Kaleidoscope (QALY in Scope): A Conceptual Framework for Contextualizing Value via Dynamic Cost-Effectiveness Threshold

OBJECTIVES: Cost-effectiveness analysis (CEA) is increasingly integral to health technology assessment (HTA) worldwide, promoting a value-based pricing approach. The cost-effectiveness threshold (CET) (i.e., the willingness-to-pay for an additional health outcome, typically measured in quality adjusted life years (QALY)), profoundly influences the pricing and accessibility of medical interventions. However, traditional static thresholds may inadequately account for nuanced factors affecting societal value judgments, particularly affecting rare and orphan diseases. Dynamic thresholds have been proposed, either applied quantitively or qualitatively and considering different dimensions, among which: disease severity, evidentiary uncertainty, etc. However, there is no consensus among health economist and other key stakeholders on whether and how to quantify such considerations. The objective was to conceptualize an explicit dynamic framework for determining the appropriate cost-effectiveness threshold per QALY tailored to specific assessment contexts.

METHODS: A review of CET literature and criteria was conducted. A specific conceptual framework was developed to inform the appropriateness of different CET per QALY in different assessment contexts.

RESULTS: A conceptual framework was developed to estimate a dynamic CET for specific assessment contexts. The considered dimensions were selected based on feasibility (criteria should be observable and quantifiable). The following criteria were considered in the framework: unmet need, disease severity, rarity, paediatric considerations, evidentiary uncertainty, relative gain versus alternatives, equity, industry presence and fiscal impacts and environmental aspects.

CONCLUSIONS: The proposed framework allows to consider the assessment context and other factors are relevant in setting the CET for an intervention, while promoting transparent, consistent and equitable decision-making in HTA processes. This framing emphasizes the need for nuanced and flexible approaches to valuing health outcomes. Further research to develop empirical tools is needed to ensure equitable and optimal access to innovation to all patients in need.