Question to the Department of Health and Social Care:

To ask His Majesty's Government what new treatments or strategies are planned to support sufferers of Cystic Fibrosis through the NHS.

NHS England commissions 47 specialised cystic fibrosis centres for adults and children across England, in addition to supporting the optimal monitoring of patients with cystic fibrosis at home, and best practice in remote consultations.

Through these centres, NHS England provides a range of innovative treatments including inhaled therapies such as nebulised antibiotics. Since 2019, thousands of people with cystic fibrosis have been able to benefit from licensed treatments, firstly Orkambi and Symkevi and then Kaftrio, following its marketing authorisation in 2020. Access to these treatments is provided under the terms of a commercial agreement reached between the manufacturer, Vertex and NHS England, with the full support of the National Institute for Health and Care Excellence (NICE).

Within the agreement between NHS England and Vertex, there is a flexible commercial mechanism to ensure continued access for patients already receiving any of the licensed treatments following the conclusion of a full NICE evaluation.

On 3 November 2023, NICE published a consultation on draft guidance that did not recommend these treatments as a cost-effective use of National Health Service resources. This draft is an initial step in the review of these medicines and does not affect patients’ continued access to these medicines on the NHS in any way. Eligible children and adults with cystic fibrosis can continue to receive ongoing treatment and be initiated onto treatment with these medicines, as clinically appropriate.

On 15 November 2023, the Medicines and Healthcare products Regulatory Agency approved a new licence extension that will mean children as young as two years old with cystic fibrosis are eligible to receive Kaftrio.

NHS England anticipate that as many as 600 children could benefit from this latest approval under the terms of the existing commercial agreement negotiated with Vertex in 2019. NHS cystic fibrosis centres across the country have plans in place to ensure that all children eligible today can be provided long term access to this life-changing treatment.