Expanding the Horizon of Cost-Effectiveness Analysis: A Comprehensive Review of Alternative Methods

OBJECTIVES: Alternative methods to classical cost-effectiveness analysis (CEA) have gained attention in recent years, particularly with the growing importance of Value Assessment in the United States. As the healthcare landscape evolves, there is a recognized need to explore and adopt alternative approaches that do not rely on quality-adjusted life years (QALYs). This study aimed to shed light on the current state and use of alternative CEA methodologies, presenting a comprehensive overview of their visibility and adoption in the existing literature.

METHODS: We performed a targeted search to capture a broad spectrum of methodologies that go beyond the conventional QALY framework, published in the last 20 years. We employed the following search terms: Generalized Risk Adjusted Cost-Effectiveness (GRACE), Generalized Cost-Effectiveness Analysis (GCEA), Equivalent Life Years Gained (evLYG), Healthy Years in Total (HYT); Efficiency Frontier (EF), Burden Augmented by Deadliness and Impact (BADI), Absolute or Proportional Shortfall to identify articles discussing alternative approaches to CEA.

RESULTS: Preliminary results show that among these methodologies, EF analysis garnered the most attention, reflected in 262 publications. GRACE, GCEA, evLYG, BADI and HYT collectively had 25 publications, with many focused on methodological descriptions and few on results of analyses applying the methods.

CONCLUSIONS: While expanded forms of the QALY, such as GRACE and GCEA, are increasingly the subject of discussion among academics and HTA bodies, the broad adoption of these alternative and theoretically superior approaches to unadjusted QALY-based CEA appears very limited. Our findings suggest a missed opportunity to address the inherent limitations and lack of evolution of classical CEA as it has been used in healthcare over multiple decades. Since these theoretically more robust alternatives to the QALY and to CEA have been described at least as early as 2018, the results of our research beg the question: what is delaying their adoption?