The National Institute for Health and Care Excellence (NICE) introduced a Highly Specialised Technology (HST) pathway in 2015 to provide more consistency in appraising high cost therapies for rare and neglected diseases. This research evaluates the number of HSTs appraised to date and the actual number of patients treated under this pathway. All HST guidance was identified and disease prevalence/incidence, and recommendations were extracted (to 25-Jun-2021). Actual uptake data was provided by NHS England in April 2021. NICE published 14 recommendations through the Highly Specialised Technology (HST) pathway (after an average of 19.6 months after EC-approval) compared to 357 recommendations through the technology appraisal pathway (single or multiple) since 2015. Approximately 1254 patients have been treated with an HST approved therapy. 5 of the 14 HSTs have exceeded the number of patients treated than the NICE-estimated eligible patient population. Notably, 373 patients had been treated with eculizumab (HST1) since January 2015, compared with an estimated 20-30 patients diagnosed-per-year (estimated total uptake of 120-180 from recommendation). However, in some instances the number of patients treated with an HST were very low: zero patients for Strimvelis® (HST7), ≤10 for eliglustat (HST5), and ≤10 for metreleptin (HST14); much lower than any reported estimates in their respective HST appraisals (Strimvelis®: 3 diagnosed/year; eliglustat: 50-100 eligible patients; metreleptin: <200 eligible patients). Relatively few therapies have been recommended under the NICE HST pathway since 2015 compared to the standard NICE process with STA/MTA. Further, these recommendations have been over 19-months from EC-approval. Even following a positive NICE recommendation, several of these have not achieved meaningful patient access, potentially due to the lack of awareness for rare diseases, and likelihood of underdiagnoses.
© 2021 Published by Elsevier Inc.