Is there a need for a universal benefit–risk assessment framework for medicines? Regulatory and industry perspectives

Abstract

Stakeholders prefer a semi-quantitative, overarching framework that incorporates a toolbox of different methodologies. A coordinating committee of relevant stakeholders should be formed to guide its development and implementation. Through engaging the stakeholders, these outcomes confirm sentiments and need for developing a universal benefit-risk assessment framework.

Pragmatic Multicriteria Decision Analysis (MCDA) Combined With Advanced Pharmacoepidemiology for Benefit-Risk Assessments of Medicines Adapted to the Real-Life Constraints of Regulators: Development and Case Study

“…As there were no head-to-head trials comparing active treatments, uncertainty about comparative benefits was also significant. This was reflected in the better reproducibility of panelists' scores for efalizumab versus placebo, which were based on RCTs (ICC [3,1] ¼ 0.978) compared to scores that were informed by indirect comparisons (ICC [3,1]¼0.800).…”

“…3 To address this issue, the MCDA evidence matrix was developed as basis for benefitrisk assessment. The content of this matrix aims at providing sufficient and necessary information for each decision criterion to facilitate understanding and interpretation of the available evidence.…”

“…Although none of the agencies reported currently using MCDA, it stood out as a relevant and useful methodology for future development of a universal benefit-risk framework. 3 The Pharmacoepidemiological Research on Outcomes of Therapeutics by a European Consortium (PROTECT) is a collaborative project, coordinated by the European Medicines Agency (EMA), that aims at addressing limitations in the field of pharmacoepidemiology and pharmacovigilance. PROTECT's Work Package (WP) 5 reviewed, tested, and compared a range of benefit-risk assessment methods for regulatory decision making regarding marketing authorization and safe and effective use of medicines.…”

Benefit–risk assessment and public communication

“…The most commonly used methodology was the qualitative approach, followed by number needed to treat (NNT) and number needed to harm (NNH), evidence-based benefi t-risk models, decision trees or infl uence/relevance diagrams, and Kaplan-Meier estimators. Nearly all of the companies but none of the agencies were using benefi t-risk visualisation tools to communicate assessments, mainly for internal purposes (Leong et al, 2013).…”

Structured Benefit–risk assessment: a review of key publications and initiatives on frameworks and methodologies

“…The final decision will be exercised based on Expert Judgment. 15 Quantitative The system is a fully quantitative model which includes a benefit-risk balance for a new medicine, and is applied across study data and contributing opinions. The conclusion is based on the cumulative outcome from this single system.…”