Requirements for Payer Recognition of Disease-Modifying Effect in New Drug Launches
Author(s)
Richard Mee, BSc.
Access Infinity, London, United Kingdom.
Access Infinity, London, United Kingdom.
OBJECTIVES: This study aimed to identify and analyse cases where brands achieved significant price premiums, defined as over 20% above the indication average at launch, by demonstrating transformative efficacy or disease-modifying potential. The goal was to understand the characteristics that supported such premium positioning and how payers evaluated these claims
METHODS: An analogue analysis was conducted of over 30 specialty, non-oncology products launched since 2015 into single indications across France, Germany, Italy, Spain, England, Canada, China, and Brazil. Products selected received positive HTA or payer feedback and had notably higher launch prices (>20% higher) compared to other products in the indication. The study examined recurring features cited by payers as value drivers, including clinical innovation, trial design, safety, and cost-effectiveness.
RESULTS: All products achieving premium pricing addressed high unmet need, either by treating severe orphan diseases (e.g., Fintepla, Cablivi) or late-line settings (e.g., Omvoh). A novel mechanism of action frequently strengthened disease-modifying treatment positioning, gaining payer recognition. While strong efficacy was consistently important, head-to-head trials were not always required, particularly when unmet need was high. However, in more competitive spaces with established standards of care (e.g., Spravato), H2H evidence became critical. Safety advantages and cost offsets (e.g., with Fintepla, Takhzyro, Tavneos) played a supportive role in reinforcing the perception of value.
CONCLUSIONS: Achieving a disease-modifying profile and premium pricing is most viable when targeting high unmet need with compelling efficacy and clinical innovation. Novel mechanisms and clear differentiation enhance payer acceptance. While safety and economic benefits can reinforce value, they are insufficient without demonstrable clinical impact. Trial design must align with the treatment landscape, especially when benchmarking against established comparators.
METHODS: An analogue analysis was conducted of over 30 specialty, non-oncology products launched since 2015 into single indications across France, Germany, Italy, Spain, England, Canada, China, and Brazil. Products selected received positive HTA or payer feedback and had notably higher launch prices (>20% higher) compared to other products in the indication. The study examined recurring features cited by payers as value drivers, including clinical innovation, trial design, safety, and cost-effectiveness.
RESULTS: All products achieving premium pricing addressed high unmet need, either by treating severe orphan diseases (e.g., Fintepla, Cablivi) or late-line settings (e.g., Omvoh). A novel mechanism of action frequently strengthened disease-modifying treatment positioning, gaining payer recognition. While strong efficacy was consistently important, head-to-head trials were not always required, particularly when unmet need was high. However, in more competitive spaces with established standards of care (e.g., Spravato), H2H evidence became critical. Safety advantages and cost offsets (e.g., with Fintepla, Takhzyro, Tavneos) played a supportive role in reinforcing the perception of value.
CONCLUSIONS: Achieving a disease-modifying profile and premium pricing is most viable when targeting high unmet need with compelling efficacy and clinical innovation. Novel mechanisms and clear differentiation enhance payer acceptance. While safety and economic benefits can reinforce value, they are insufficient without demonstrable clinical impact. Trial design must align with the treatment landscape, especially when benchmarking against established comparators.
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
EE649
Topic
Economic Evaluation, Health Policy & Regulatory
Disease
No Additional Disease & Conditions/Specialized Treatment Areas