Adaptive and Platform Trials in Remote Damage Control Resuscitation

The traditional approach to clinical trial design requires assuming precise values for multiple unknown parameters, resulting is a trial design that is unlikely to perform well if one or more of those assumptions turn out to be incorrect. During conduct of the trial, trial characteristics are often held fixed, even if incoming data suggest that one or more design assumptions were incorrect. This leads to an increased risk of a failed trial. In contrast, an adaptive clinical trial is designed to take advantage of partial, incoming data during the conduct of the trial, modifying key clinical trial characteristics according to prespecified rules, in order to avoid a failed or inconclusive trial, improve statistical efficacy, better treat patients within the trial, or achieve other scientific or ethical goals. The concept of an adaptive trial can be expanded to a platform trial, a clinical trial that is intended to evaluate multiple treatments or combinations of treatments, often for patients with any of a group of related diseases, and to continue beyond the evaluation of any particular treatment. Platform trial design strategies can be applied to the problem of finding the best treatment strategy for patients suffering from post-traumatic hemorrhagic shock. We present the rationale and considerations surrounding adaptive and platform trial design and apply these concepts to the problem of investigating strategies for remote damage control resuscitation. This manuscript is submitted for consideration for publication in the THOR meeting supplement of the Journal of Trauma. Presentations: This work was presented at the Traumatic Hemostasis and Oxygenation Research Network 2017 Remote Damage Control Resuscitation Symposium in Norway. Conflict of Interests: Dr. Tolles has no conflicts of interest to declare. Dr. Lewis is the Senior Medical Scientist at Berry Consultants, LLC, a statistical consulting firm that specializes in the design, implementation, oversight, and interpretation of adaptive and platform clinical trials. Both Dr. Lewis and his institution are compensated for this work. Funding: No funding was received for this work. Corresponding Author: Juliana Tolles, MD, MHS, Department of Emergency Medicine, Harbor-UCLA Medical Center, 1000 W. Carson St Box #21, Torrance, CA 90502. Fax: 310-782-1763. Email: jtolles@emedharbor.edu © 2018 Lippincott Williams & Wilkins, Inc.

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