Vertex, NHS England no closer to Orkambi settlement

In a statement, Vertex said it had made the "best offer in the world" to NHS England that includes Orkambi (lumacaftor/ivacaftor) as well as Kalydeco (ivacaftor) and future medicines still in development.

It also provides "budget certainty to NHS England and assurance that patients will have rapid access to advances in medicines soon to come, just like agreements made in other countries," the firm noted.

However, it seems NHS England has rejected the offer, as Vertex said it was now calling on the government to intervene.

"We find it outrageous that NHS England does not see a path forward to provide access for thousands of children and young people to the only medicines that treat the underlying cause of CF.

"We find it unconscionable that the Government is unable to value the importance of these CF medicines and provide access. They are placing a lower value on the life of a CF patient than other countries around the world," the firm argues.

However, in response, a spokesperson for NHS England told PharmaTimes: "This country has a long established, internationally respected independent body NICE which recommends whether or not a drug company is proposing a fair price.

"NICE has been clear that Vertex’ pricing is unsupportable. If Vertex really believe they are offering a reasonable deal they should waive their confidentiality clause and let patients and taxpayers judge whether it is fair."

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.

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. As just 5 percent of CF patients are eligible for treatment with Kalydeco, Vertex did not feel that this was a fair starting point.

In April, ministers from the Department of Health wrote a letter asking that Vertex bring negotiations with NHS England over access to Orkambi "to an urgent resolution", following a debate in parliament sparked by a public petition asking that the drug be made available on the NHS.

"We stand ready to meet any time, any place, to ensure patient access to these life-changing and transformative medicines. Patients do not have time to wait and we share their urgency," Vertex said.

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