Question to the Department of Health and Social Care:

To ask His Majesty's Government what factors informed the decision to limit treatment eligibility to 300 people in its licensed indication, or 500 people across all indications, in England under the second routing criterion of the National Institute for Health and Care Excellence’s Highly Specialised Technologies programme, and what scope there is for change to these limits in the forthcoming consultation.

The criteria for routing topics to the National Institute for Health and Care Excellence’s (NICE) Highly Specialised Technologies (HST) programme for evaluation were developed through stakeholder and public consultation. The HST programme aims to strike a balance between the desirability of supporting access to treatments for ultra rare diseases, and the associated reduction in overall health gain across the National Health Service.

The eligible population number was based on an analysis undertaken of previous, current, and potential future HST topics, along with referencing to NHS England and NHS Improvement’s criteria for defining populations eligible for highly specialised services.

The NICE is currently reviewing the criteria and is presenting its proposal to its board on 11 December 2024 and, if the board approves, will proceed with a public consultation from 19 December 2024. The proposed revisions aim to enhance the predictability and transparency of the application of the HST routing criteria, while maintaining the intent of the HST vision.