Workshop on the use of Bayesian statistics in clinical development

• Date:
  Tuesday, 17 June 2025, All day
• Location:
  Online
  European Medicines Agency, Amsterdam, the Netherlands

Event summary

As part of the published Methodology Working Party 2025-2027 workplan and the Accelerating Clinical Trials in the EU (ACT EU) multi-annual workplan 2025-2026, a workshop on the use of Bayesian statistics in clinical development will be held on 17 June 2025 at EMA premises in Amsterdam.

The workshop aims to engage all stakeholders, including but not limited to patients, healthcare professionals, industry and academia.

The objectives of this workshop are to:

• Discuss the potential benefits and challenges when using Bayesian statistics in clinical development
• Discuss what information might be useful and relevant to include in upcoming guidance documents
• Better understand the trial designs that are being proposed and used, so that future guidance is fit for purpose

Documents

Agenda - Workshop on the use of Bayesian statistics in clinical development

Presentation - Bayesian statistics in regulatory decision-making: Panacea or Pandora’s Box? (K.Karlsson, MWP)

Presentations: Session 1

Presentation - Bayesian borrowing in clinical trial test decisions: Frequentist type I error rate and power (A. Kopp-Schneider, DKFZ)

Presentation - Opportunities and challenges of using Bayesian Methods to support regulatory decision making (N. Best, GSK)

Presentations: Session 2

Presentation - EU Concept Paper on the use of Bayesian statistics in clinical development (P.van de Ven, CBG MEB)

Presentation - ICH E20: an industry perspective (U.Bretz, Novartis)

Presentation - ICH E20: a regulatory perspective (F.Petavy, A.Koch, EMA)

Presentations: Session 3

Presentation - Bayesian methods without borrowing in ultrarare diseases (N.Muehlemann, J.Priel, Cytel)

Presentation - Bayesian Adaptive Design for a Practice-Changing Platform Trial in a Rare Paediatric Cancer: The Glo-BNHL Trial (L.Billingham, University of Birmingham)

Presentation - Bayesian methods to support clinical interpretation of treatment effects, interim decision making and handling of intercurrent events (N.Best, P.Dalvi, GSK)

Presentations: Session 4

Presentation - Bayesian borrowing for paediatric extrapolation: The DINAMO study (M.Sailer, I.Tartakovsky, Boehringer Ingelheim Pharma GmbH & Co. KG)

Presentation - A regulator’s perspective on pediatric Bayesian Methods (J.Travis, US FDA)

Presentations: Session 5

Presentation - Bayesian shrinkage methods for routine estimation of subgroup treatment effects (B.Bornkamp, Novartis; N.Best, D.Bratton, GSK; M.Jelizarow, UCB; N.Muhlemann, Cytel; J.McMurray, University of Glasgow)

Presentation - ICH-M15 for Bayesian Modelling: A systematic model assessment framework to support design submission and discussion (T.Mielke, Johnson&Johnson)

Video recording