Publications

Analysis of the challenges in ensuring robust and effective clinical evaluation of AI-based digital medical devices

S Boulet, V Thévenet, J Abecassis, C Collignon, B Giraudeau, E Lhomme, ... BMJ digital health & AI 2 (1), e000043, 2026

Eliciting clinical trial estimands for cystic fibrosis: a Bayesian framework

W Shin, M Ursino, D Muilwijk, CK van der Ent, I Huys, S Zohar Journal of Cystic Fibrosis 25, S118, 2026

Conditional bootstrap for non-linear mixed effects models

S Kaisaridi, M Ursino, E Comets arXiv preprint arXiv:2605.02534, 2026

Evaluation of the European Union Health Technology Assessment landscape and suggestion of tools to assess AI-based digital medical devices: Analyse de l’évaluation des …

S Boulet, E Tavares, Y Binois, J Abecassis, M Boers, C Collignon, ... Journal of Epidemiology and Population Health 74, 203417, 2026

Comparison of two modelling approaches for two-population phase I/II dose-finding trials: Comparaison de deux approches de modélisation pour des essais de phase I/II de …

J Meyring, M Ursino, S Chevret, L Biard Journal of Epidemiology and Population Health 74, 203404, 2026

Clinical trial evaluation of digital medical devices for clinical decision support: a simulation study: Évaluation en essai clinique des dispositifs médicaux numériques d’aide …

B Archambaud, S Boulet, M Ursino, R Thiébaut, S Zohar Journal of Epidemiology and Population Health 74, 203415, 2026

When should the next clinical trial be conducted? A Bayesian borrowing approach for AI-based digital medical devices: Quand initier le prochain essai clinique? Une approche …

C Lawless, S Boulet, M Ursino, R Thiébaut, S Zohar Journal of Epidemiology and Population Health 74, 203416, 2026

Innovative Clinical Trial Approach for Evaluating Digital Medical Devices Under European Fast‐Track Regulatory Frameworks

M Ursino, S Boulet, C Collignon, F Francis‐Oliviero, E Lhomme, ... Statistics in Medicine 45 (10-12), e70572, 2026

A comparative analysis of Phase I dose-finding designs incorporating pharmacokinetics information

A Vuorinen, E Comets, M Ursino The American Statistician 80 (2), 286-300, 2026

Prophylactic treatment of patent ductus arteriosus with acetaminophen: a randomized clinical trial

JC Rozé, G Cambonie, C Flamant, J Patkaï, T Mühlbacher, G Gascoin, ... JAMA pediatrics 180 (4), 374-383, 2026

In silico clinical trials in drug development: a systematic review

B Chen, LC Schneider, C Röver, E Comets, MC Elze, A Hooker, J IntHout, ... Therapeutic Innovation & Regulatory Science 60 (2), 423-439, 2026

Latent Neural-ODE for Model-Informed Precision Dosing: Overcoming Structural Assumptions in Pharmacokinetics

B Maurel, A Guilloux, S Zohar, M Ursino, JB Woillard arXiv preprint arXiv:2602.03215, 2026

A novel workflow integrating whole-body PET microdosing data and therapeutic-dose pharmacokinetics across species to inform first-in-human dose selection

BT Nguyen, A Barrail-Tran, M Ursino, S Goutal, F Caillé, T Naninck, ... bioRxiv, 2026.09. 03.748870, 2026

Sample size reassessment in Bayesian hybrid clinical trials

M Ratta, P Mozgunov, S Boulet, M Ursino arXiv preprint arXiv:2512.19853, 2025

U-DESPE: a Bayesian Utility-based methodology for dosing regimen optimization in early-phase oncology trials based on Dose-Exposure, Safety, Pharmacodynamics, Efficacy

A Andrillon, S Micallef, M Ursino, P Mozgunov, MK Riviere arXiv preprint arXiv:2511.17376, 2025

Modeling Whole-Body Dynamic PET Microdosing Data to Predict the Whole-Body Pharmacokinetics of Glyburide in Humans: L. Comin et al.

L Comin, S Marie, M Ursino, S Zohar, N Tournier, E Comets Clinical Pharmacokinetics 64 (11), 1709-1722, 2025

Narrative review on the clinical evaluation of AI-based digital medical devices from a methodological perspective

S Boulet, V Thévenet, J Abécassis, C Collignon, B Giraudeau, E Lhomme, ... SbN 2025-10th Statistics & Biopharmacy Conference, 2025

Digital pharmacological twins: Bridging multi-scale modelling and artificial intelligence for precision medicine: The DIGPHAT consortium

JB Woillard, S Benzekry, J Josse, M White-Koning, E Chatelut, E Comets, ... Therapies, 2025

Modelling, Extrapolation and Simulation approaches for rare diseases-Review of two case-studies in the INVENTS project

E Comets, M Ursino, Invents WP1. 1 group medRxiv, 2025.09. 17.25335979, 2025

Straightforward Phase I Dose-Finding Design for Healthy Volunteers Accounting for Surrogate Activity Biomarkers

S Boulet, E Comets, A Guillon, LBS Aulin, R Michelet, C Kloft, S Zohar, ... Statistics in Biopharmaceutical Research 17 (3), 496-505, 2025

Effects of the right to be forgotten for childhood or breast cancer survivors: results of a quasi-experimental study in France

N Bougas, TS Dabakuyo Yonli, E Assogba, N Haddy, F De Vathaire, ... Journal of Cancer Survivorship, 1-11, 2025

A seamless hybrid phase II/III design with bayesian interim subgroup selection

B Duputel, N Stallard, F Montestruc, S Zohar, M Ursino Statistics in Medicine 44 (13-14), e70144, 2025

Stress Management in UHR Individuals: The STAR-T Randomised Controlled Trial

V Lucarini, L Petersen, C Jantac, M Ursino, A Mansoux, M Charre, ... EARLY INTERVENTION IN PSYCHIATRY 19, 2025

P02-Innovative clinical trial approach for evaluating digital medical devices under European fast-track regulatory frameworks

M Ursino, S Boulet, R Porcher, E Lhomme, F Francis-Oliviero, ... Journal of Epidemiology and Population Health 73, 203032, 2025

Modelling the whole-body pharmacokinetics of radiolabeled glyburide in healthy volunteers

L Comin, S Marie, M Ursino, S Zohar, N Tournier, E Comets EMIM 2025-20th European Molecular Imaging Meeting, 2025

Modélisation de la pharmacocinétique sur corps entier (WBPK) de microdose du glyburide radiomarqué (11C-glyburide) chez des volontaires sains

L Comin, S Marie, M Ursino, S Zohar, N Tournier, E Comets Médecine Nucléaire 49 (2), 135, 2025

TREOCAPA: prophylactic treatment of the ductus arteriosus in preterm infants by acetaminophen—statistical analysis plan for the randomized phase III group sequential trial

M Ursino, C Alberti, G Cambonie, R Kemp, A Vanhecke, L Levoyer, ... Trials 26 (1), 52, 2025

SPIRIT-DEFINE explanation and elaboration: recommendations for enhancing quality and impact of early phase dose-finding clinical trials protocols

M Ursino, G Villacampa, J Rekowski, M Dimairo, O Solovyeva, D Ashby, ... EClinicalMedicine 79, 2025

CONSORT-DEFINE explanation and elaboration: recommendations for enhancing reporting quality and impact of early phase dose-finding clinical trials

J Rekowski, C Guo, O Solovyeva, M Dimairo, M Rouhifard, D Patel, ... EClinicalMedicine 79, 2025

Meta‐analyses of phase I dose‐finding studies: Application for the development of protein kinase inhibitors in oncology

L Caquelin, P Badra, L Poulain, B Laviolle, M Ursino, C Locher Research Synthesis Methods 15 (6), 964-977, 2024

Home-Based Connected Devices Combined With Statistical Process Control for the Early Detection of Respiratory Exacerbations by Patients With Cystic Fibrosis: Pilot …

E Le Roux, M Ursino, I Milovanovic, P Picq, J Haignere, G Rault, ... JMIR Formative Research 8, e51753, 2024

Survival without severe neonatal morbidity after antenatal betamethasone dose reduction: a post hoc analysis of a randomized non-inferiority trial

O Baud, L Sentilhes, M Ursino, M Doret-Dion, C Alberti, C Aupiais, ... American Journal of Obstetrics and Gynecology 231 (4), 458. e1-458. e16, 2024

Enhancing Clinical Development-Bayesian Methods and Mathematical Modeling for Leveraging Available Information

M Ursino Univerité Paris Cité, 2024

Modelling the whole-body pharmacokinetics (WBPK) of radiolabeled glyburide in healthy volunteers

L Comin, S Marie, M Ursino, S Zohar, N Tournier, E Comets PAGE meeting 2024-Thirty-second meeting, 2024

Coping with information loss and the use of auxiliary sources of data: A report from the NISS Ingram Olkin forum series on unplanned clinical trial disruptions

S Calderazzo, S Tarima, C Reid, N Flournoy, T Friede, N Geller, ... Statistics in Biopharmaceutical Research 16 (2), 141-157, 2024

Bayesian framework for multi-source data integration-Application to human extrapolation from preclinical studies

S Boulet, M Ursino, R Michelet, LBS Aulin, C Kloft, E Comets, S Zohar Statistical methods in medical research 33 (4), 574-588, 2024

Enhancing reporting quality and impact of early phase dose-finding clinical trials: CONSORT Dose-finding Extension (CONSORT-DEFINE) guidance

C Yap, O Solovyeva, J de Bono, J Rekowski, D Patel, T Jaki, A Mander, ... bmj 383, 2023

Enhancing quality and impact of early phase dose-finding clinical trial protocols: SPIRIT Dose-finding Extension (SPIRIT-DEFINE) guidance

C Yap, J Rekowski, M Ursino, O Solovyeva, D Patel, M Dimairo, CJ Weir, ... bmj 383, 2023

Development of consensus-driven SPIRIT and CONSORT extensions for early phase dose-finding trials: the DEFINE study

O Solovyeva, M Dimairo, CJ Weir, SW Hee, A Espinasse, M Ursino, ... BMC medicine 21 (1), 246, 2023

P163-Meta-analysis of phase I dose-finding studies: application for protein kinase inhibitors developed in oncology

L Caquelin, P Badra, B Laviolle, M Ursino, C Locher Revue d'Épidémiologie et de Santé Publique 71, 101816, 2023

Using dichotomized survival data to construct a prior distribution for a Bayesian seamless Phase II/III clinical trial

B Duputel, N Stallard, F Montestruc, S Zohar, M Ursino Statistical Methods in Medical Research 32 (5), 963-977, 2023

Development process of consensus-driven SPIRIT and CONSORT extensions for early phase dose-finding trials: the DEFINE (DosE FIndiNg Extensions) study

O Solovyeva, M Dimairo, C Weir, S Hee, M Ursino, A Espinasse, D Patel, ... OSF, 2023

Implementation of a centralized pharmacovigilance system in academic pan‐European clinical trials: Experience from EU‐Response and conect4children consortia

V Terzić, L Levoyer, M Figarella, E Bigagli, N Mercier, L De Gastines, ... British Journal of Clinical Pharmacology 89 (4), 1318-1328, 2023

79MO Developing international consensus-driven SPIRIT and CONSORT extensions for early phase dose-finding clinical trials: The DEFINE (DosE FIndiNg Extensions) study

O Solovyeva, M Dimairo, C Weir, JS de Bono, A Bedding, AW Chan, ... ESMO Open 8 (1), 2023

Devenir néonatal à la sortie de l’hôpital après une réduction de 50% de la dose de bétaméthasone prénatale: analyse post-hoc de l’essai BETADOSE

T Schmitz, L Sentilhes, M Doret-Dion, C Alberti, M Ursino, C Aupiais, ... Perfectionnement en Pédiatrie 6 (1), 85, 2023

Postoperative analgesia after surgical repair of distal radius fracture: a randomized comparison between distal peripheral nerve blockade and surgical site infiltration.

D Sciard, K Xu, A Soulier, G Dufour, M Ursino, C Alberti, M Beaussier Minerva anestesiologica 89 (10), 876-883, 2023

Riluzole for treating spasticity in patients with chronic traumatic spinal cord injury: study protocol in the phase ib/iib adaptive multicenter randomized controlled RILUSCI trial

M Cotinat, I Boquet, M Ursino, C Brocard, E Jouve, C Alberti, ... PloS one 18 (1), e0276892, 2023

DICE: A Bayesian model for early dose finding in phase I trials with multiple treatment courses

M Ursino, L Biard, S Chevret Biometrical Journal 64 (8), 1486-1497, 2022

A straightforward meta‐analysis approach for oncology phase I dose‐finding studies

C Röver, M Ursino, T Friede, S Zohar Statistics in Medicine 41 (20), 3915-3940, 2022

Neonatal outcomes for women at risk of preterm delivery given half dose versus full dose of antenatal betamethasone: a randomised, multicentre, double-blind, placebo-controlled …

T Schmitz, M Doret-Dion, L Sentilhes, O Parant, O Claris, L Renesme, ... The Lancet 400 (10352), 592-604, 2022

Bayesian framework for extrapolation from multi-source preclinical data to human

S Boulet, M Ursino, R Michelet, LB Aulin, C Kloft, E Comets, S Zohar ISBA 2022-World Meeting International Society for Bayesian Analysis, 2022

Bayesian dose regimen assessment in early phase oncology incorporating pharmacokinetics and pharmacodynamics

E Gerard, S Zohar, HT Thai, C Lorenzato, MK Riviere, M Ursino Biometrics 78 (1), 300-312, 2022

Bayesian modeling of a bivariate toxicity outcome for early phase oncology trials evaluating dose regimens

E Gerard, S Zohar, C Lorenzato, M Ursino, M Riviere Statistics in Medicine 40 (23), 5096-5114, 2021

Impact of early corticosteroids on 60-day mortality in critically ill patients with COVID-19: A multicenter cohort study of the OUTCOMEREA network

C Dupuis, E de Montmollin, N Buetti, D Goldgran-Toledano, J Reignier, ... PLoS One 16 (8), e0255644, 2021

Conditional Non-parametric Bootstrap for Non-linear Mixed Effect Models: Comets, Rodrigues, Jullien and Ursino

E Comets, C Rodrigues, V Jullien, M Ursino Pharmaceutical Research 38 (6), 1057-1066, 2021

Seamless master protocol with account for correlations within subgroups

B Duputel, S Zohar, F Montestruc, M Ursino Revue d'Épidémiologie et de Santé Publique 69, S14, 2021

Modélisation bayésienne d’une toxicité bivariée en essais de phase précoce en oncologie évaluant des régimes de dose

E Gérard, S Zoha, M Ursino, M Rivière Revue d'Épidémiologie et de Santé Publique 69, S13-S14, 2021

Extrapolation bayésienne de la préclinique à la clinique

S Boulet, M Ursino, R Michelet, C Kloft, E Comets, S Zohar Revue d'Épidémiologie et de Santé Publique 69, S13, 2021

The use of translational modelling and simulation to develop immunomodulatory therapy as an adjunct to antibiotic treatment in the context of pneumonia

R Michelet, M Ursino, S Boulet, S Franck, F Casilag, M Baldry, J Rolff, ... Pharmaceutics 13 (5), 601, 2021

L'équipe-projet HeKA

A Coulet, S Allassonniere, F Angoulvant, A Burgun, X Chen, D Drummond, ... Bulletin de l'Association Française pour l'Intelligence Artificielle, 29-32, 2021

Random-effects meta-analysis of phase I dose-finding studies using stochastic process priors

M Ursino, C Röver, S Zohar, T Friede The Annals of Applied Statistics 15 (1), 174-193, 2021

Multistate modeling of COVID-19 patients using a large multicentric prospective cohort of critically ill patients

M Ursino, C Dupuis, N Buetti, E de Montmollin, L Bouadma, ... Journal of clinical medicine 10 (3), 544, 2021

Estimating similarity of dose–response relationships in phase I clinical trials—case study in bridging data package

A Ollier, S Zohar, S Morita, M Ursino International journal of environmental research and public health 18 (4), 1639, 2021

LB03 Dose reduction of antenatal betamethasone in women at risk of very preterm delivery (BETADOSE trial)

T Schmitz, M Doret, L Sentilhes, C Alberti, M Ursino, C Aupiais, O Baud American Journal of Obstetrics & Gynecology 224 (2), S723-S724, 2021

Bayesian approaches for confirmatory trials in rare diseases: opportunities and challenges

M Ursino, N Stallard International Journal of Environmental Research and Public Health 18 (3), 1022, 2021

Association between early invasive mechanical ventilation and day-60 mortality in acute hypoxemic respiratory failure related to coronavirus disease-2019 pneumonia

C Dupuis, L Bouadma, E de Montmollin, D Goldgran-Toledano, ... Critical Care Explorations 3 (1), e0329, 2021

An adaptive power prior for sequential clinical trials–application to bridging studies

A Ollier, S Morita, M Ursino, S Zohar Statistical methods in medical research 29 (8), 2282-2294, 2020

Integration of elicited expert information via a power prior in Bayesian variable selection: Application to colon cancer data

S Boulet, M Ursino, P Thall, B Landi, C Lepère, S Pernot, A Burgun, ... Statistical methods in medical research 29 (2), 541-567, 2020

Nissen fundoplication and dyspeptic symptoms: is the water load test useful?

E Battaglia, V Boano, M Ursino, C Elia, L Russo, C Sguazzini, ... Minerva Gastroenterologica e Dietologica 66 (1), 11-16, 2019

A Bayesian non-inferiority approach using experts’ margin elicitation–application to the monitoring of safety events

C Aupiais, C Alberti, T Schmitz, O Baud, M Ursino, S Zohar BMC Medical Research Methodology 19 (1), 187, 2019

Bayesian knowledge integration for an in vitro–in vivo correlation model

EM Erhardt, M Ursino, J Biewenga, T Jacobs, M Gasparini Biometrical Journal 61 (5), 1104-1119, 2019

Bayesian sequential integration within a preclinical pharmacokinetic and pharmacodynamic modeling framework: Lessons learned

F La Gamba, T Jacobs, H Geys, T Jaki, J Serroyen, M Ursino, A Russu, ... Pharmaceutical statistics 18 (4), 486-506, 2019

Integration of elicited expert information via a power prior in Bayesian variable selection

S Boulet, M Ursino, P Thall, B Landi, C Lepère, S Pernot, A Burgun, ... ISCB 2019-40th Annual Conference of the International Society for Clinical …, 2019

Bayesian variable selection based on clinical relevance weights in small sample studies—Application to colon cancer

S Boulet, M Ursino, P Thall, AS Jannot, S Zohar Statistics in Medicine 38 (12), 2228-2247, 2019

Intégration de l’élicitation d’experts dans une méthode de sélection de variables en Bayésien par la méthode du power prior-Application au cancer colorectal

S Boulet, M Ursino, P Thall, B Landi, C Lepère, S Pernot, A Burgun, ... EPICLIN 2019-13e Conférence Francophone d’Epidémiologie Clinique, 2019

Une approche bayésienne de non-infériorité, utilisant des marges élicitées chez des experts, comme aide à la décision lors du monitoring d’événements secondaires rares

C Aupiais, M Ursino, S Thomas, O Baud, S Zohar, C Alberti Revue d'Épidémiologie et de Santé Publique 67, S157, 2019

Full versus half dose of antenatal betamethasone to prevent severe neonatal respiratory distress syndrome associated with preterm birth: study protocol for a randomised …

T Schmitz, C Alberti, M Ursino, O Baud, C Aupiais, ... BMC pregnancy and childbirth 19 (1), 67, 2019

A dose finding design for seizure reduction in neonates

M Ursino, Y Yuan, C Alberti, E Comets, G Favrais, T Friede, F Lentz, ... Journal of the Royal Statistical Society Series C: Applied Statistics 68 (2 …, 2019

Bayesian treatment comparison using parametric mixture priors computed from elicited histograms

PF Thall, M Ursino, V Baudouin, C Alberti, S Zohar Statistical methods in medical research 28 (2), 404-418, 2019

BETADOSE study group and the GROG (Groupe de Recherche en Gynécologie Obstétrique). Full versus half dose of antenatal betamethasone to prevent severe neonatal respiratory …

T Schmitz, C Alberti, M Ursino, O Baud, C Aupiais BMC Pregnancy Childbirth 19 (1), 67, 2019

Levetiracetam optimal dose-finding as first-line treatment for neonatal seizures occurring in the context of hypoxic-ischaemic encephalopathy (LEVNEONAT-1): study protocol of a …

G Favrais, M Ursino, C Mouchel, E Boivin, V Jullien, S Zohar, E Saliba BMJ open 9 (1), e022739, 2019

Dose-finding designs for cumulative toxicities using multiple constraints

SM Lee, M Ursino, YK Cheung, S Zohar Biostatistics 20 (1), 17-29, 2019

Recent advances in methodology for clinical trials in small populations: the InSPiRe project

T Friede, M Posch, S Zohar, C Alberti, N Benda, E Comets, S Day, ... Orphanet journal of rare diseases 13 (1), 186, 2018

Unified approach for extrapolation and bridging of adult information in early-phase dose-finding paediatric studies

C Petit, A Samson, S Morita, M Ursino, J Guedj, V Jullien, E Comets, ... Statistical methods in medical research 27 (6), 1860-1877, 2018

Nouvelle méthode bayésienne de sélection de variables pour des échantillons de petite taille incorporant l’expertise clinique. Application au cancer colorectal

S Boulet, M Ursino, P Thall, AS Jannot, S Zohara Revue d'Épidémiologie et de Santé Publique 66, S137, 2018

A new parsimonious model for ordinal longitudinal data with application to subjective evaluations of a gastrointestinal disease

M Ursino, M Gasparini Statistical methods in medical research 27 (5), 1376-1393, 2018

dfpk: an R-package for Bayesian dose-finding designs using pharmacokinetics (PK) for phase I clinical trials

A Toumazi, E Comets, C Alberti, T Friede, F Lentz, N Stallard, S Zohar, ... Computer Methods and Programs in Biomedicine 157, 163-177, 2018

Dose‐finding methods for phase I clinical trials using pharmacokinetics in small populations

M Ursino, S Zohar, F Lentz, C Alberti, T Friede, N Stallard, E Comets Biometrical Journal 59 (4), 804-825, 2017

A Bayesian weighted quasi-likelihood design for phase I/II clinical trial with repeated dose administration in preterm newborns

M Ursino, Y Yuan, C Alberti, E Comets, T Friede, F Lents, N Stallard, ... TRIALS 18, 2017