An AI powered tool to identify and assess fit-for use registries for real-world evidence
Event: EuroDURG 2025, Uppsala, Sweden Authors: Ghinwa Y. Hayek, Pascal Godbillot, Coriande Clemente, Sonia Zebachi, Gaëtan Pinon, Boris Kopin, Elisabeth Bakker, Sieta T. de Vries, Peter G.M. Mol, Billy Amzal View publication Abstract The selection of appropriate real-world data (RWD) sources, particularly registries, is of primary concern for academia, industry, regulators, and health technology assessment […]
Navigating the Real World: A Scoping Review of Structured Frameworks to Effectively Identify, Evaluate, and Select Real-World Data Sources for Fit-for-Purpose Studies
Publisher: Clinical Pharmacology and Therapeutics Authors: Sonia Zebachi, Julien Tanniou, Elisabeth Bakker, Sieta T. de Vries, Rossella Di Bidino, Entela Xoxi, Anna Glaser, Gianluigi Savarese, Jan Hillert, Peter G.M. Mol, Kelly Plueschke, Billy Amzal, Ghinwa Y. Hayek, Jeverson Moreira View publication Abstract The potential of real-world data (RWD), particularly from patient registries, has been increasingly recognized […]
Navigating the Real World: A Scoping Review of Structured Frameworks to Effectively Identify, Evaluate, and Select Real-World Data Sources for Fit-for-Purpose Studies
Event: EuroDURG 2025, Uppsala, Sweden Authors: Sonia Zebachi, Julien Tanniou, Elisabeth Bakker, Sieta T. de Vries, Rossella di Bidino, Entela Xoxi, Anna Glaser, Gianluigi Savarese, Jan Hillert, Peter G.M. Mol, Kelly Plueschke, Ghinwa Hayek, Billy Amzal, Jeverson Moreira View Poster The potential of real-world data (RWD), particularly from patient registries, has been increasingly recognised over […]
Decision-Making Criteria and Methods for Initiating Late-Stage Clinical Trials in Drug Development from a Multi-Stakeholder Perspective: A Scoping Review
Event: PSI 2025, Verona, Italy Authors: Ce Jiang, Céline Beji, Sonia Zebachi, Ghinwa Y. Hayek, Aysun Cetinyurek-Yavuz, Muhammad Bergas N. Fayyad, Laura Rodwell, Kit C. B. Roes, Billy Amzal, Christoph Gerlinger, Raphaël Porcher, Julien Tanniou View Abstract The decision-making process in drug development involves “go/no-go” decisions, particularly at the transition from early to late-stages trials. While the decisions are solely made by drug developers, they must […]
Federated Learning: A Privacy-Preserving Approach to Data-Centric Regulatory Cooperation
Publisher: Frontiers in Drug Safety and RegulationAuthors: Alexander Horst, Paul Loustalot, Sanjeev Yoganathan, Ting Li, Joshua Xu, Weida Tong, David Schneider, Nicolas Löffler-Perez, Erminio Di Renzo, Michael Renaudin View Publication Abstract Regulatory agencies aim to ensure the safety and efficacy of medical products but often face legal and privacy concerns that hinder collaboration at the […]
Comparison of Bayesian methods for extrapolation of treatment effects: a large scale simulation study
Publisher: arXivAuthors: Tristan Fauvel, Julien Tanniou, Pascal Godbillot, Billy Amzal View Publication Abstract Extrapolating treatment effects from related studies is a promising strategy for designing and analyzing clinical trials in situations where achieving an adequate sample size is challenging. Bayesian methods are well-suited for this purpose, as they enable the synthesis of prior information through the use of […]
Decision-Making Criteria and Methods for Initiating Late-Stage Clinical Trials in Drug Development from a Multi-Stakeholder Perspective: A Scoping Review
Publisher: Clinical Pharmacology and Therapeutics Authors: Ce Jiang, Céline Beji, Sonia Zebachi, Ghinwa Y. Hayek, Aysun Cetinyurek-Yavuz, Muhammad Bergas N. Fayyad, Laura Rodwell, Kit C. B. Roes, Billy Amzal, Christoph Gerlinger, Raphaël Porcher, Julien Tanniou View Publication The decision-making process in drug development involves “go/no-go” decisions, particularly at the transition from early to late-stages trials. […]
On the Concepts, Methods, and Use of “Probability of Success” for Drug Development Decision-Making: A Scoping Review
Publisher: Clinical Pharmacology and Therapeutics Authors: Aysun Cetinyurek Yavuz, Muhammad Bergas Nur Fayyad, Ce Jiang, Florie Brion Bouvier, Celine Beji, Sonia Zebachi, Ghinwa Y. Hayek, Billy Amzal, Raphael Porcher, Julien Tanniou, Kit Roes, Laura Rodwell View publication Abstract Drug development is a lengthy process with considerable uncertainty at each milestone. Several trials are needed to progress […]
Existing practices for the identification, selection or assessment of registries for regulatory/HTA purposes: a More-EUROPA project systematic review
Event: ISPOR Europe 2023, Copenhagen, Denmark Authors: S. Zebachi, J. Moreira, J. Tanniou, P. Godbillot, P. Tang, B. Amzal View Poster Objectives Real-world data (RWD), particularly patient registries, represent a potential useful source of information and may be required at various stages of the drug lifecycle. Different questions may be asked throughout the regulatory and/or […]
What are unmet needs in the use of registries in regulatory/HTA decision-making in Europe? A survey-based approach from the More-EUROPA project
Event: ISPOR Europe 2023, Copenhagen, Denmark Authors: J. Moreira, J. Tanniou, P. Tang, P. Godbillot, F. Ahrens, B. Amzal View Poster Objectives The More-EUROPA project, involving 14 public and private organizations from 7 EU countries, evaluates the effective and ethical use of registry data to support patient-centered decisions by drug regulators and Health Technology Assessment (HTA) agencies in Europe. Specifically, one […]
