Platform trial

A platform trial is a type of prospective, disease-focused,[1] adaptive, randomized clinical trial (RCT) that compares multiple, simultaneous and possibly differently-timed interventions against a single, constant control group.[2] As a disease-focused trial design (compared to an intervention-focused), platform trials attempt to answer the question "which therapy will best treat this disease". Platform trials are unique in their utilization of both: a common control group and their opportunity to alter the therapies it investigates during its active enrollment phase. Platform trials commonly take advantage of Bayesian statistics, but may incorporate elements of frequentist statistics and/or machine learning.[3]

  1. ^ Park JJ, Harari O, Dron L, Lester RT, Thorlund K, Mills EJ (September 2020). "An overview of platform trials with a checklist for clinical readers". Journal of Clinical Epidemiology. 125: 1–8. doi:10.1016/j.jclinepi.2020.04.025. PMID 32416336. S2CID 218670123.
  2. ^ Cite error: The named reference Angus_2020 was invoked but never defined (see the help page).
  3. ^ Angus, Derek C.; Alexander, Brian M.; Berry, Scott; Buxton, Meredith; Lewis, Roger; Paoloni, Melissa; Webb, Steven A. R.; Arnold, Steven; Barker, Anna; Berry, Donald A.; Bonten, Marc J. M. (October 2019). "Adaptive platform trials: definition, design, conduct and reporting considerations". Nature Reviews Drug Discovery. 18 (10): 797–807. doi:10.1038/s41573-019-0034-3. ISSN 1474-1784. PMID 31462747. S2CID 201652338.