Global Expert Panel Publishes New Guidance on Designing, Conducting, and Analyzing Quantitative Benefit-Risk Assessment

Published Apr 11, 2023

A Good Practices Report of an ISPOR Task Force

Value in HealthLawrenceville, NJ, USA—April 11, 2023—Value in Health, the official journal of ISPOR—The Professional Society for Health Economics and Outcomes Research, announced today the publication of new good practice guidelines for the design, conduct, analysis, and communication of quantitative benefit-risk assessments (qBRA) to support regulatory decision making of medical products. The report, “Quantitative Benefit-Risk Assessment in Medical Product Decision Making: A Good Practices Report of an ISPOR Task Force,” was published in the April 2023 issue of Value in Health.

“While the benefits of many medical products clearly outweigh their risks, sometimes the benefit-risk balance is difficult to establish using solely qualitative clinical judgement. In those cases, quantitative benefit-risk assessment can provide additional insights that are invaluable for decision making,” said the report’s lead author Tommi Tervonen, PhD, chief scientist at Kielo Research in Switzerland. “By following this new guidance, analysts can develop robust and fit-for-purpose benefit-risk assessments that appropriately reflect the values of patients or other stakeholders.”

“Various frameworks and tools have already focused on the qualitative aspect of benefit-risk assessments of medical products,” noted task force co-lead, Martin Ho, MS, former associate director at the Center for Biologics Evaluation and Research and the Center for Devices and Radiological Health of the US Food and Drug Administration. “As a perfect supplement to those existing frameworks, this new report addresses the quantitative aspect of benefit-risk assessment of medical products: it provides a set of pragmatic and actionable steps for developing high-quality studies that incorporate explicit decision weights. Thanks to formal modeling and input parameters, qBRA is also an effective communication tool for discussing complex benefit-risk cases in a transparent manner between different stakeholders. This report provides an indispensable roadmap of qBRA—for regulators, researchers, and developers of medical products—that ultimately benefits patients.”

The report describes good practices of developing quantitative benefit-risk assessment with the following 5 core steps:

  1. Research Question: The research question needs to identify the needs of decision makers, characterize the requirements for preference data, and specify the role of external experts.
  2. Model Development: The formal analysis model should be developed by selecting benefit and safety endpoints while eliminating double counting and considering attribute value dependence.
  3. Preference Elicitation: The reference elicitation method needs to be chosen, attributes should be framed appropriately within the elicitation instrument, and quality of the data should be evaluated.
  4. Analysis: Analysis may need to normalize the preference weights, base-case and sensitivity analyses should be conducted, and the effect of preference heterogeneity should be analyzed.
  5. Communication of Results: Results should be communicated efficiently to decision makers and other stakeholders.

In addition to the detailed and actionable recommendations of these individual steps, the report also provides a useful checklist for reporting quantitative benefit-risk assessments. The task force believes that the checklist can be a useful tool not only for improving the transparency of qBRA studies, but also for preparing and submitting manuscripts for publication.

The good practice guidelines are demonstrated in a case study developed by members of the task force, “Illustrating Emerging Good Practices for Quantitative Benefit-Risk Assessment: A Hypothetical Case Study of Systemic Biologic Treatments for Plaque Psoriasis”, published in the same issue of Value in Health.

The report was accompanied by an editorial from Salah Ghabri, MSc, PhD, HDR, Haute Autorité de Santé, Paris, France. His editorial, “Emerging Good Practices for Quantitative Benefit-Risk Assessment: A Step Forward,” also published in the April 2023 issue of Value in Health, provides important background and context for those interested in qBRA. “This ISPOR Good Practices Report enriches the literature on benefit-risk assessment in several ways,” said Ghabri. “First, it includes explicit outcome weighting on the importance of desirable and undesirable outcomes, rather than the qualitative judgment of current frameworks. Second, its recommendations were developed by an international multistakeholder team of experts assembled to address this specific gap in qBRA guidance. Finally, it proposes options to handle issues related to the interpretation of decisions based on a qualitative framework that are preference sensitive.”

The editorial notes that, “Successful implementation of the qBRA necessitates both sufficient knowledge of the dimensions influencing the decision making and handling of many practical constraints.” Ghabri concludes that the task force recommendations on qBRA “are timely and enrich the methodological literature while also complementing qualitative and existing regulatory benefit-risk approaches, thanks to a structured analytical process with detailed steps.”

This is ISPOR’s 5th patient preference methods good practices task force report. To view these reports, please click here


ISPOR, the professional society for health economics and outcomes research (HEOR), is an international, multistakeholder, nonprofit dedicated to advancing HEOR excellence to improve decision making for health globally. The Society is the leading source for scientific conferences, peer-reviewed and MEDLINE®-indexed publications, good practices guidance, education, collaboration, and tools/resources in the field.
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Value in Health (ISSN 1098-3015) is an international, indexed journal that publishes original research and health policy articles that advance the field of health economics and outcomes research to help healthcare leaders make evidence-based decisions. The journal’s 2021 impact factor score is 5.156 and its 5-year impact factor score is 6.779. Value in Health is ranked 9th of 88 journals in health policy and services, 18th of 109 journals in healthcare sciences and services, and 50th of 381 journals in economics. Value in Health is a monthly publication that circulates to more than 10,000 readers around the world.
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ISPOR has earned an international reputation for research excellence based, in part, on its Good Practices Reports. These highly cited reports are expert consensus recommendations on good practice standards for outcomes research (clinical, economic, and patient-reported outcomes) and on the use of this research in healthcare decision making. ISPOR Task Forces comprise subject matter experts representing different stakeholders from diverse work environments (ie, regulators, payers, manufacturers, technology assessors, etc from research, government, academic, and industry sectors around the world). All ISPOR Good Practices Reports are published in the Society’s scientific journal, Value in Health, and are made freely available as part of the Society’s mission. The Society’s Good Practices Reports have been recognized with an ASAE “Power of A” award that acknowledges innovative, effective, and broad-reaching programs that have a positive impact on the world.

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