NICE Approves Vertex’s Modulator Drugs for Cystic Fibrosis: A Landmark Decision for the NHS

The National Institute for Health and Care Excellence (NICE) has granted approval for the life-changing cystic fibrosis (CF) modulator drugs—Kaftrio, Symkevi, and Orkambi—to be made available through the NHS in England. This milestone follows extensive advocacy by the Cystic Fibrosis Trust and the broader CF community, marking a pivotal moment for thousands affected by this debilitating condition.

CF is a chronic, life-limiting genetic disorder with no current cure. The availability of these modulator therapies on the NHS offers new hope and a significantly improved quality of life for patients. However, despite being a hugely positive moment, David Ramsden, chief executive of Cystic Fibrosis Trust, advised that ‘We should not forget though, that these treatments are not a cure and simply don’t work for some people. With the support of our incredible community, clinicians and researchers, a lot has been achieved, but we know there is still lots more to do”

In November 2023, NICE acknowledged the clinical efficacy of Vertex’s CF treatments in draft guidance but stopped short of recommending them as cost-effective, causing disappointment in patients and their families. The draft stated that "even when considering the condition’s severity ... the most likely cost-effectiveness estimates for [Kaftrio], [Symkevi], and [Orkambi] are above the range that NICE considers an acceptable use of NHS resources." This echoed a prolonged standoff over the pricing of Vertex’s CF drugs that had previously been resolved in 2019 with a four-year government reimbursement deal, which included Orkambi and other CF drugs. Kaftrio was added to this scheme in 2020. 

Vertex announced the updated long-term reimbursement deal with the NHS on Thursday, ensuring access to Kaftrio, Symkevi, and Orkambi for all existing and future eligible cystic fibrosis patients in England. This agreement follows NICE’s positive recommendation for Vertex’s CFTR modulators, reflecting successful negotiations that address both clinical efficacy and cost concerns.

The new deal, a result of collaboration among Vertex, NICE, and the NHS, ensures not only immediate access to these critical drugs but also includes provisions for future license extensions and rapid access to new treatments. Vertex is working on a next-generation CF therapy combining vanzacaftor, tezecaftor, and deutivacaftor, which will also be covered under this agreement pending regulatory approval.

Ludovic Fenaux, Senior Vice President of Vertex International, expressed his satisfaction with the agreement: “We are delighted to have agreed extended long-term access to Kaftrio, Symkevi, and Orkambi for eligible CF patients in England. I would like to acknowledge the collaboration of NHS England, NICE, and the Scottish Medicines Consortium (SMC), and thank the CF community for highlighting the value these innovative medicines bring to patients.”

This decision represents a significant advancement in the treatment of cystic fibrosis, promising to transform the lives of those affected by this challenging condition.

Similar access agreements are anticipated to be formalized soon in Scotland, Wales, and Northern Ireland, ensuring consistent availability of these therapies across the UK.

Vertex’s portfolio of CF medicines is broadly available in over 60 countries, including Australia, France, Italy, Germany, the Republic of Ireland, the Netherlands, Spain, and the U.S. The company continues to work closely with regulatory and health authorities worldwide to ensure broad access to its innovative treatments.

As the pharmaceutical community reflects on this landmark decision, it underscores the importance of collaboration and negotiation in bringing life-saving treatments to patients while balancing cost-effectiveness and accessibility.