Utilizing shared internal control arms and historical information in small-sized platform clinical trials

Feiran Jiao, Wenda Tu, Sara Jimenez, Victor Crentsil, Yeh Fong Chen

Research output: Contribution to journalArticle

Abstract

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.

Original languageEnglish (US)
JournalJournal of Biopharmaceutical Statistics
DOIs
StateAccepted/In press - Jan 1 2019
Externally publishedYes

Fingerprint

Clinical Trials
Internal
Pooling
Rare Diseases
Hierarchical Modeling
Sample Size
Bayesian Modeling
Patient Selection
Pediatrics
Meta-Analysis
Historical Data
Simulation Methods
Concurrent
Drugs
Pharmaceutical Preparations

Keywords

  • Bayesian
  • historical borrowing
  • Platform trials
  • rare disease

ASJC Scopus subject areas

  • Statistics and Probability
  • Pharmacology
  • Pharmacology (medical)

Cite this

Utilizing shared internal control arms and historical information in small-sized platform clinical trials. / Jiao, Feiran; Tu, Wenda; Jimenez, Sara; Crentsil, Victor; Chen, Yeh Fong.

In: Journal of Biopharmaceutical Statistics, 01.01.2019.

Research output: Contribution to journalArticle

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