Comparative-effectiveness research to aid population decision making by relating clinical outcomes and quality-adjusted life years

Abstract

Background: Comparative-effectiveness research (CER) at the population level is missing standardized approaches to quantify and weigh interventions in terms of their clinical risks, benefits, and uncertainty.

Objectives: We proposed an adapted CER framework for population decision making, provided example displays of the outputs, and discussed the implications for population decision makers.

Methods: Building on decision-analytical modeling but excluding cost, we proposed a 2-step approach to CER that explicitly compared interventions in terms of clinical risks and benefits and linked this evidence to the quality-adjusted life year (QALY). The first step was a traditional intervention-specific evidence synthesis of risks and benefits. The second step was a decision-analytical model to simulate intervention-specific progression of disease over an appropriate time. The output was the ability to compare and quantitatively link clinical outcomes with QALYs.

Conclusions: The outputs from these CER models include clinical risks, benefits, and QALYs over flexible and relevant time horizons. This approach yields an explicit, structured, and consistent quantitative framework to weigh all relevant clinical measures. Population decision makers can use this modeling framework and QALYs to aid in their judgment of the individual and collective risks and benefits of the alternatives over time. Future research should study effective communication of these domains for stakeholders.