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 potential quality of life benefits of potentially one-time treatments for patients with life-long and chronic diseases.

The Government would like all National Health Service patients in England to benefit from innovative and effective new treatments in a way that represents value to taxpayers. All new medicines, including cell and gene therapies, are appraised by the National Institute for Health and Care Excellence (NICE) which makes recommendations for the NHS on whether they should be funded by the NHS, considering clinical and cost effectiveness. NICE aims to publish guidance on new medicines around the time of licensing wherever possible and the NHS is legally required to make funding available for NICE recommended treatments.

The NHS has struck commercial deals with the manufacturers of several cell and gene therapies enabling NICE to recommend them for NHS patients, including through the Government’s £340 million Cancer Drugs Fund. NHS patients in England were among the first in the world to benefit from access to CAR-T cancer therapy and, NHS England has also negotiated deals to secure lifesaving gene therapies Zolgensma® and Libmeldy® for patients with spinal muscular atrophy and metachromatic leukodystophy, respectively.