To ask His Majesty's Government whether they plan to hold discussions with the National Institute for Health and Care Excellence about its appraisal system to ensure it is sufficiently flexible to respond to treatments and medicines for rare diseases.
Answered on
8 May 2024
The Department regularly meets with the National Institute for Health and Care Excellence (NICE) to discuss a range of issues including access to and availability of medicines. NICE’s methods and processes for health technology evaluation have been proven to be suitable for medicines for rare diseases where companies price their products responsibly, and NICE introduced a number of changes to its methods and processes in 2022 that ensure that its processes are appropriate to the evaluation of emerging new technologies.
NICE also operates a highly specialised technologies programme for the evaluation of a small number of medicines for the treatment of very rare diseases. As of 31 March 2024, NICE has recommended 88% of medicines licensed for the treatment of rare diseases for some or all of the eligible patient population, which is comparable to NICE’s approval rate for all medicines.
The Innovative Medicines Fund, building on the success of the Cancer Drugs Fund, provides a mechanism for consistent and transparent managed access process for companies offering promising non-cancer medicines at a responsible price. The Fund has already provided early access for National Health Service patients to several innovative new treatments, including for patients with rare diseases such as graft-versus-host disease and Wolman disease.