OBJECTIVES

: In 2017, the Institute of Clinical and Economic Review (ICER) in the United States (US) adapted its value framework for the assessment of drugs indicated in ultra-rare diseases. Among key changes are higher cost-effectiveness thresholds and societal perspective induced in economic analysis. The objective of this study was to analyse the impact of ICER framework adaptations on HTA outcomes for ultra-orphan drugs.

METHODS

: ICER reports for ultra-orphan drugs evaluated after the framework adaptation in 2017 were obtained from ICER website. ICER reports were analysed and following date extracted: long-term value for money, annual prices for each drug (wholesale acquisition costs, WAC), price discounts to achieve value-based price thresholds, as well as contextual considerations.

RESULTS

: Six reports providing assessment for 14 ultra-orphan drugs were identified. Long-term cost-effectiveness for assessed drugs varied between $135,331/QALY to $5.95 million/QALY. Only three drugs (voretigene neparvovec, C1 esterase inhibitor and deflazacort) out of 14 achieved long-term value below the highest threshold adopted for ultra-orphan drugs ($500,000/QALY). From that, only one (voretigene neparvovec) did not require discount to reach value-based price thresholds of $100,000–$150,000/QALY, and that was only under societal perspective, another adaptation of the framework for ultra-orphan drugs.

CONCLUSIONS

: Despite US ICER special adaptations to its framework for ultra-orphan drugs, demonstrating long-term value for money still represents significant challenge for such products. Moreover, since thresholds for value-based price benchmarks have not been adjusted ($100,000–$150,000/QALY), extensive price cuts are also required. However, such framework adaptations (including value at higher thresholds, societal perspective and contextual considerations) provide wider backgound for payers to inform their formulary decision making of expensive ultra-orphan drugs.