Rare Diseases: Medical Treatments

(asked on 21st November 2023) - View Source

Question to the Department of Health and Social Care:

To ask the Secretary of State for Health and Social Care, with reference to section 6 of the National Institute for Health and Care Excellence's (NICE) health technology evaluations: the manual, published on 31 January 2022, whether she has had discussions with NICE on the potential impact of the severity modifier on technology appraisals for (a) cystic fibrosis and (b) other rare diseases.


Answered by
Andrew Stephenson Portrait
Andrew Stephenson
Minister of State (Department of Health and Social Care)
This question was answered on 28th November 2023

Departmental officials regularly discuss a range of issues with the National Institute for Health and Care Excellence (NICE), including in relation to the application of the severity modifier that was introduced in 2022. NICE is responsible for determining whether a severity weighting should apply to the evaluation of individual medicines in line with its published methods and processes. NICE has recently published draft guidance for consultation on the use of Orkambi, Symkevi and Kaftrio for the treatment of cystic fibrosis and stakeholders now have an opportunity to comment on NICE’s recommendations. NICE continues to work with stakeholders to address the issues highlighted by the committee in the draft guidance.

Reticulating Splines