TY - JOUR
T1 - The Evolving Role of Data & Safety Monitoring Boards for Real-World Clinical Trials
AU - Bunning, Bryan J.
AU - Hedlin, Haley
AU - Chen, Jonathan H.
AU - Ciolino, Jody D.
AU - Ferstad, Johannes Opsahl
AU - Foxf, Emily
AU - Garcia, Ariadna
AU - Go, Alan
AU - Johari, Ramesh
AU - Lee, Justin
AU - Maahs, David M.
AU - Mahaffey, Kenneth W.
AU - Opsahl-Ong, Krista
AU - Perez, Marco
AU - Rochford, Kaylin
AU - Scheinker, David
AU - Spratt, Heidi
AU - Turakhia, Mintu P.
AU - Des, Manisha
N1 - Publisher Copyright:
© 2023 Cambridge University Press. All rights reserved.
PY - 2023
Y1 - 2023
N2 - Introduction: Clinical trials provide the "gold standard" evidence for advancing the practice of medicine, even as they evolve to integrate real-world data sources. Modern clinical trials are increasingly incorporating real-world data sources - data not intended for research and often collected in free-living contexts. We refer to trials that incorporate real-world data sources as real-world trials. Such trials may have the potential to enhance the generalizability of findings, facilitate pragmatic study designs, and evaluate real-world effectiveness. However, key differences in the design, conduct, and implementation of real-world vs traditional trials have ramifications in data management that can threaten their desired rigor. Methods: Three examples of real-world trials that leverage different types of data sources - wearables, medical devices, and electronic health records are described. Key insights applicable to all three trials in their relationship to Data and Safety Monitoring Boards (DSMBs) are derived. Results: Insight and recommendations are given on 4 topic areas: A. Charge of the DSMB; B. Composition of the DSMB.; C. Pre-launch Activities; and D. Post-launch Activities. We recommend stronger and additional focus on data integrity. Conclusions: Clinical trials can benefit from incorporating real-world data sources, potentially increasing the generalizability of findings and overall trial scale and efficiency. The data, however, present a level of informatic complexity that relies heavily on a robust data science infrastructure. The nature of monitoring the data and safety must evolve to adapt to new trial scenarios to protect the rigor of clinical trials.
AB - Introduction: Clinical trials provide the "gold standard" evidence for advancing the practice of medicine, even as they evolve to integrate real-world data sources. Modern clinical trials are increasingly incorporating real-world data sources - data not intended for research and often collected in free-living contexts. We refer to trials that incorporate real-world data sources as real-world trials. Such trials may have the potential to enhance the generalizability of findings, facilitate pragmatic study designs, and evaluate real-world effectiveness. However, key differences in the design, conduct, and implementation of real-world vs traditional trials have ramifications in data management that can threaten their desired rigor. Methods: Three examples of real-world trials that leverage different types of data sources - wearables, medical devices, and electronic health records are described. Key insights applicable to all three trials in their relationship to Data and Safety Monitoring Boards (DSMBs) are derived. Results: Insight and recommendations are given on 4 topic areas: A. Charge of the DSMB; B. Composition of the DSMB.; C. Pre-launch Activities; and D. Post-launch Activities. We recommend stronger and additional focus on data integrity. Conclusions: Clinical trials can benefit from incorporating real-world data sources, potentially increasing the generalizability of findings and overall trial scale and efficiency. The data, however, present a level of informatic complexity that relies heavily on a robust data science infrastructure. The nature of monitoring the data and safety must evolve to adapt to new trial scenarios to protect the rigor of clinical trials.
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U2 - 10.1017/cts.2023.582
DO - 10.1017/cts.2023.582
M3 - Article
AN - SCOPUS:85167651675
SN - 2059-8661
JO - Journal of Clinical and Translational Science
JF - Journal of Clinical and Translational Science
ER -