New guidance provides framework for health-state utility data collection in clinical studies

Research Triangle Park, NC—The International Society for Pharmacoeconomics and Outcomes Research (ISPOR) Task Force on Good Practices for Outcome Research—Measurement of Health-State Utility Values for Economic Models in Clinical Studies—has published new practice guidance that provides a framework for the collection of health-state utility data in clinical studies.

The report, “Estimating Health-State Utility for Economic Models in Clinical Studies: An ISPOR Good Research Practices Task Force Report,” was recently published  in Value in Health. The report was co-authored by RTI Health Solutions, a business unit of RTI International.

Health care researchers are increasingly trying to incorporate data in clinical trials that demonstrate a health benefit for patients. One of the measurements that can capture this vital information is health-state utility, which reflects the strength of preference for a given health-related outcome in comparison with full health.

“Clinical trials represent a valuable opportunity to gather health utility data. However, because most trials are designed chiefly to collect efficacy and safety data to support market approval (not to collect data for health economic models), an important opportunity to gather health utility data is often missed or problems can arise when applying data that has been collected in cost-utility models,” said Sorrel E. Wolowacz, PhD, RTI Health Solutions Head of European Health Economics and corresponding author.

Health-state utility data provide a measurement of quality-of-life, which is part of a long-term calculation of treatment benefit. Health-state utility data are important inputs in cost-utility models that are used in many countries to establish whether the cost of a new intervention can be justified in terms of health benefits.

Collection of health state utility data within the clinical trial offers a unique opportunity to directly capture key aspects of treatment benefit; however, careful planning is needed to ensure that these important measures are incorporated in the design of the clinical trial to collect high-quality data for economic models.

“Careful planning is needed early in the process to establish a well-thought out design for health-state utility collection in clinical studies,” Wolowacz said. “The Task Force recommendations are intended to help researchers to design the optimum program of utility measurement in clinical studies and to develop a strong scientific rationale for that design.”

Utility Measurement Guidance

The Task Force identified five areas of guidance that will enable researchers to design an optimum utility measurement program within clinical studies. The guidance focused on these specific areas:

1) Early planning of health utility data collection within the research and development program

2) Design of health utility data collection during protocol development for a planned clinical trial

3) Design of prospective and cross-sectional observational studies for estimating health utility

4) Use of alternative study types for health-state utility estimation

5) Statistical analyses and reporting to maximize the value of patient-level health utility data for economic models

Short Course Available at ISPOR European Congress

Sorrel Wolowacz and Lynda Doward will be presenting a new short course at the ISPOR 19th Annual European Congress, Vienna, Austria, along with Prof Andy Briggs, University of Glasgow, and Andrew Lloyd of Bladon Associates.

The course provides an opportunity to review and discuss the content of the guideline, with a view to improving the effectiveness of utility data measurement in clinical studies. The focus will be on optimizing the collection of utility data to provide health state utility estimates for economic models.

Specifically, the course will address key challenges surrounding study design, data collection and analysis. This will include how to anticipate and address common issues that may affect data quality, alignment with the needs of economic model, acceptability to the model audience, and how to apply good research practices for health state utility estimation in future research.

You can register now to attend Collecting Health-State Utility Estimates for Economic Models in Clinical Studies, which will be presented Saturday 29 October.