Question to the Department of Health and Social Care:

To ask His Majesty's Government what assessment they have made of the recommendation in the report by the UK BioIndustry Association From innovation to impact: unlocking patient access to innovative rare disease medicines, published on 4 December 2025, to develop a separate evaluation pathway for innovative orphan medicines where a cost-effectiveness based evaluation is not appropriate, and of its implementation.

There are no plans to introduce a separate evaluation process for orphan medicines. Most medicines, including orphan medicines, are assessed through the National Institute for Health and Care Excellence’s (NICE) standard technology appraisal programme, with a small number of treatments for very rare and severe conditions considered through the highly specialised technologies programme, which applies a higher cost-effectiveness threshold.

NICE’s methods are suitable for evaluating rare disease medicines where prices are set fairly. Approval rates for rare disease medicines are in line with overall NICE recommendations, and between April 2024 and April 2025 NICE recommended all 15 rare disease medicines assessed through the standard programme.

We are also investing approximately 25% more in innovative treatments through an increase to NICE’s cost-effectiveness threshold and changes to how health benefits are valued. This will support access to medicines delivering significant health benefits, including for rare diseases, that may previously have been declined on cost-effectiveness grounds.