Using the principles of randomized controlled trial design to guide test evaluation.

The decision to use a new test should be based on evidence that it will improve patient outcomes or produce other benefits without adversely affecting patients. In principle, long-term randomized controlled trials (RCTs) of test-plus-treatment strategies offer ideal evidence of the benefits of introducing a new test relative to current best practice. However, long-term RCTs may not always be necessary. The authors advocate using the hypothetical RCT as a conceptual framework to identify what types of comparative evidence are needed for test evaluation. Evaluation begins by stating the major claims for the new test and determining whether it will be used as a replacement, add-on, or triage test to achieve these claims. A flow diagram of this hypothetical RCT is constructed to show the essential design elements, including population, prior tests, new test and existing test strategies, and primary and secondary outcomes. Critical steps in the pathway between testing and patient outcomes, such as differences in test accuracy, changes in treatment, or avoidance of other tests, are displayed for each test strategy. All differences between the tests at these critical steps are identified and prioritized to determine the most important questions for evaluation. Long-term RCTs will not be necessary if it is valid to use other sources of evidence to address these questions. Validity will depend on issues such as the spectrum of patients identified by the old and new test strategies.