Question to the Department of Health and Social Care:

To ask the Secretary of State for Health and Social Care, what assessment his Department has made of the availability of innovative treatments for patients with (a) sickle cell disease and (b) cystic fibrosis.

The National Institute for Health and Care Excellence (NICE) makes recommendations for the National Health Service on the clinical and cost effectiveness of new medicines. The NHS is legally required to fund medicines positively appraised by NICE, usually within three months of the publication of its final guidance. NICE has recently recommended crizanlizumab for the prevention of sickle cell crises, subject to a managed access agreement between the manufacturer and NHS England and NHS Improvement.

In 2020, NHS England and NHS Improvement agreed a four year interim access deal with Vertex for its portfolio of cystic fibrosis medicines in line with the marketing authorisations. This is supported by NICE and will allow eligible NHS patients to benefit from access to innovative treatments for cystic fibrosis while further evidence is collected to inform a future NICE evaluation. Further groups of patients may access these treatments if licences are updated during the term of the agreement.