Question to the Department of Health and Social Care:

To ask the Secretary of State for Health and Social Care, whether the National Institute for Health and Care Excellence’s Single Technology Appraisal pathway is compatible with emerging innovations including gene therapies.

The National Institute for Health and Care Excellence (NICE) evaluates gene therapies through both its technology appraisal programme and its highly specialised technologies programmes as appropriate. NICE’s processes have been proven to be suitable for the evaluation of advanced therapeutic medicinal products (ATMPs) where companies are willing to price their products in a way that represents value to the taxpayer. NICE has recommended 80% of the ATMPs it has evaluated for use by the National Health Service and they are now available for the treatment of NHS patients, including through the Cancer Drugs Fund and managed access agreements negotiated between the NHS and the manufacturer.

Most recently, in April 2023, NICE recommended eladocagene exuparvovec, the first and currently only gene therapy for children with an ultra-rare genetic disorder, aromatic L-amino acid decarboxylase deficiency, enabling its use on the NHS in England.