Vertex Pharmaceuticals’ cystic fibrosis drugs available on the NHS

Vertex Pharmaceuticals' cystic fibrosis drugs available on the NHS

NHS England has announced that it has secured a definitive agreement with Vertex Pharmaceuticals to make all three of its UK-licensed cystic fibrosis drugs available on the NHS.

This means NHS patients will now have full access to Orkambi, Symkevi and Kalydeco, and around 5000 people may now take up these treatments. There is no cap on patient numbers, and each and every patient in England who might benefit can now get these treatments, free on the NHS. Clinicians will be able to begin prescribing these drugs within 30 days.

NHS England has been able to finalise this negotiation because the company agreed confidential commercial terms that constitute good value for British taxpayers and agreed to submit its drugs for full NICE appraisal.

The agreement provides access to all three drugs for all current licensed indications, as well as future licence extensions too.

Announcing the agreement, NHS Chief Executive Simon 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.

“I’m pleased that Vertex has now agreed a deal that is good for our patients and fair to British taxpayers.”

NICE has also been able to back the agreement, and a binding condition of the deal is that Vertex will submit its full portfolio – including in due course its new triple therapy – to NICE for comprehensive appraisal.

The company has also agreed to a flexible commercial mechanism which will ensure continued patient access to these three medicines following completion of the NICE appraisal.

David Ramsden, Chief Executive of the Cystic Fibrosis Trust, said: “This is a very special day and I want to thank people with cystic fibrosis, their families and everyone who has been part of this campaign for their persistence and determination to keep on fighting. We know it doesn’t end here – we will continue to fight for similar access in Wales and Northern Ireland so that all people with cystic fibrosis in the UK are able to benefit from these life-saving drugs.

“Also, with news that the new triple therapy, Trikafta, which 90% of people with cystic fibrosis could stand to benefit from, could be licenced for use in the coming year, we will continue to campaign so that people with cystic fibrosis never have to wait again for the best available medicines.”

Meindert Boysen, Director of the Centre for Health Technology Evaluation at NICE, said: “We are pleased an agreement has been reached between NHS England and Vertex that brings these medicines to patients. It is also welcome that the company will now re-engage with the NICE process.

“We look forward to working with the company and patient organisations to get ready for the appraisals, including developing the approach to the collection of evidence.”

Commercial terms of the agreement are confidential, but the approach achieved is consistent with NHS England’s other managed access agreements reached with the pharmaceutical industry.

Simon Stevens has written to the Chair of Parliament’s Health and Social Care Committee setting out more detail.

Investment in world-class, cutting edge technologies and treatments is at the heart of the NHS Long Term Plan. Over the last two years, as envisaged by the Accelerated Access Review, NHS England has established an expert commercial drugs team which has been working closely with industry and NICE, and has successfully negotiated a number of innovative deals, making new and promising treatments available for NHS patients.

Recent such ‘smart deals’ include:

  • a deal to make ocrelizumab, an innovative multiple sclerosis drug available;
  • a ground-breaking deal which could eliminate Hepatitis C;
  • a £300m saving after negotiating deals with five manufacturers on low cost versions of the health service’s most costly drug, adalimumab;
  • striking the first full access deal in Europe for CAR-T therapy via the Cancer Drugs Fund, which can potentially cure some children and adults with blood cancers where other treatments have failed;
  • reaching a deal to make the life-extending lung cancer drug pembrolizumab available for routine use on the NHS;
  • a deal to make nusinersen available – the first treatment that targets the underlying cause of spinal muscular atrophy.