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Measuring Effectiveness Based on Patient Experience (Instead of QALYs) in US Value Assessments

Maksat Jumamyradov () and Benjamin M. Craig
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Maksat Jumamyradov: University of South Florida
Benjamin M. Craig: University of South Florida

PharmacoEconomics, 2025, vol. 43, issue 2, No 4, 176 pages

Abstract: Abstract Background A key challenge in value assessment is how to summarize effectiveness, particularly the impact of interventions on patient health-related quality of life (HRQoL). One approach is to quantify the gains in HRQoL and life expectancy together as quality-adjusted life years (QALYs); however, this approach has faced various criticisms regarding its potential discriminatory aspects toward persons with disabilities, older adults, and the most vulnerable individuals in society. Methods Instead of QALYs, we provide an alternative approach that summarizes HRQoL gains from the perspective of its stakeholders (e.g., patients, parents, and caregivers) using an “experience” scale. On an experience scale, a positive value signifies an experience better than having no experience at all, while a negative value indicates an experience worse than having no experience. To illustrate the merits of this approach, we examine US preferences on the relief of child health problems, namely a discrete choice experiment (DCE) with kaizen tasks and alternatives described using the EQ-5D-Y-3L. Results Using this approach, we demonstrate the differences in perspectives between parents (N = 179), mothers (N = 99), and fathers (N = 80) of children younger than 18 years of age, as well as the feasibility of this patient-centered approach using a brief DCE survey of less than 100 respondents each (and without QALYs). Specifically, we found that mothers place a higher value on the child’s feelings than fathers. The results also suggest other differences between the perspectives of mothers and fathers, but these differences were not statistically significant (p-values

Date: 2025
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DOI: 10.1007/s40273-024-01444-1

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