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 […]
AI-Driven Disease and Care Modeling and Simulation: A Framework for Simulating Real-World Scenarios in Non-Small Cell Lung Cancer (NSCLC)
Event: ELCC 2025, Paris, France Authors: Antoine Movschin, Lise Bosquet, David Pérol, Melissa Rollot, Louise Dry, Coriande Clemente, Farah Al Nakib, Mathilde Berthelot, Margaux Törnqvist View Poster Background Advances in personalized medicine have driven targeted therapy for NSCLC through mutation identification, but their rarity in real-world (RW) settings limits traditional RCTs. External control arms (ECAs) offer an […]
Identification of risk profiles for liver injury in adults with multiple sclerosis using artificial intelligence method
Event: The Liver Meeting – AASLD (American Association for the Study of Liver Diseases) 2024, San Diego, California, USA Authors: Dominique Larrey, Fang Liz Zhou, Claire Brulle-Wohlhueter, Myriam Benamor, Jeffrey Chavin, Neda Razaz, Raphael Bejuit, Julien Dauriat, Théophile Reppelin, Romane Péan, Sam Ekhtiari, Nicolas Wagner, Fred Lublin View Poster Background The evolution of multiple sclerosis (MS) […]
Identification of risk profiles for liver injury in adults with multiple sclerosis using artificial intelligence method
Authors: Dominique Larrey, Fang Liz Zhou, Claire Brulle-Wohlhueter, Myriam Benamor, Jeffrey Chavin, Neda Razaz, Raphael Bejuit, Julien Dauriat, Théophile Reppelin, Romane Péan, Sam Ekhtiari, Nicolas Wagner, Fred Lublin Event: Congrès AFEF (Association Française pour l’Étude du Foie) View Poster Introduction et Objectif L’évolution du traitement de la sclérose en plaques (SEP) au cours des deux dernières […]
Analyzing Recurrent Events in Multiple Sclerosis: A Review of Statistical Models with Application to MSOAC Trial
Event: ECTRIMS 2024, Copenhagen, Denmark Authors: D. Herman, J. Tanniou, N. Lambert, P. Loustalot, Q. Pilard View Poster Introduction In Multiple Sclerosis (MS), patients are likely to face repeated event such as confirmed disability progression (CDP). However, clinical trials often focus on analyzing time to first events, ignoring subsequent events, which are of clinical importance, despite the availability of statistical […]
Tailored diagnostic decision tree resulting from machine learning to improve early diagnosis of ASMD
Event: WORLDSymposium 2024 San Diego, USA Authors: M. Domenica Cappellini, R. Giugliani, M. Törnqvist, P. Guilmin, C.Clémente, M. Montmerle, A. Chiorean, T. Reppelin, Stefaan Sansen, Alexandra Dumitriu, Neha Shah, Maja Gasparic View Poster Abstract Acid sphingomyelinase deficiency (ASMD) is a rare and debilitating lysosomal storage disease and delays in diagnosis are common. We employed machine learning (ML) on electronic health […]
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 […]
Criteria and methods applied to decision-making to embark on late-stage clinical trials with a multi-stakeholder perspective
Event: ISPOR Europe 2023, Copenhagen, Denmark Authors: C. Jiang, J. Tanniou, B. Amzal View Poster Objectives The More-EUROPA project aims at establishing the value of registry-based RWD in enhancing RCT data and enabling a more effective and ethical use of registry data to support patient-centred regulatory and HTA decision-making. Work package 1 (WP1) is specifically […]
Existing automated tools to assist evidence generation and better qualification of registries and real-world data. A systematic review from the More-EUROPA project
Event: ISPOR Europe 2023, Copenhagen, Denmark Authors: P. Godbillot, P. Tang, J. Moreira, J. Tanniou S. Zebachi, B. Amzal View Poster Objectives The More-EUROPA project consortium brings together 14 public and private entities from 7 countries with the aim of enhancing the ethical and effective utilization of registry data to facilitate patient-centered decision-making by drug regulators and Health Technology Assessment […]
