Ministers from the Department of Health have written a letter asking that Vertex bring negotiations with NHS England over access to cystic fibrosis drug Orkambi “to an urgent resolution”.
The move follows a debate in parliament sparked by a public petition asking that the drug be made available on the NHS - which at last count had gathered more than 116,000 signatures - during which MPs sought government help in securing further negotiations between the two sides.
Orkambi (lumacaftor/ivacaftor) 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.
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.
In February this year, Vertex presented NHS England with what it called 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.
However, NHS England’s counter offer proposed 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.
And given that just 5 percent of CF patients are eligible for treatment with Kalydeco, Vertex does not feel that this is a fair starting point for negotiations, a company spokesperson told PharmaTimes.
However, in the letter to Vertex, which the firm says it only became aware of with its emergence in media reports, Steve Brine, under secretary of state for Public Health and Primary Care, and Lord O’Shaughnessy, under secretary of state for health, argue that the counter-offer from NHS England “was an important demonstration of its significant commitment to taking a collaborative and flexible approach in any discussions with you if you can commit to pricing that is responsible and proportionate to the benefits patients receive.”
Noting that “time really is of the essence,” the ministers call on Vertex “to proceed with negotiations in a way that is constructive and supports our joint aim of securing access to your medicines currently licenced in the UK at a price that is cost-effective and fair.
“We urge that you provide all available data supporting the cost-effectiveness of your proposal promptly to ensure progress towards an agreement.”
Vertex told PharmaTimes that it was particularly surprised to learn of the letter given that a meeting date for further negotiations with NHS England had already been set, and as it had been asked by NHS England to sign a non-disclosure agreement to keep the debate out of the media. The spokesperson also confirmed that the firm is yet to actually receive the letter.
“We are pleased that the Government is in agreement that we urgently need to find a solution to get our medicines to cystic fibrosis patients as soon as possible and are encouraged that we already have a date in the diary in the coming days to start a meaningful dialogue,” the firm said in a statement.
“The current counter offer amounts to a refusal to make any additional funding available for Orkambi or future medicines….We hope we can agree an approach with NHS England that benefits all eligible patients equally, as soon as possible. We call on the NHS to bring a positive and flexible approach in the best interest of CF patients.”
Also in defence of Orkambi’s pricing, the Vertex spokesperson highlighted that 70 percent of the firm’s revenues over the last five years has been re-invested in R&D, in the hope of eventually being able to treat all patients with the CF given that only around 50 percent are currently eligible for treatment with its medicines.