Putting the Patient in Patient Reported Outcomes: A Robust Methodology for Health Outcomes Assessment

Abstract

This paper proposes a novel two-stage approach to modeling health-related quality-of-life (HRQoL) outcomes. The approach allows a patient-centered interpretation by estimating regression coefficients based on the multi-dimensional HRQoL profile, but maintains the parsimony of the quality-adjusted life-year (QALY) framework by re-interpreting incremental effects in the QALY domain. Through a series of Monte Carlo simulations, the proposed methodology is shown to be more robust to the distributional properties of QALYs, thereby avoiding the potential bias introduced when converting the multi-dimensional HRQoL into a one-dimensional QALY. The approach also allows researchers to more easily update cost- and comparative-effectiveness results as new HRQoL scoring algorithms develop for new patient populations. I provide an application to the estimation of incremental effects of demographic and clinical variables on HRQoL following surgical treatment for adult scoliosis and spinal deformity patients.

Keywords: cost-effectiveness, comparative-effectiveness, patient-reported outcome measures, quality-adjusted life years, scoliosis, adult spinal deformity

JEL Classification: I10, C24, C25, C34, C35, C51