Question to the Department of Health and Social Care:

To ask the Secretary of State for Health and Social Care, what steps his Department is taking to ensure that people living with rare diseases in England have timely access to innovative medicines.

The National Institute for Health and Care Excellence (NICE) is the independent body responsible for developing authoritative, evidence-based recommendations for the National Health Service on whether new medicines represent a clinically and cost-effective use of resources. NICE plays a key role in supporting the adoption of innovative new medicines to improve outcomes for patients, at a price that reflects their value and is fair to the taxpayer.

NICE has a strong track record of recommending medicines for the treatment of rare diseases and its standard technology appraisal process has been proven to be suitable for the evaluation of these medicines, where companies engage constructively in the appraisal and commercial discussions with NHS England. NICE’s approval rate for medicines for rare diseases is in line with its overall approval rate for new medicines, with 91% of medicines licensed for rare diseases recommended for some or all of the eligible patient population since April 2024.

NICE’s also operates a separate highly specialised technologies programme (HST) which is reserved for a small number of medicines for very rare and very severe diseases. The HST programme uses a much higher cost-effectiveness threshold than a standard NICE appraisal, that recognises the challenges of developing medicines for very small patient populations.

The Government remains committed to improving the lives of people living with rare diseases through the UK Rare Diseases Framework, which includes improving access to specialist care, treatment, and drugs as one of its four priorities. We are committed to ensuring that this priority is a key driver of the action plans published under this framework.