PRO117 ACCESS TO ORPHAN DRUGS IN THE UNITED KINGDOM: A REVIEW OF NICE DECISIONS FROM 2017-2018

Abstract

This study examined how National Institute for Health and Care Excellence (NICE) decisions and resultant patient access differ by drug characteristics and appraisal process in the UK. Published NICE technology appraisal guidance documents for therapies evaluated from January 2017-December 2018 were reviewed; characteristics and results were assessed in a standardised manner to categorise drugs by orphan status, disease condition (oncology vs non-oncology), review type, and NICE decision (“recommend without restrictions,” “recommend with restrictions,” and “not recommended”). The proportion of each decision type was compared numerically between categories. During 2017-2018, NICE published 191 decisions, 73% for drugs indicated for non-orphan conditions and 27% for orphan conditions. Forty-eight percent of orphan indications were recommended without restriction vs 36% of non-orphan indications. Of the drugs for orphan conditions, 75% were for oncology, and a higher proportion of oncology indications were recommended without restriction (54%) vs non-oncology indications (31%). Of 13 orphan drugs for non-oncology conditions, 62% were for adults; 54% were reviewed through the highly specialised technologies (HST) process and the remainder through single technology appraisal (STA). HST utilises a higher threshold for incremental cost-effectiveness ratio to evaluate medicines compared with STA. Of the 6 orphan drugs for non-oncology conditions reviewed through STA, none were recommended without restrictions, 5 were recommended with restrictions on their marketing authorisation and contingent upon the availability of a patient access scheme, and 1 was not recommended. Conversely, 4 of 7 orphan drugs for non-oncology conditions reviewed through HST were recommended without restrictions, and 3 were recommended with restrictions. All orphan drugs for non-oncology conditions in paediatrics (38%) were evaluated through HST. These findings suggest that orphan non-oncology medicines evaluated through STA have a lower likelihood of achieving a recommendation within the full marketing authorisation than orphan oncology medicines or those evaluated through HST.