Rare Diseases: Health Services
(asked on 9th October 2026) - View Source
Question to the Department of Health and Social Care:
To ask the Secretary of State for Health and Social Care, what recent discussions she has had with NHS leaders on improving outcomes for people with rare genetic diseases and conditions over the longer term.
The Government is committed to improving the lives of those living with rare genetic conditions and diseases, such as GNB-1.
Through the UK Rare Diseases Framework, we support access to specialist care, treatment, and drugs as a priority. This has included NHS England developing a strategic approach to commissioning advanced therapy medicinal products (ATMPs) to facilitate rapid access to National Institute for Health and Care Excellence approved therapies.
We continue to invest in ATMPs through initiatives such as the Cell and Gene Therapy Catapult. With £17.9 million of new funding via the National Institute of Health and Care Research (NIHR), the Advanced Therapy Treatment Centres are driving forward work to enhance the United Kingdom’s environment for ATMP clinical trials and to progress research within the NHS to ensure patients can get safe access to these innovative therapies.
NIHR’s ‘Be Part of Research’ programme allows individuals to see if there is any current research taking place into their conditions, and express their interest in taking part. Further information on NIHR’s ‘Be Part of Research’ is available at the following link: