PRO108 ANALYSIS OF NICE AND US ICER HTA OUTCOMES FOR ULTRA-ORPHAN MEDICINAL PRODUCTS

Abstract

The National Institute for Health and Care Excellence (NICE) in the United Kingdom and the Institute of Clinical and Economic Review (ICER) in the United States adapted their frameworks for the assessment of costly drugs used in ultra-rare diseases. The objective of this study was to analyse actual outcomes of both frameworks by reviewing HTA reports released by both agencies. ICER reports for drugs evaluated with the framework modified for ultra-orphan drugs (adapted in 2017) were obtained from ICER’s website. For the same drugs, Highly Specialized Technology (HST) reports were searched on the NICE website. In case HST reports were not found, a subsequent search was conducted to check if the drug was assessed under another NICE programme (e.g. Single Technology Appraisal (STA)). Total of 14 ultra-orphan drugs was evaluated by ICER. Among them, 9 cases were identified on NICE website (5 under HST and 4 under STA), but recommendations were currently published only for 3 cases: inotersen for hereditary transthyretin-related amyloidosis, lumacaftor/ivacaftor for cystic fibrosis and nusinersen for spinal muscular atrophy. Only inotersen was assessed as HST while remaining two under STA. All 3 drugs were reported not cost-effective according to ICER, while NICE assessed inotersen as cost-effective (HST) and nusinersen as potentially cost-effective (STA) indicating uncertainties that should be addressed. Both received positive NICE recommendations. Only lumacaftor/ivacaftor was not recommended by NICE due to cost-effectiveness ratio exceeding acceptable range (STA). While both organizations included special adaptations for ultra-orphan drugs to their assessment frameworks, drugs are less commonly qualified for the dedicated programme in NICE than for ultra-orphan framework in ICER. However, despite the use of specific criteria, demonstration of cost-effectiveness under ICER framework still represents a significant challenge. In NICE, HST pathway may increase chances for positive outcome while additional discounts should be applied under standard STA programme.