Bridging the evidence gap in trial inclusion with real world safety profile assessment in relapsed/refractory multiple myeloma patients
Publisher: BMC Cancer Authors: Rana Jreich, Paul Loustalot, Sacha Levy, Kahina Lounaci, Ruiyi Yuan, Helgi Van de Velde, Zhaoling Meng View Article Background The underrepresentation of key populations in clinical trials can create critical gaps in our understanding of treatment efficacy and the generalizability of safety outcomes across broader patient populations. Ensuring that therapies […]
Impacts du traitement au long cours par lanadelumab sur les dépenses de santé des patients ayant un angioedème héréditaire: Résultats issus de la base nationale française SNDS
Publisher: La Revue de Médecine Interne Authors: Laurence Bouillet; Julien Dauriat; Cinira Lefèvre; Mouna Chebchoub; Diane Vincent; Sacha Lévy; Cédric Strub; Olivier Fain View Article Introduction L’angioœdème héréditaire (AOH) est caractérisée par des crises d’œdème récurrentes, imprévisibles et potentiellement mortelles. Le lanadelumab, un anticorps anti kallicréine, est un traitement prophylactique au long cours (LTP), […]
Patient-reported outcomes and treatment adherence in type 2 diabetes using natural language processing: Wave 8 of the Observational International Diabetes Management Practices Study
Publisher: Journal of Diabetes Investigation Authors: Juliana CN Chan, Jean Claude Mbanya, Jean-Marc Chantelot, Marina Shestakova, Ambady Ramachandran, Hasan Ilkova, Lucille Deplante, Melissa Rollot, Lydie Melas-Melt, Juan Jose Gagliardino, Pablo Aschner View Publication Aims/Introduction We analyzed patient-reported outcomes of people with type 2 diabetes to better understand perceptions and experiences contributing to treatment adherence. Materials and […]
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 […]
Policy makers must adopt agile signal detection tools to strengthen epidemiological surveillance and improve pandemic preparedness
Publisher: Health Policy Authors: Cédric Mahé, Aimo Kannt, AIOLOS Consortium View publication Abstract The SARS-COV2 pandemic has highlighted the urgent need for agile and responsive disease surveillance systems. To strengthen epidemiological surveillance and improve pandemic preparedness, policymakers must adopt real-time signal detection tools that integrate multisource data, including non-traditional health data, advanced analytics, and artificial […]
A text-to-tabular approach to generate synthetic patient data using LLMs
Publisher: IEEE XploreAuthors: Margaux Tornqvist, Jean-Daniel Zucker, Tristan Fauvel, Nicolas Lambert, Mathilde Berthelot, Antoine Movschin View publication Abstract Access to large-scale high-quality healthcare databases is key to accelerate medical research and make insightful discoveries about diseases. However, access to such data is often limited by patient privacy concerns, data sharing restrictions and high costs. To […]
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 […]
Analyzing recurrent events in multiple sclerosis: a review of statistical models with application to the MSOAC database
Publisher: Journal of NeurologyAuthors: David Herman, Julien Tanniou, Emmanuelle Leray, Chloe Pierret, Quentin Pilard View Publication Abstract Patients with multiple sclerosis (MS) are susceptible to experience recurrent events of disability progression and relapses. Many studies still focus on analyzing MS events with traditional methods such as Cox proportional hazards, Poisson, and logistic regression that either […]
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. […]
