Objectives: New medicines commercialized in the UK are appraised through the National Institute for Health and Care Excellence (NICE). For selected ultra-orphan indications, NICE has provided an alternative approach through the Highly Specialised Technologies (HST) appraisal, with higher cost-effectiveness thresholds rewarding innovation in rare diseases that are unlikely to achieve access through the standard Single Technology Appraisal. Our study is designed to critically assess the utility of the HST for patients, the NHS and pharmaceutical manufacturers.

Methods: A live database has been developed, tracking NICE HST publications and publically available associated documentation. Analysis enables assessment of appraisal timelines, cost-effectiveness evaluation (e.g., QALY, ICER), outcomes / access / restrictions, Managed Access Agreements (MAA) (including key data uncertainties) and Commercial Access Agreements (CAA).

Results: Prior to June 2020, 12 HST appraisals have been published and included in the database. All 12 were approved for funding (including 1 [8%] restricted approval) in a median of 432 days [SD: 212] from evidence submission to decision publication. 10 [83%] were funded through CAAs, including 4 [33%] with additional data collection requirements through MAAs. Initial utilization was slow, with only 2 [16%] HST published in 2015, though an increase in publication rate is observed (4 [32%] in 2019). Furthermore, 16 HSTs are currently in development and 5 HSTs are in the proposal process.

Conclusions: Our study is able to provide insight into HST appraisal opportunities for manufacturers as well as expected timelines and outcomes. With increasing innovation in emerging medical treatments and an ongoing NICE methods review, we expect the HST appraisal to rapidly increase in importance for access to patients in the UK, and therefore in scrutiny. In light of future NICE HST publications (to September 2020), an updated analysis will be presented with additional analysis.