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 […]
Artificial Intelligence (AI) ToOLs for Outbreak Detection and response: a transnational platform for surveillance, monitoring and decision support
Event: ISPOR Europe 2023, Copenhagen, Denmark Authors: M. Génin, J. Botz, L. Coudeville, M. Eisenlauer, T. Fauvel, F. Huschka, N. Lambert, D. Wang, V. Bosch Castells, C. Commaille-Chapus, B. Frandji, M. Haberstroh, JY. Robin, P. Roehn, H. Sippel, P. Thiele, E. Thommes, C. Weber, B. Amzal, H. Fröhlich, A. Kannt, C. Mahé View Poster Objectives […]
Machine learned decision tree for diagnosis of ASMD among patients with unexplained Interstitial Lung Disease
Event: European Respiratory Society (ERS) International Congress 2023, Milan, Italy Authors: I. Noth, F. Bonella, W.A. Wuyts,P. Guilmin, M. Törnqvist, S. Sansen, A. Dumitriu, N. Shah, M. Gasparic, M. Montmerle View Poster Background Patients with acid sphingomyelinase deficiency (ASMD), a rare lysosomal storage disease, suffer interstitial lung disease (ILD) as a common clinical manifestation. Patients […]
Enhancing Real-World Evidence with Biomedical Knowledge Graphs: Exploring Atopic Dermatitis Endotypes
Event: ISMB/ECCB 2023, Lyon, France Authors: F. Frau, P. Loustalot, M. Törnqvist, N. Temam, J. Cupe, F. Augé View Poster Background Precision Medicine is defined by the U.S. Food & Drug Administration as “an innovative approach to tailoring disease prevention and treatment that considers differences in people’s genes, environments and lifestyles. Methods To succeed in […]
L’intelligence artificielle au service de la modélisation de maladies en vie réelle : vision, exemples et impact sur l’évaluation des médicaments
Event: Colloque de Données de Santé en vie réelle, AFCROs 2023, Boulogne-Billancourt, France Authors: M. Génin, M. Rollot, A. Movschin, B. Amzal, J. Tanniou View Poster Introduction Le rôle des données de vie réelle (RWD) devient de plus en plus important dans les évaluations réglementaires et HTA, en compléments des essais randomisés (RCT), notamment pour anticiper […]
