Cystic fibrosis drugs to be available on NHS in England within 30 days

Every patient in England with cystic fibrosis who could benefit from one of the new precision drugs that target certain mutations underlying the condition will have access to them within 30 days, NHS England has said after striking a deal with the US manufacturer Vertex Pharmaceuticals.

NHS England has been trying to secure access to Orkambi (a combination of lumacaftor and ivacaftor), Symkevi (tezacaftor and ivacaftor), and Kalydeco (ivacaftor) for more than 18 months. Pressure mounted last month after the Scottish government negotiated a confidential discount with Vertex to make Orkambi available to 400 patients.1

Vertex had wanted £105 000 (€123 000; $138 000) per patient for Orkambi. Last July it rejected what NHS England said was a “fair and final offer” of around £500m over five years for all of the company’s approved drugs and any future drugs.2

At the time the company argued that it couldn’t accept the offer—which amounted to around £10 000 a year per patient—because other countries would demand the same deal, which would not allow it to develop the next generation of drugs to treat cystic fibrosis.

Details of how much NHS England will pay for the drugs has not been disclosed, although the chief executive, Simon Stevens, described the deal as “good for our patients and fair to British taxpayers.” He said that Vertex had agreed to submit its full portfolio of drugs (including, in due course, its new triple therapy) to the National Institute for Health and Care Excellence for appraisal.

The UK has around 8200 people with cystic fibrosis, of whom 4387 would be eligible for treatment because of the latest announcement. A new triple therapy, Trikafta (elexacaftor/tezacaftor/ivacaftor), which is expected to be licensed next year, could benefit 90% of people with cystic fibrosis, said David Ramsden, chief executive at the Cystic Fibrosis Trust.

In 2016 NICE rejected Orkambi for use in the NHS, saying that while it reduced admissions to hospital and use of antibiotics among people with cystic fibrosis, the short term benefits to lung function were modest and the long term benefits uncertain.3

At the time the Cystic Fibrosis Trust argued that, in the two years since NICE had conducted its appraisal, new data showed that Orkambi almost halves the rate of decline in lung function in people with the disease over two years.

Protest at lack of access to the drug led to a debate in parliament last March after an online petition gathered more than 100 000 signatures in just 10 days. Sixty MPs attended the debate, many of whom spoke passionately about constituents desperate to get the treatment.4 Many also criticised NICE’s appraisal processes for concentrating on price and ignoring the ethics of denying someone the chance of a better life.

Sarah Wollaston, the Liberal Democrat MP for Totnes and chair of the health and social care select committee, described the news of the deal as “wonderful.” Her committee has been holding an inquiry into the availability of Orkambi and been pushing for a resolution in negotiations over access on the NHS.

Announcing the deal, Stevens said, “The UK has the second highest prevalence of cystic fibrosis of any country in the world, so today is an important and long hoped for moment for children and adults living with cystic fibrosis.

“That fact also means that any drug company wanting to succeed commercially in this field needs to work constructively with the NHS.”

In a letter to Wollaston, Stevens said that the NICE appraisal was expected to conclude by September 2021. He said, “The company has also agreed a flexible contractual mechanism which means that in all circumstances these three medicines will continue to be available following completion of the NICE appraisal.”

Stevens also said that Vertex had agreed to make the drugs available in Wales and Northern Ireland under the same terms as for England.