Optimal multiarm multistage platform randomized trials using Stata: Rationale, design, and implementation

Babak Choodari-Oskooei
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Babak Choodari-Oskooei: University College London

Biostatistics and Epidemiology Virtual Symposium 2024 from Stata Users Group

Abstract: Multiarm multistage (MAMS) platform randomized clinical trials are an efficient, adaptive approach for testing many treatments within one master protocol. They include flexible features such as early stopping of accrual to treatments for lack of benefit, as well as the opportunity to add new treatment arms over the course of the trial. The MAMS framework has been used in several confirmatory randomized clinical trials for the treatment of COVID-19, cancer, infectious diseases, neurodegenerative diseases, and surgery. This talk introduces the MAMS design, elucidates its underlying rationale, and highlights its advantages. Utilizing the nstage suite of commands in Stata and two ongoing real MAMS trials in prostate cancer (STAMPEDE) and surgery (ROSSINI-2), it illustrates how to calculate the required sample size for such a complex design. Further, it addresses the challenge of identifying efficient MAMS trial designs with specific overall pairwise or familywise operating characteristics and provides guidelines on how to implement these designs.

Date: 2024-02-22
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http://repec.org/biep2024/Bio24_Choodari-Oskooei.pdf presentation materials (application/zip)

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Persistent link: https://EconPapers.repec.org/RePEc:boc:biep24:04

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