Precedents for Reimbursed Highly Specialised Technologies Appraised By the National Institute for Health and Care Excellence: Key Takeaways and Recommendations
Author(s)
Bappoo Y1, Sandhu D1, Wright W1, Burton M1, Freemyer K2
1FIECON, London, LON, UK, 2FIECON, Bloomfield, NJ, USA
OBJECTIVES: To determine precedents for decision-making and acceptability of evidence in completed National Institute of Health and Care Excellence (NICE) highly specialized technology (HST) appraisals.
METHODS: The NICE website was searched in May 2024 for completed HST assessments from 2015-present (assessments currently ‘in development’ were excluded). Positive reimbursement outcome, carer disutility, discount, and QALY weighting were key metrics that were extracted. RESULTS: Of 28 completed HSTs, 27/28 were recommended for reimbursement, of which 6 included a managed access agreement (MAA) and 21 included a simple patient access scheme (PAS). No HSTs have entered the innovative medicine fund (IMF) since its inception in 2022. The NICE committee accepted carer disutilities in 15 of the 21 HSTs in which the manufacturer submitted with carer disutilities (13 accepted in the base-case, 2 in scenario analyses). A 1.5% discount on costs and QALYs was accepted in only 1/27 appraisals. An incremental quality-adjusted life years (QALY) gain of 10–30 was accepted by NICE in 16/27 appraisals, resulting in an ICER threshold of between £100,000–£300,000. Vignette studies and Delphi panels were accepted as alternative sources of quality of life (QoL) outcomes in the absence of data directly from the clinical trial.CONCLUSIONS: This analysis of completed HSTs since 2015 highlighted several key takeaways for manufacturers: (1) It is extremely difficult to achieve the 1.5% discount rate; (2) NICE continues to prefer a simple PAS and this may be preferable versus entering the IMF; (3) carer disutilities are generally accepted in HSTs and can demonstrate broader product value; (4) Delphi panels or vignette studies are accepted in HSTs.
Conference/Value in Health Info
Value in Health, Volume 27, Issue 12, S2 (December 2024)
Code
HTA198
Topic
Economic Evaluation, Study Approaches
Topic Subcategory
Cost-comparison, Effectiveness, Utility, Benefit Analysis, Literature Review & Synthesis
Disease
Rare & Orphan Diseases