Journal article
CITIES: Clinical trials with intercurrent events simulator
Biometrical journal, v 66(1), 2200103
01 Jan 2024
PMID: 37740165
Featured in Collection : UN Sustainable Development Goals @ Drexel
Abstract
Although clinical trials are often designed with randomization and well-controlled protocols, complications will inevitably arise in the presence of intercurrent events (ICEs) such as treatment discontinuation. These can lead to missing outcome data and possibly confounding causal inference when the missingness is a function of a latent stratification of patients defined by intermediate outcomes. The pharmaceutical industry has been focused on developing new methods that can yield pertinent causal inferences in trials with ICEs. However, it is difficult to compare the properties of different methods developed in this endeavor as real-life clinical trial data cannot be easily shared to provide benchmark data sets. Furthermore, different methods consider distinct assumptions for the underlying data-generating mechanisms, and simulation studies often are customized to specific situations or methods. We develop a novel, general simulation model and corresponding Shiny application in R for clinical trials with ICEs, aptly named the Clinical Trials with Intercurrent Events Simulator (CITIES). It is formulated under the Rubin Causal Model where the considered treatment effects account for ICEs in clinical trials with repeated measures. CITIES facilitates the effective generation of data that resemble real-life clinical trials with respect to their reported summary statistics, without requiring the use of the original trial data. We illustrate the utility of CITIES via two case studies involving real-life clinical trials that demonstrate how CITIES provides a comprehensive tool for practitioners in the pharmaceutical industry to compare methods for the analysis of clinical trials with ICEs on identical, benchmark settings that resemble real-life trials.
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Details
- Title
- CITIES: Clinical trials with intercurrent events simulator
- Creators
- Ahmad Hakeem Abdul Wahab - Decision Sciences (United States)Yongming Qu - Eli Lilly (United States)Hege Michiels - Ghent UniversityJunxiang Luo - Moderna Therapeutics (United States)Run Zhuang - Purdue University West LafayetteDominique McDaniel - Drexel University, Epidemiology and BiostatisticsDong Xi - Gilead Sciences (United States)Elena Polverejan - Decision Sciences (United States)Steven Gilbert - Pfizer (United States)Stephen Ruberg - Analytix Thinking LLC, Indianapolis, IN, United StatesArman Sabbaghi - Purdue University West Lafayette
- Publication Details
- Biometrical journal, v 66(1), 2200103
- Resource Type
- Journal article
- Language
- English
- Academic Unit
- Epidemiology and Biostatistics
- Web of Science ID
- WOS:001068400700001
- Scopus ID
- 2-s2.0-85171750823
- Other Identifier
- 991021861297704721
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- Collaboration types
- Industry collaboration
- Domestic collaboration
- International collaboration
- Web of Science research areas
- Mathematical & Computational Biology
- Statistics & Probability