Question to the Department of Health and Social Care:

To ask the Secretary of State for Health and Social Care, with reference to the recommendation from NICE that modulator therapies used to treat cystic fibrosis should no longer be funded on the NHS, when she plans to make a decision on this matter: and what steps her Department is taking to ensure that life-saving treatment is made available for children with cystic fibrosis.

The National Institute for Health and Care Excellence (NICE) is the independent body that makes evidence-based recommendations for the National Health Service on whether medicines should be routinely funded by the NHS based on an assessment of their costs and benefits. NICE ensures that the price that the NHS pays for medicines represents value and it recommends the vast majority of medicines for use on the NHS, often as a result of commercial arrangements proposed during the NICE evaluation process. The NHS is legally required to fund medicines recommended by NICE.

Since 2019, NHS patients have been able to access Orkambi, Symkevi and Kaftrio through an interim access agreement between NHS England, the company (Vertex), NICE, and the UK Cystic Fibrosis Trust. The agreement makes the medicines available for a limited time at a reduced price, while further information has been collected to inform a NICE appraisal. On 15 November 2023, the Medicines and Healthcare products Regulatory Agency approved a new licence extension and children as young as two years old with cystic fibrosis are now eligible to receive Kaftrio through the interim access agreement.

NICE is currently consulting on its draft recommendations on the use of Orkambi, Symkevi and Kaftrio and has not yet published its final recommendations. NICE continues to work with stakeholders to address the issues highlighted by the committee in the draft guidance. Under the terms of the interim access agreement for the cystic fibrosis medicines, Orkambi, Symkevi and Kaftrio, eligible children and adults with cystic fibrosis can continue to receive ongoing treatment and be initiated onto treatment with these medicines, as clinically appropriate, while NICE concludes its evaluation.