Question to the Department of Health and Social Care:

To ask His Majesty's Government what assessment they have made of the impact of the “cliff edge” in incremental cost-effectiveness ratios on orphan medicines that narrowly miss the criteria to be appraised by the National Institute for Health and Care Excellence's highly specialised technologies programme, and the resulting impact on access to treatments for people living with rare diseases.

The National Institute for Health and Care Excellence’s (NICE) highly specialised technologies programme makes recommendations for the National Health Service on the use of medicines for the treatment of patients with very rare diseases with a high unmet need. The NICE's methods and processes for the evaluation of highly specialised technologies represents a deliberate departure from its standard technology appraisal methods, as it recognises the challenges of bringing treatment for very rare diseases to market.

Decisions on whether medicines are routed to the highly specialised technologies programme are taken on the basis of a set of criteria that have been carefully developed through public and stakeholder engagement, and which have recently been subject to a public consultation. The NICE is currently considering responses to the consultation and expects to implement the final updated criteria in April 2025. The NICE is able to recommend most orphan medicines evaluated through its standard technology appraisal programme for use in the NHS.