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Chapter 7.2: How to Use and Interpret the Results of a Platform Trial

Jay J. H. Park; Michelle A. Detry; Srinivas Murthy; Gordon Guyatt; Edward J. Mills

Clinical Scenario

CLINICAL SCENARIO

A hospitalist physician is evaluating a 60-year-old patient with severe COVID-19 who was admitted to the intensive care unit (ICU) and is receiving high-flow oxygen via nasal cannula. A resident physician on the ICU team is considering administration of intravenous hydrocortisone to the patient. The hospitalist physician is requested to assess the evidence regarding that decision. The physician is aware of a platform trial, the Randomized, Embedded, Multifactorial Adaptive Platform Trial for Community-Acquired Pneumonia (REMAP-CAP), that investigated hydrocortisone vs no hydrocortisone for patients with severe COVID-19.1 REMAP-CAP is an ongoing, international, multicenter platform trial that aims to determine best treatment strategies for patients with severe pneumonia in pandemic and nonpandemic settings.2 This trial is investigating multiple interventions including corticosteroids.

The article reporting the corticosteroid findings of the REMAP-CAP trial presented results on organ support–free days, in-hospital mortality, and other clinical outcomes of 2 hydrocortisone regimens vs no hydrocortisone.1 To assess this article, and in particular to determine what inferences can be drawn from platform trials compared with conventional randomized clinical trials (RCTs), the physician uses the framework in the Users’ Guide to Platform Trials (Box 7.2-1).

BOX 7.2-1

Questions Addressing Risk of Bias in Platform Trials

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BOX 7.2-1

Questions Addressing Risk of Bias in Platform Trials

  • Were prespecified plans for interim and statistical analyses used in the trial? Were the prespecified plans applied equally to all interventions?a

  • Did the investigators include nonconcurrent randomized participants or limit statistical comparisons to concurrently randomized patients?a

  • Did the investigators minimize risk of bias from information flow within and outside the specific platform?

  • Did the investigators follow reporting guidelines in published reports of the trials?

aAddresses risk of bias specifically for platform trials.

Fundamental Principles of Platform Trials

Platform trials are RCTs that allow for multiple interventions to be simultaneously evaluated and new interventions to be added after the trial is initiated (Figure 7.2-1).3-5 The platform provides a single infrastructure wherein interventions can be added and discontinued at different times after their clinical questions are answered through comparisons against a control group, sometimes shared across interventions.3-5 The control group and the standard care provided can be updated during platform trials as interventions are shown to be effective or ineffective over time.5

FIGURE 7.2-1

Illustrative Example of a Platform Trial Schema

The platform initially starts as a 3-group randomized clinical trial with placebo as a common control, and new treatments are introduced into the platform over time. Green circles indicate start of random assignment of participants to a new intervention, and red circles indicate stopping of assignment and/or testing of that intervention.

A diagram illustrates the duration of the platform trial placebo and treatments.
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In the setting of multiple competing but unestablished treatments with new treatments on ...

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