Question to the Department of Health and Social Care:

To ask the Secretary of State for Health and Social Care, what assessment he has made of the adequacy of the National Institute for Health and Care Excellence (NICE)'s current Highly Specialised Technologies Guidance, specifically where it does not recommend a treatment for a rare disease where treatment would have a substantial effect of a medicine on quality and length of life, but is not recommended due to cost-effectiveness estimates being higher than what NICE usually considers an acceptable use of NHS resources for Highly Specialised Technologies.

The National Institute for Health and Care Excellence (NICE) operates a separate Highly Specialised Technologies (HST) programme to evaluate a very small number of medicines and treatments developed for ultra‑rare, severe and life‑limiting conditions. It uses specific methods and a much higher cost‑effectiveness threshold than standard technology appraisals, enabling NICE to recommend treatments at prices that reflect the complexities of ultra‑rare diseases. The HST programme has secured access for National Health Service patients with very rare diseases to effective treatments that NICE would not have been able to recommend through its standard technology appraisal process, with 33 out of the 35 medicines that it has evaluated through the HST programme recommended for NHS use.

There will unfortunately always be occasions when NICE is unable to recommend a treatment through the HST programme despite the use of a much higher cost-effectiveness threshold. These are very difficult decisions to make, and it is right that they are taken independently and on the basis of a thorough assessment of the available evidence.