Presentation (CTI)

OBJECTIVES: Evaluating the cost-effectiveness of emerging treatments for type 1 diabetes (T1D), particularly those with disease-modifying potential such as teplizumab, requires a broader and more nuanced approach than that of traditional frameworks. While cost-effectiveness models based on cost per quality-adjusted life year (QALY) remain central to health technology assessment, they often focus narrowly on direct clinical outcomes, potentially overlooking other important dimensions of value. For example, therapies that preserve beta-cell function may offer benefits beyond QALY gains, such as delaying disease progression, reducing emotional and caregiver burden, postponing the need for intensive disease management, and preserving future treatment options. This study proposes a novel value framework that incorporates a wider range of value domains to more comprehensively estimate the total value of healthcare technologies in T1D.
METHODS: Based on a review of current value assessment methodologies, we propose a comprehensive framework designed to align with the evolving landscape of healthcare technologies. This proposal synthesises established approaches with forward-looking considerations to address emerging trends and challenges in the field of T1D.
RESULTS: Our framework proposes the consideration of a broader set of value elements beyond traditional health outcomes. These include healthcare system capacity, diagnostic and educational infrastructure, behavioural interactions, resource utilisation, financial risk protection, equity, opportunities for future treatments, and macro-level effects such as workforce productivity and environmental sustainability. This expanded perspective aligns with the ISPOR "value flower" framework, which advocates for the inclusion of multiple value domains in economic evaluations. For example, therapies that delay the progression of T1D may contribute to the "value of hope"—the psychological benefit and future-oriented optimism experienced by patients when new treatment options become available.
CONCLUSIONS: Using this multidimensional approach to value assessment, decision-makers can make more informed decisions that reflect the full spectrum of benefits of new health technologies.