Predicting subgroup treatment effects for a new study: Motivations, results and learnings from running a data challenge in a pharmaceutical corporation

• Published in: Pharmaceutical statistics : the journal of the pharmaceutical industry 2024-07, Vol.23 (4), p.495-510
• Main Authors: Bornkamp, Björn, Zaoli, Silvia, Azzarito, Michela, Martin, Ruvie, Müller, Carsten Philipp, Moloney, Conor, Capestro, Giulia, Ohlssen, David, Baillie, Mark
• Format: Article
• Language: English
• Subjects: Biostatistics - methods; Clinical Trials, Phase III as Topic - methods; common task framework; Data Interpretation, Statistical; data science; Drug Industry; Humans; machine learning; Motivation; Research Design; subgroup analysis; subgroup identification; Treatment Outcome
• ISSN: 1539-1604; 1539-1612; 1539-1612
• DOI: 10.1002/pst.2368

We present the motivation, experience, and learnings from a data challenge conducted at a large pharmaceutical corporation on the topic of subgroup identification. The data challenge aimed at exploring approaches to subgroup identification for future clinical trials. To mimic a realistic setting, participants had access to 4 Phase III clinical trials to derive a subgroup and predict its treatment effect on a future study not accessible to challenge participants. A total of 30 teams registered for the challenge with around 100 participants, primarily from Biostatistics organization. We outline the motivation for running the challenge, the challenge rules, and logistics. Finally, we present the results of the challenge, the participant feedback as well as the learnings. We also present our view on the implications of the results on exploratory analyses related to treatment effect heterogeneity.