TY - JOUR
T1 - Utilizing shared internal control arms and historical information in small-sized platform clinical trials
AU - Jiao, Feiran
AU - Tu, Wenda
AU - Jimenez, Sara
AU - Crentsil, Victor
AU - Chen, Yeh Fong
N1 - Funding Information:
This work was supported by the ORISE Research Program of the U.S. Food and Drug Administration. The authors express their gratitude to Dr. Kathleen Donohue for her valuable and constructive suggestions during the planning and development of this research work.
Publisher Copyright:
© 2019, © 2019 Taylor & Francis Group, LLC.
PY - 2019/9/3
Y1 - 2019/9/3
N2 - Recruitment of patients in concurrent control arms can be very challenging for clinical trials for pediatric and rare diseases. Innovative approaches, such as platform trial designs, including shared internal control arm(s), can potentially reduce the needed sample size, improving the efficiency and speed of the drug development program. Furthermore, historical borrowing, which involves leveraging information from control arms in previous relevant clinical trials, may further enhance a clinical trial’s efficiency. In this paper, we discuss platform trials highlighting their advantages and limitations. We then compare various strategies that borrow historical data or information, such as pooling data from different studies, analyzing data from studies separately, test-then-pool, dynamic pooling, and Bayesian hierarchical modeling, which focuses on the meta-analytic-predictive (MAP) prior. We further propose a procedure to illustrate the feasibility of utilizing historical controls under a platform setting and describe the statistical performance of our method via simulations.
AB - Recruitment of patients in concurrent control arms can be very challenging for clinical trials for pediatric and rare diseases. Innovative approaches, such as platform trial designs, including shared internal control arm(s), can potentially reduce the needed sample size, improving the efficiency and speed of the drug development program. Furthermore, historical borrowing, which involves leveraging information from control arms in previous relevant clinical trials, may further enhance a clinical trial’s efficiency. In this paper, we discuss platform trials highlighting their advantages and limitations. We then compare various strategies that borrow historical data or information, such as pooling data from different studies, analyzing data from studies separately, test-then-pool, dynamic pooling, and Bayesian hierarchical modeling, which focuses on the meta-analytic-predictive (MAP) prior. We further propose a procedure to illustrate the feasibility of utilizing historical controls under a platform setting and describe the statistical performance of our method via simulations.
KW - Bayesian
KW - Platform trials
KW - historical borrowing
KW - rare disease
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U2 - 10.1080/10543406.2019.1657132
DO - 10.1080/10543406.2019.1657132
M3 - Article
C2 - 31462131
AN - SCOPUS:85071319618
SN - 1054-3406
VL - 29
SP - 845
EP - 859
JO - Journal of biopharmaceutical statistics
JF - Journal of biopharmaceutical statistics
IS - 5
ER -