Rare Diseases: Drugs

Department of Health and Social Care written question – answered on 3rd August 2020.

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Photo of Bob Blackman Bob Blackman Conservative, Harrow East

To ask the Secretary of State for Health and Social Care, how many and what proportion of medicine assessments conducted by NICE (a) were for medicines for rare diseases since the start of the highly specialised technologies appraisal process in 2013; how many medicines for rare diseases were approved by NICE as a proportion of total approvals since 2013; and how many medicines have been approved via the highly specialised technologies appraisal process since 2013.

Photo of Jo Churchill Jo Churchill The Parliamentary Under-Secretary for Health and Social Care

The National Institute for Health and Care Excellence (NICE) has undertaken 69 assessments considering ‘orphan drugs’ since the start of its highly specialised technologies (HST) programme in 2013.

57 of these were appraised by its technology appraisals (TA) programme and NICE made 64 recommendations in total as some topics have multiple recommendations. 49 of these recommendations were positive. A further seven appraisals were terminated as the company did not make an evidence submission. This equates to 16% of NICE’s appraisal activity.

86% of NICE’s TA recommendations for orphan drugs have been positive.

NICE has published HST guidance on 12 individual topics, making 12 recommendations, all of which have been positive.

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