Benefit-Risk Balance for Medicinal Products: Report Summary from the Council for International Organizations of Medical Sciences (CIOMS) Working Group XII.

Abstract

Benefit-risk assessment (BRA) is a foundation of regulatory decision making and clinical practice for medicinal products. Although historically assessments relied on expert judgment and narrative summaries, over time, expectations have shifted toward approaches that are more structured, transparent, and patient centered. At the same time, therapeutic innovation, new data sources, and evolving stakeholder roles have increased the complexity and uncertainty of benefit-risk decision making. The Council for International Organizations of Medical Sciences (CIOMS) Working Group XII developed updated consensus guidance for BRA through multidisciplinary international collaboration among regulators, industry, and academia. This narrative review summarizes the CIOMS Working Group XII consensus framework, which advances a structured, transparent, and life-cycle based approach to BRA. The framework puts forward patient‑centric approaches by moving benefit-risk evaluation from a post‑hoc exercise to integrated assessments that enable holistic patient-level BRAs. The structured benefit-risk framework harmonizes qualitative and quantitative methods, integrates patient perspectives, and emphasizes transparency in communication. Ultimately, the CIOMS Working Group XII consensus report advances the science and practice of BRAs, ensuring that medicinal products are assessed in a structured and systematic format from early development throughout the lifecycle. By embedding structured, patient-centered BRA into lifecycle management, the CIOMS Working Group XII report provides a roadmap for transparent, evidence-based decisions that safeguard public health and optimize therapeutic outcomes.