volume 23 issue 4

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

Björn Bornkamp 1
Silvia Zaoli 1
Michela Azzarito 1
Ruvie Martin 2
Carsten Philipp Müller 3
Conor Moloney 4
Giulia Capestro 1
David Ohlssen 2
Mark Baillie 1
1
 
Global Drug Development Novartis Pharma AG Basel Switzerland
2
 
Global Drug Development Novartis Pharmaceuticals Corporation East Hanover New Jersey USA
3
 
Data Digital Beyond Conception GmbH Altendorf Switzerland
4
 
Global Drug Development Novartis Pharma AG Dublin Ireland
Publication typeJournal Article
Publication date2024-02-07
scimago Q1
wos Q2
SJR1.074
CiteScore3.2
Impact factor1.4
ISSN15391604, 15391612
PubMed ID:  38326967
Pharmacology
Pharmacology (medical)
Statistics and Probability
Abstract

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.

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Bornkamp B. et al. Predicting subgroup treatment effects for a new study: Motivations, results and learnings from running a data challenge in a pharmaceutical corporation // Pharmaceutical Statistics. 2024. Vol. 23. No. 4.
GOST all authors (up to 50) Copy
Bornkamp B., Zaoli S., Azzarito M., Martin R., Müller C. P., Moloney C., Capestro G., Ohlssen D., Baillie M. Predicting subgroup treatment effects for a new study: Motivations, results and learnings from running a data challenge in a pharmaceutical corporation // Pharmaceutical Statistics. 2024. Vol. 23. No. 4.
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RIS Copy
TY - JOUR
DO - 10.1002/pst.2368
UR - https://doi.org/10.1002/pst.2368
TI - Predicting subgroup treatment effects for a new study: Motivations, results and learnings from running a data challenge in a pharmaceutical corporation
T2 - Pharmaceutical Statistics
AU - Bornkamp, Björn
AU - Zaoli, Silvia
AU - Azzarito, Michela
AU - Martin, Ruvie
AU - Müller, Carsten Philipp
AU - Moloney, Conor
AU - Capestro, Giulia
AU - Ohlssen, David
AU - Baillie, Mark
PY - 2024
DA - 2024/02/07
PB - Wiley
IS - 4
VL - 23
PMID - 38326967
SN - 1539-1604
SN - 1539-1612
ER -
BibTex
Cite this
BibTex (up to 50 authors) Copy
@article{2024_Bornkamp,
author = {Björn Bornkamp and Silvia Zaoli and Michela Azzarito and Ruvie Martin and Carsten Philipp Müller and Conor Moloney and Giulia Capestro and David Ohlssen and Mark Baillie},
title = {Predicting subgroup treatment effects for a new study: Motivations, results and learnings from running a data challenge in a pharmaceutical corporation},
journal = {Pharmaceutical Statistics},
year = {2024},
volume = {23},
publisher = {Wiley},
month = {feb},
url = {https://doi.org/10.1002/pst.2368},
number = {4},
doi = {10.1002/pst.2368}
}