Dynamic enrichment of Bayesian small‐sample, sequential, multiple assignment randomized trial design using natural history data: a case study from Duchenne muscular dystrophy

• Published in: Biometrics Vol. 79; no. 4; pp. 3612 - 3623
• Main Authors: Wang, Sidi, Kidwell, Kelley M., Roychoudhury, Satrajit
• Format: Journal Article
• Language: English
• Published: United States Blackwell Publishing Ltd 01.12.2023
• Subjects: Bayesian analysis; Bayesian hierarchical model; clinical trial; Clinical trials; Control data (computers); Drug development; Drug dosages; Duchenne muscular dystrophy; Duchenne's muscular dystrophy; Estimators; Heterogeneity; Muscular dystrophy; Patients; Placebos; rare disease; Rare diseases; Robustness; small sample; clinical trial; Bayesian hierarchical model; rare disease; Duchenne muscular dystrophy; small sample

In Duchenne muscular dystrophy (DMD) and other rare diseases, recruiting patients into clinical trials is challenging. Additionally, assigning patients to long‐term, multi‐year placebo arms raises ethical and trial retention concerns. This poses a significant challenge to the traditional sequential...