Question to the Department of Health and Social Care:

To ask the Secretary of State for Health and Social Care, whether his Department plans to examine options for a separate evaluation process for orphan medicines claiming significant benefit over existing treatments.

There are no plans to introduce a separate evaluation process for orphan medicines. The National Institute for Health and Care Excellence (NICE) makes recommendations for the National Health Service on whether all new licensed medicines, including medicines for rare diseases, should be routinely funded by the NHS based on an assessment of their costs and benefits. NICE operates a separate highly specialised technologies programme for the evaluation of a small number of treatments for very rare, very severe diseases which uses a much higher cost-effectiveness threshold that recognises the challenges of bringing treatments for very rare diseases to market.

NICE’s methods have been proven to be suitable for the evaluation of rare disease medicines, where companies are willing to price their medicines fairly. NICE’s approval rate for medicines for rare diseases is in line with its overall approval rate for new medicines, and from April 2024 to April 2025, NICE recommended all 15 of the rare disease drugs that it evaluated through its standard technology appraisal programme.