MPs call on government to help with Orkambi debate

During a parliamentary debate, which came on the back of a public petition - now with over 115,000 signatures - asking that the drug be made available on the NHS, MPs sought government help in securing further negotiations between the two sides.

Some MPs also reportedly voiced concern that the National Institute for Health and Care Excellence’s appraisal process was not appropriate for assessing rare disease drugs like Orkambi, according to the publication BioCentury.

"The scale of parliamentary engagement in this debate echoes and amplifies the cystic fibrosis (CF) community’s sense of urgency on access to new Vertex medicines," a company spokesperson said.

"As many MPs reflected in the debate, the key challenge we face is the NICE approval process which was developed nearly 20 years ago before precision medicines like Orkambi (lumacaftor/ ivacaftor) for small patient numbers existed – as one participant in the debate said – ‘it is an analogue process for a digital age’.

Orkambi is the first drug for CF directed at treating the cause of the disease in people who have two copies of the F508del mutation, accounting for around 45 percent of cases. This mutation causes the production of an abnormal protein that disrupts how water and chloride are transported in the body.

Data from the Phase III TRAFFIC and TRANSPORT studies show that the therapy significantly improved lung function (as measured by FEV1) by 2.6-4.0 percent, and reduced the rate of pulmonary exacerbations by 30-39 percent in target CF patients.

But in July 2016 NICE published final guidelines rejecting NHS funding for the drug, after concluding that while it was clinically effective its benefits were not sufficient to justify its listed cost of £104,000 per patient for every year of treatment.

Last month, Vertex presented NHS England with an "innovative long-term framework proposal" that hoped to secure access for all eligible patients to its current and future portfolio of cystic fibrosis (CF) medicines.

The firm didn’t reveal specifics of the offer, but said the so-called ‘portfolio approach’ should ensure timely access to its medicines while providing budget certainty and value to the NHS.

However, just days ago the group said it was "astonished and dismayed" by NHS England’s initial response to the offer, which it said "amounts to a refusal to make any additional funding available for Orkambi or future medicines."

According to a source familiar with the situation cited by BioCentury, the NHS suggested that Orkambi and any future CF drugs should be included in the current budget for the firm’s Kalydeco (ivacaftor), thus proposing that Vertex provide Orkambi free of charge.

"The NHS can only offer treatments which are both effective for patients and offer good value for taxpayers, so it’s crucial that drugs companies work with the NHS to get a positive outcome," an NHS England spokesperson, commenting on the situation.

"Following advice from NICE, the NHS has asked this particular drug company to review its proposed pricing, and unless this happens further progress at this time is frankly unlikely."

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