Question to the Department of Health and Social Care:

To ask His Majesty's Government what assessment they have made of the barriers to patient access for orphan medicines that do not meet the population size criteria for the Highly Specialised Technologies pathway.

The National Institute for Health and Care Excellence (NICE) highly specialised technologies programme is reserved for the evaluation of a small number of medicines licensed for the treatment of very rare, very severe diseases. Most medicines, including most orphan medicines, are assessed through NICE’s standard technology appraisal (STA) programme. NICE has a strong track record of recommending medicines for rare diseases, and its STA process has been shown to be suitable for the evaluation of these medicines where companies engage constructively in the appraisal process and commercial discussions with NHS England.

The Government has recently announced a number of pilots and projects following the United States and United 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.

NICE has recently refined the criteria for its Highly Specialised Technologies programme and is now undertaking further work to review how rare disease medicines are valued, helping to ensure that its approaches remain appropriate and evidence based.