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Rare Diseases: Medical Treatments

Question for Department of Health and Social Care

UIN 125626, tabled on 30 January 2018

To ask the Secretary of State for Health and Social Care, what steps his Department is taking to develop an additional appraisal process for new rare disease treatments which do not meet the criteria for the NICE Highly Specialised Technology appraisal route.

Answered on

7 February 2018

The Department has no plans to establish a new assessment process for the evaluation of rare disease treatments that do not meet the criteria for the National Institute for Health and Care Excellence (NICE) Highly Specialised Technology appraisal route. NICE develops guidance on most new drugs through its technology appraisal programme, and has been able to recommend a number of drugs for rare diseases for routine use on the National Health Service.

There is an established topic selection process for the technology appraisal and highly specialised technology evaluation programme that includes a public consultation and is designed to ensure that treatments are only referred for assessment by NICE where its guidance will add value.