As part of the More Europa Work Package 1, the article “Decision-Making Criteria and Methods for Initiating Late-Stage Clinical Trials in Drug Development from a Multi-Stakeholder Perspective: A Scoping Review” has been recently published.
In this paper, Ce Jiang and WP1 colleagues from Quinten Health, Paris Cité University and Radboud UMC offer a comprehensive review of criteria and methods used in decision-making between phase II and phase III trials, with a focus on adopting the perspectives of several stakeholders including pharmaceutical industries, HTA bodies, payers, patient’s advocacy groups, and ethics committees. Recently, the Probability of Success (PoS) has become a central element in determining whether a drug should continue to be assessed in late-stage trials, generally focusing on efficacy and safety endpoints. This paper broadens the scope of PoS beyond efficacy only, exploring different success definitions such as regulatory approval, market access, financial viability, and competitive performance. It complements the manuscript by Cetinyurek-Yavuz et al. (2025), which focuses on efficacy related PoS methodologies.
To support the decision-making process, several quantitative methodologies are used including Bayesian and hybrid frequentist-Bayesian approaches. A significant gap in methodologies that fully incorporate the perspectives of the different stakeholders has been identified. These entities evaluate the drug development process through distinct lenses—such as reimbursement, pricing and patient access—that often are not adequately represented in existing decision frameworks. Although the methodologies were primarily designed to assess some endpoints on clinical trials data, the selected articles stress the importance of integrating real-world data (RWD) and highlight that current approaches largely overlook these valuable data sources, despite their potential to improve the decision-making process.
Key findings of the review:
This study enhances the understanding of current PoS methodologies and paves the way for a more balanced and stakeholder-aligned approach to late-stage trial decisions, ultimately enhancing the quality of future decision-making and the success of drug development programs. The findings aim to promote more inclusive, data-driven and adaptive decision-making practices, supporting the development of safer and more effective treatments by integrating real-world data into the decision-making process.
