Presentation (CTI)

OBJECTIVES: Health Technology Assessment (HTA) agencies assess new treatments against a comparator reflecting the current standard of care (SoC). Where the SoC is a generic or no active therapy exists, treatments face a higher incremental-cost bar to demonstrate cost-effectiveness than those compared against on-patent or newer alternatives. This penalises innovation in areas of greatest unmet need. This research identifies and critically assesses potential solutions to this low-cost comparator (LCC) problem.
METHODS: We perform a targeted review of 10 HTA bodies spanning Cost-Effectiveness Analysis (CEA)-driven and therapeutic added-value (TAV) systems, examining how treatments addressing unmet needs are assessed. We complement this with a review of the academic literature to identify potential solutions to the LCC problem. Additional solutions were generated through internal group discussions and further explored through ad-hoc literature searches and reviews. For each solution identified, we conducted a qualitative analysis of its merits and drawbacks, identified the types of treatments it would be most suitable for, and provided relevant international precedents, if available.
RESULTS: We identified and defined five solutions within the CEA approach (unmet need modifier, innovation modifier, incorporating lifecycle pricing in CEA models, comparator patent pricing, reference comparator approach) and one outside the CEA approach (clinical value approach), through the HTA and literature reviews and group discussions. The effect of each solution will largely depend on how it is implemented and integrated within existing decision-making processes. Each solution addresses different aspects of the LCC problem and presents distinct challenges.
CONCLUSIONS: The LCC problem remains overlooked in current HTA systems and the academic literature, despite its potential detrimental impact on innovation. Identified solutions address different aspects of the problem while introducing distinct trade-offs. Further research is needed to assess the feasibility and real-world impact of each proposed solution.