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Cystic fibrosis: What next for the “wonder drug” Kaftrio?

BMJ 2023; 383 doi: https://doi.org/10.1136/bmj.p2766 (Published 23 November 2023) Cite this as: BMJ 2023;383:p2766
  1. Jacqui Wise
  1. Kent

NHS England has promised that children aged over 2 will have treatment for life with the groundbreaking drug, but NICE says that it’s not cost effective—leaving new parents distressed, reports Jacqui Wise

What is Kaftrio?

The management of patients with cystic fibrosis has been transformed over the past few years by the advent of treatments designed to correct the malfunctioning protein made by the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Kaftrio is the latest of these CFTR modulator treatments and consists of a triple treatment combination of ivacaftor, tezacaftor, and elexacaftor. It’s a long term treatment for patients with cystic fibrosis (CF) who have at least one F508del mutation in the CFTR gene—around 90% of people with CF.

In 2019, after a lengthy battle over cost, an agreement was reached between NHS England and the drug company Vertex to make two earlier CFTR modulator treatments—Orkambi (lumacaftor-ivacaftor) and Symkevi (ivacaftor-tezacaftor)—available on the NHS.12 As part of the interim access agreement NHS England, the National Institute for Health and Care Excellence (NICE), the UK Cystic Fibrosis Trust, and Vertex would collect and share real world data over four years to help resolve uncertainties in the evidence base. In 2020 this agreement was updated to include Kaftrio.3

The NHS described Kaftrio as a “miracle” treatment in January 2022, and Sajid Javid, then health secretary, described it as “life changing.”4 In the UK 10 600 people have CF, and data from 2021 showed that 72% were taking a …

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