Question to the Department of Health and Social Care:

To ask the Secretary of State for Health and Social Care, with reference to the UK Rare Diseases Framework, what assessment he has made of the implications of the rejection of the first gene therapy that has proceeded through NICE’s Standard Technology Appraisal process.

The National Institute for Health and Care Excellence (NICE) has already recommended a number of cell and gene therapies through its technology appraisal programme and these treatments are now available to National Health Service patients in line with NICE’s recommendations.

NICE continues to develop guidance on several other gene therapies, including betibeglogene autotemcel for the treatment of transfusion-dependent beta-thalassaemia and published its draft guidance in February this year. The draft recommendations on betibeglogene autotemcel may be subject to change following public consultation. The next committee meeting for this technology appraisal is on 14 July, with an expected final publication date of 15 September 2021.