What Is Required for "Disease-Modifying Therapy" Value Perception From Leading HTA?
Author(s)
Richard Mee1, Thomas Gilboy, MSc2.
1Access Infinity Ltd, London, United Kingdom, 2Access Infinity, London, United Kingdom.
1Access Infinity Ltd, London, United Kingdom, 2Access Infinity, London, United Kingdom.
OBJECTIVES: Looking to assess products that achieved a positive HTA and higher than average list prices and to understand the common characteristics and aspects of their profiles that payers and HTA feedback cited as positive value drivers
METHODS: Detailed analogue analysis using a comprehensive product database, including research of HTA and P&MA outcomes, product clinical evidence and economic and RWE in the public domain. Research included more than 30 analogues that were launched into a single indication since 2015, products in focus were those that launched at a significantly (>20%) higher price vs the average in their indications at the time of launch, looking at a range of Specialist, non-oncologic indications (inc. but not limited to genetic diseases, CNS and autoimmune disorders Geographic scope was France, Germany, Italy, Spain, England, Canada, China and Brazil
RESULTS: All analogues with significant premium pricing addressed high unmet need populations, whether through targeting severe orphan diseases (e.g. Fintepla, Cablivi) or last lines of other diseases (e.g. Omvoh)
Launching with a novel mechanism of action (Fintepla, Cablivi and Takhzyro) makes payers more willing to accept a claim of being a DMT
Efficacy differentiation is key but H2H studies are not essential, especially if targeting a high unmet need population. However, the more crowded the space and the better established a standard of care in the population, the more H2H studies become critical
Safety and cost differentiation can provide secondary support to efficacy benefit, especially when SoC has serious side effects or underlying costs exist
CONCLUSIONS: • DMT perception, and the resultant price premium, is easier to achieve if demonstrating significant efficacy in a high unmet need population
METHODS: Detailed analogue analysis using a comprehensive product database, including research of HTA and P&MA outcomes, product clinical evidence and economic and RWE in the public domain. Research included more than 30 analogues that were launched into a single indication since 2015, products in focus were those that launched at a significantly (>20%) higher price vs the average in their indications at the time of launch, looking at a range of Specialist, non-oncologic indications (inc. but not limited to genetic diseases, CNS and autoimmune disorders Geographic scope was France, Germany, Italy, Spain, England, Canada, China and Brazil
RESULTS: All analogues with significant premium pricing addressed high unmet need populations, whether through targeting severe orphan diseases (e.g. Fintepla, Cablivi) or last lines of other diseases (e.g. Omvoh)
Launching with a novel mechanism of action (Fintepla, Cablivi and Takhzyro) makes payers more willing to accept a claim of being a DMT
Efficacy differentiation is key but H2H studies are not essential, especially if targeting a high unmet need population. However, the more crowded the space and the better established a standard of care in the population, the more H2H studies become critical
Safety and cost differentiation can provide secondary support to efficacy benefit, especially when SoC has serious side effects or underlying costs exist
CONCLUSIONS: • DMT perception, and the resultant price premium, is easier to achieve if demonstrating significant efficacy in a high unmet need population
Conference/Value in Health Info
2025-11, ISPOR Europe 2025, Glasgow, Scotland
Value in Health, Volume 28, Issue S2
Code
HTA366
Topic
Health Technology Assessment
Topic Subcategory
Decision & Deliberative Processes