Question to the Department of Health and Social Care:

To ask the Secretary of State for Health and Social Care, if she will take steps to make (a) Orkambi, (b) Symkevi and (c) Kaftrio available for everyone with cystic fibrosis.

Since 2019, patients in the National Health Service have been able to access Orkambi, Symkevi and Kaftrio through an interim access agreement between NHS England, the company (Vertex), the National Institute for Health and Care Excellence (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 (MHRA) 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 NICE continues to work with stakeholders to address the issues highlighted by the committee in the draft guidance.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.