Abstract:
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The call for patient-focused drug development is loud and clear, as expressed in the 21st Century Cures Act and in recent regulatory guidelines and initiatives. Among the factors contributing to modernized drug development and improved healthcare are easily interpretable measures of clinical benefit. Special care is needed for cancer trials with time-to-event endpoints, especially if the proportional hazards assumption is questionable. In addition to hypothesis testing and estimation of hazard ratios and median survival times, we propose the predictive individual effect as a more tangible measure of clinical benefit. It uses the data from the actual trial to quantify a new patient's potential survival gain, that is, the difference between the patient’s predicted survival times under the test and control treatment. The predictive individual effect has a clear and simple interpretation. The usefulness of the new measure is illustrated with four examples from recent Oncology trials.
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