Wales and NI follow England in backing Vertex CF therapies

Three of the four UK nations have agreed a long-term deal with Vertex Pharma for reimbursement of its cystic fibrosis therapies, ensuring permanent access to the drugs for eligible patients.

The new agreement with England, Wales, and Northern Ireland marks the end of a long-standing and sometimes bitter dispute over the high cost of Vertex's CFTR modulator drugs.

It covers all three Vertex products approved for the treatment of CF patients in the UK – Kaftrio (ivacaftor/tezacaftor/elexacaftor), Symkevi (tezacaftor/ivacaftor), and Orkambi (lumacaftor/ivacaftor) – and comes after NICE rejected coverage by the NHS last November due to cost-effectiveness concerns.

NICE had a change of heart in final draft guidance (PDF) published earlier this month, saying it had taken into account the severity of CF and the positive impact of these drugs on patients’ quality and length of life.

Negotiations remain ongoing with the Scottish Medicines Consortium (SMC) for a similar agreement, which also includes access to any future licence extensions of these medicines, in the coming days.

The Cystic Fibrosis Trust’s chief executive, David Ramsden, said the agreements will help “to ensure that everyone now, and in the future, can access these life-changing medicines,” adding: “This is an important moment, but we never forget that these medicines are not a cure, and do not work for some people and we will not stop until everyone with CF can live a life unlimited.”

Meanwhile, Vertex is also in discussions with the NHS and health technology assessment (HTA) bodies about its next-generation CF treatment, generally known as the ‘vanza triple’, which is expected to be filed for approval for use in CF patients ages six and older with regulators around the world in the coming weeks.

In 2020, an interim deal was reached between the NHS in the four UK nations and Vertex, which allowed interim access to the drugs while real-world evidence on their benefits was collected.

Ludovic Fenaux, senior vice president of Vertex International, said the company was “delighted” that a finalised reimbursement deal had been reached to provide extended long-term access to the drugs.

“I would like to acknowledge the collaboration of [the] NHS, […] NICE and the SMC, and also thank the CF community for their contribution in describing the value that these innovative medicines bring to patients,” he said.