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 2025 Highly Specialised Technologies routing criteria updates on the number of orphan medicines being diverted to the Standard Technology Appraisal (STA) pathway; and what steps they are taking to ensure that medicines for populations of 300 to 500 patients are not unfairly penalised by lower cost-effectiveness thresholds under the STA route.

The National Institute for Health and Care Excellence (NICE) highly specialised technologies programme (HST) is reserved for the evaluation of a small number of medicines licensed for the treatment of very rare, very severe diseases. Decisions on whether medicines are routed to the HST programme are taken against a set of published criteria that were updated in April 2025 following public and stakeholder engagement. The purpose of the new criteria is not to change the number or nature of the topics 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 Government has recently announced a number of pilots and projects following the United States and Untied Kingdom partnership on pharmaceuticals that will shape a commercial environment that actively encourages innovation and improves patient access. As part of that work, NICE will review the approach to valuing rare disease medicines through its Health Technology Assessment Innovation Laboratory. This research will consider both technology appraisal and highly specialised technologies routes. The review will assess a range of both radical and incremental options to improve the current framework.