OBJECTIVES: The National Institute of Health and Care Excellence (NICE) evaluates the clinical and economic impact of new healthcare technologies to inform public reimbursement recommendations. Consultees can lodge appeals to NICE recommendations within 15 days of final draft guidance. These are subject to scrutiny to see if they fall within the grounds for appeal, and if so, an appeal panel is convened. This research systematically evaluated NICE technology appraisal appeals.

METHODS: All NICE appeals were identified from www.nice.org.uk and key information was extracted (to 22-Dec-2020).

RESULTS: 114 appeals were identified: 61 for Multiple Technology Appraisals, 50 for Single Technology Appraisals (STAs) and 3 for Highly Specialized Technologies (HSTs). 188 MTAs, 463 STAs and 13 HST guidance have been published in total. Of 39 STA and HST appeals with publicly-available information, 38% were upheld, 23% dismissed, 21% rejected, 8% cancelled, and 10% ongoing. For these upheld appeals, 46% resulted in a substantive recommendation improvement (where “not recommended” changed to “recommended”/”optimized”), 33% did not, and 20% had not yet published. 35% of these appeals were manufacturer-submitted, 24% by other parties, and 41% by both. 64% of STA/HST appeals were for oncology drugs. 19% of STAs before 2013 were subject to an appeal versus 7% of those subsequently published. Notably, the only two HSTs with “not recommended” Final Appraisal Determinations (FADs) were subject to successful appeals (with new appraisal committee meetings scheduled). Similarly, the only post-Cancer Drug Fund (CDF) review with a “not recommended” FAD is also subject to an ongoing appeal.

CONCLUSIONS: The NICE appeals process has been leveraged by manufacturers and other stakeholders to successfully challenge FAD guidance. Although the number of appeals has notably reduced in recent years, they are being used increasingly in relation to recent novel NICE access pathways (HST and CDF) and this may set a future trend.