Abstract

BackgroundHealth state utility (HSU) is a core component of QALYs and cost-effectiveness analysis, although HSU is rarely estimated among a representative sample of patients. We explored the feasibility of assessing HSU in head and neck cancer from the French National Hospital Discharge database.MethodsAn exhaustive sample of 53,258 incident adult patients with a first diagnosis of head and neck cancer was identified in 2010–2012. We used a cross-sectional approach to define five health states over two periods: three "cancer stages at initial treatment" (early, locally advanced or metastatic stage); a "relapse state" and otherwise a "relapse-free state" in the follow-up of patients initially treated at early or locally advanced stage. In patients admitted in post-acute care, a two-parameter graded response model (Item Response Theory) was estimated from all 144,012 records of six Activities of Daily Living (ADLs) and the latent health state scale underlying ADLs was calibrated with the French EQ-5D-3 L social value set. Following linear interpolation between all assessments of the patient, daily estimates of utility in post-acute care were averaged by health state, patient and month of follow-up. Finally, HSU was estimated by health state and month of follow-up for the whole patient population after controlling for survivorship and selection in post-acute care.ResultsHead and neck cancer was generally associated with poor HSU estimates in a real-life setting. As compared to “distant metastasis at initial treatment”, mean HSU was higher in other health states, although numerical differences were small (0.45 versus around 0.54). It was primarily explained by the negative effects on HSU of an older age (38.4% aged ≥70 years in “early stage at initial treatment”) and comorbidities (> 50% in other health states). HSU estimates significantly improved over time in the “relapse-free state” (from 8 to 12 months of follow-up).ConclusionsHSU estimates in head and neck cancer were primarily driven by age at diagnosis, comorbidities, and time to assessment of cancer survivors. This feasibility study highlights the potential of estimating HSU within and across severe conditions in a systematic way at the national level.

Highlights

  • Health state utility (HSU) is a core component of Quality-Adjusted Life Years (QALYs) and cost-effectiveness analysis, HSU is rarely estimated among a representative sample of patients

  • If the same preference-based, generic health-related quality-of-life (HRQoL) instrument was administered in all patient surveys, all HRQoL profiles of the patients could be converted into HSU estimates with use of country-specific social value sets [4]

  • As compared to the health state “distant metastasis at initial treatment”, other health states were associated with a better mean HSU, numerical differences were small around 0.54. It was primarily explained by the negative effects on HSU of an older age in the health state “early stage at initial treatment” (38.4% patients were aged ≥70 years) and comorbidities (> 50%) in other health states

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Summary

Introduction

Health state utility (HSU) is a core component of QALYs and cost-effectiveness analysis, HSU is rarely estimated among a representative sample of patients. Cost-effectiveness analysis is used in most high-income countries for pricing and reimbursement of new health interventions [1] In such analysis, effectiveness is generally measured by Quality-Adjusted Life Years (QALYs) where the expected number of years to be lived in different health states is weighted by community preferences for each health state [1, 2]. Effectiveness is generally measured by Quality-Adjusted Life Years (QALYs) where the expected number of years to be lived in different health states is weighted by community preferences for each health state [1, 2] These health state utility (HSU) estimates are typically among the most important and uncertain drivers of cost-effectiveness results – a paradoxical situation that seems detrimental to fair pricing and reimbursement decisions across competing new health interventions. The variability of HSU estimates may still remain considerable due to the scarcity, small sample size, and lack of representativeness of patient surveys as recently illustrated in the context of relapsed/metastatic head and neck cancer [5,6,7]

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