Day 1 – Monday, 19th September 2022


12:30 – 13:30 Registration

13:30 – 13:45 WELCOME by Rodolphe Thiébaut & Frédéric Dubois

Session 1: Big data to the rescue of drug development challenges?


13:45 – 14:30 Chris HOLMES - Oxford – Big Data Institute (UK) [ SLIDE ]

Big data, prediction, and inference: a Bayesian perspective on data fusion

14:30 – 15:15 Harald BINDER (University of Freiburg (DE)

Deep learning and dynamic modeling for rare disease evidence from small data

15:15 – 15:45 COFFEE BREAK, EXHIBITORS & POSTERS SESSION

15:45 – 16:05 Bénédicte COLNET [ SLIDE ]

Combining randomized and observational data: toward new clinical evidence?

16:05 – 16:25 Bruno BOULANGER [ SLIDE ]

Leveraging natural history data in one and two arm hierarchical bayesian studies of rare disease progression

16:25 – 17:15 5’ POSTERS overview
  • Edouard LHOMME: COVERAGE France platform: a national multicenter adaptive randomized clinical trial using a multiarm multistage (MAMS) design to evaluate several experimental treatments for covid-19 in the outpatient setting

  • Vincent BONNEMAINS: Impact of the kidney transplantation moratorium in France because of the COVID-19 pandemic: a cohort-based study

  • Manel RAKEZ: Deep-learning and statistical methods for sequential breast density estimation and dynamic breast cancer risk evaluation in women involved in breast cancer screening programs

  • Corine BAAYEN: Group sequential trials for repeated measurements with pipeline patients at interim decision points: concepts and case study

  • Samuel SALVAGGIO: The net treatment benefit: an intuitive statistical tool involving individual patients in treatment decision-making

  • Alice GOSSELIN: A multiple cohort Ph2 study design with decision making based on Bayesian hierarchical modelling

  • Federico ROTOLO: Validation of minimal residual disease as surrogate for a time-to-event clinical endpoint

17:15 – 20:30 WINE & POSTERS

Day 2 – Tuesday, 20th September 2022

Session 2: Statistics and Machine Learning: friends or foes?


09:00 – 09:45 Ewout STEYERBERG - Leiden University Medical Center (NL) [ SLIDE ]

Statistics and machine learning: friends or foes?

09:45– 10:30 Gary COLLINS - University of Oxford (UK) [ SLIDE ]

Methodological and reporting issues in machine learning based clinical prediction models

10:30 – 11:00 COFFEE BREAK, EXHIBITORS & POSTERS SESSION

11:00 – 11:20 Michael LEBLANC

Subgroup rule induction for patient outcomes: examples and experience with machine learning methods for clinical data

11:20 – 11:40 Jenna CARCIONE [ SLIDE ]

Evaluation of machine learning methods in propensity score estimation

11:40 – 12:00 Yohann FOUCHER [ SLIDE ]

Super Learner for survival prediction from censored data: a simulation-based study.

12:00 – 13:30 LUNCH BUFFET, EXHIBITORS & POSTERS SESSION

Session 3: Getting medicines to patients faster - the role of innovative designs


13:30 – 14:15 Emilie KAUFMANN - University of Lille (FR) [ SLIDE ]

On multi-armed bandit algorithms for early stage clinical trials

14:15 – 15:00 Wolfgang JACQUET - FAGG - AFMPS (BE) [ SLIDE ]

Robust decision making and precise estimation.
Complex designs, artificial intelligence, and real-world data, how to move forward?

15:00 – 15:30 COFFEE BREAK, EXHIBITORS & POSTERS SESSION

15:30 – 15:50 Alessandra SERRA [ SLIDE ]

An order restricted multi-arm multi-stage clinical trial design

15:50 – 16:10 Fulvio DI STEFANO

Incorporation of phase I healthy volunteers data on receptor occupancy into a phase II proof-of-concept trial using a Bayesian dynamic borrowing design

16:10 – 16:30 Benjamin DUPUTEL [ SLIDE ]

Bayesian seamless master protocols and how to transfer information in Phase II/III binary/survival outcome – Theory and application on Atalante-1 study

16:30 – 17:00 COFFEE BREAK, EXHIBITORS & POSTERS SESSION

17:00 – 17:20 Pavel MOZGUNOV [ SLIDE ]

A dose-finding design for dual-agent trials with patient-specific doses for one agent with application to an opiate detoxification trial

17:20 – 17:40 Mickaël DE BACKER [ SLIDE ]

The use of Generalized Pairwise Comparisons for designing trials that are tailored to patients’ wishes and needs

19:30 – 22:30 CONFERENCE DINNER

Day 3 – Wednesday 21th September 2022

Discussion/Debate session

  • Michal ABRAHAMOWICZ [ SLIDE ]

    Analytical Challenges in Real-World studies of Drug Safety: If, Where and How Statistical Modeling may Help ?

  • Antoine CHAMBAZ [ SLIDE ]

    Statistical vs. Machine Learning approaches - a debate

12:30 – 14:00 LUNCH BUFFET, EXHIBITORS & POSTERS SESSION

Session 4: Lessons learnt from the covid-19 experience


14:00 – 14:45 Marc BUYSE - IDDI and Hasselt University (BE) [ SLIDE ]

COVID and the need for pragmatism in clinical trials

14:45 – 15:30 Stephen SENN - Statistical Consultant Edinburgh (UK) [ SLIDE ]

COVID and concurrent control

15:30 – 15:50 An VANDEBOSCH

Modeling and simulation to guide a COVID-19 Vaccine Phase 3 trial: Janssen case study

15:50 – 16:00 CLOSING WORDS by Rodolphe Thiébaut