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<p>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.</p><p>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.</p><p>86% of NICE’s TA recommendations for orphan
drugs have been positive.</p><p>NICE has published HST guidance on 12 individual topics,
making 12 recommendations, all of which have been positive.</p><p> </p>
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