2018, Vertex presented an ‘innovative long-term framework proposal’, but this was rejected by NHS England due to cost. This month NHS England put forward a new proposal to Vertex but this was also rejected.

A spokesperson from NHS England told PharmaTimes that it was ‘unlikely’ the drug would be available unless Vertex reviewed its proposed pricing.

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