From Clinical Outcomes to Health Utilities: The Role of Mapping to Bridge the Evidence Gap

Abstract

The assessment of health-related quality of life is crucially important in the evaluation of healthcare technologies and services. In many countries, economic evaluation plays a prominent role in informing decision making often requiring preference-based measures (PBMs) to assess quality of life. These measures comprise two aspects: a descriptive system where patients can indicate the impact of ill health, and a value set based on the preferences of individuals for each of the health states that can be described. These values are required for the calculation of quality adjusted life years (QALYs), the measure for health benefit used in the vast majority of economic evaluations. The National Institute for Health and Care Excellence (NICE) has used cost per QALY as its preferred framework for economic evaluation of healthcare technologies since its inception in 1999. However, there is often an evidence gap between the clinical measures that are available from clinical studies on the effect of a specific health technology and the PBMs needed to construct QALY measures. Instruments such as the EQ-5D have preference-based scoring systems and are favored by organizations such as NICE but are frequently absent from clinical studies of treatment effect. Even where a PBM is included this may still be insufficient for the needs of the economic evaluation. Trials may have insufficient follow-up, be underpowered to detect relevant events, or include the wrong PBM for the decision- making body. Often this gap is bridged by “mapping”—estimating a relationship between observed clinical outcomes and PBMs, using data from a reference dataset containing both types of information. The estimated statistical model can then be used to predict what the PBM would have been in the clinical study given the available information. There are two approaches to mapping linked to the structure of a PBM. The indirect approach (or response mapping) models the responses to the descriptive system using discrete data models. The expected health utility is calculated as a subsequent step using the estimated probability distribution of health states. The second approach (the direct approach) models the health state utility values directly. Statistical models routinely used in the past for mapping are unable to consider the idiosyncrasies of health utility data. Often they do not work well in practice and can give seriously biased estimates of the value of treatments. Although the bias could, in principle, go in any direction, in practice it tends to result in underestimation of cost effectiveness and consequently distorted funding decisions. This has real effects on patients, clinicians, industry, and the general public. These problems have led some analysts to mistakenly conclude that mapping always induces biases and should be avoided. However, the development and use of more appropriate models has refuted this claim. The need to improve the quality of mapping studies led to the formation of the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) Mapping to Estimate Health State Utility values from Non-Preference-Based Outcome Measures Task Force to develop good practice guidance in mapping.