6th CEN Conference

Session

Clinical trials 4

21 May 2026, 13:45

Room 13 B

Conveners

Clinical trials 4

• Annette Kopp-Schneider (DKFZ)

188. Difference-in-difference estimators in randomized trials with external controls

Christiana Drake (University of California, Davis)

21/05/2026, 13:45

oral presentation

Randomized trials often utilize a select group of study participants. This group does not typically represent the general population. Furthermore, sample sizes are often small to reduce cost. To improve power and generalizability, external control groups may be added to the randomized study. It is possible to incorporate a suitably selected external control group into a randomized clinical...

148. Evidence Generation Using External Controls: Opportunities and Challenges – A Regulator’s Perspective

Armin Schüler (BfArM - Federal Institute for Drugs and Medical Devices)

21/05/2026, 14:03

oral presentation

Randomised controlled trials (RCTs) are the gold standard of evidence to support causal conclusions on the benefits and risks of medicines in regulatory decision making along the lifecycle [1]. However, single-arm trials (SATs) are also frequently used for various reasons during drug development. While RCTs allow adjustment for confounding via design, the contextualization of SATs requires...

116. Bayesian Methods Integrating Causal Inference Approaches for Borrowing Historical Control Data in RCTs: A Neutral Comparison Study

David Jesse (F. Hoffmann-La Roche AG, Basel, Switzerland; Department of Medical Statistics, University Medical Center Göttingen, Göttingen, Germany)

21/05/2026, 14:21

oral presentation

Bayesian dynamic borrowing (BDB) methods are popular for incorporating historical data in rare disease or paediatric clinical trials, in particular with regard to control groups. They can be used to leverage the historical information while mitigating the consequences of potential prior-data conflicts to some degree. However, these methods do not consider baseline covariate information that...

210. Identification of subtrial-specific optimal biological dose (OBD) with robust borrowing of information

Zhi Cao (MRC Biostatistics Unit, University of Cambridge)

21/05/2026, 14:39

oral presentation

Drug development in the era of precision medicine increasingly uses basket trials and other multi-subgroup designs, where targeted therapies are evaluated across biomarker-defined patient subtrials. For many targeted agents and immunotherapies, the objective in early development is no longer the maximum tolerated dose (MTD), but the optimal biological dose (OBD) that achieves the best...