Rare Diseases: Drugs
The question
To ask the Secretary of State for Health and Social Care, if his Department will make an assessment of the potential impact of National Institute for Health and Care Excellence proposed changes to its Highly Specialised Technology routing criteria on access to medicines for people living with rare diseases.
Answered by Karin Smyth
Decisions on whether medicines should be evaluated by the National Institute for Health and Care Excellence (NICE) through its highly specialised technologies (HST) programme are taken by NICE itself against a set of published criteria that have been developed through public and stakeholder engagement.
NICE is currently reviewing its HST criteria and has recently closed its public consultation on proposed changes. NICE will discuss the updated criteria at its Public Board Meeting on 19 March 2025.
The purpose of the review is not to change the number or nature of the topics that are evaluated through the HST programme, but to ensure that the criteria are sufficiently clear and predictable for companies and patient groups and are aligned to the HST vision. The aims of the HST programme will remain unchanged. It is intended to: encourage research on, and innovation for, very rare conditions when there are challenges in generating an evidence base that is robust enough to bring the product to market; secure fairer and more equitable treatment access for very small populations with very rare diseases; and recognise that an approach that maximises health gain for the NHS may not always be acceptable; it could deliver results that are not equitable.
Since 2022/23, NICE has been able to recommend 13 out of 14 medicines that it has appraised through its HST programme.
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