Evidence Generation for Patient Reported Outcome (PRO) Label Claims (FDA Requirements)

Faculty - Jill A. Bell, PhD

Module Description:

In 2009, the US Food and Drug Administration (FDA) released a final guidance for industry on the use of Patient Reported Outcomes (PROs) in medical product development to support labelling claims (PRO Guidance). As defined in the FDA PRO Guidance, PRO instruments capture a patient’s health status based on information obtained directly from the patient about how they function or feel in relation to a health condition and its therapy, without interpretation by clinicians or others. Incorporating PROs into clinical trials provides an assessment of the meaningful changes that patients may experience as a result of treatment. Further, PROs also provide evidence of patients’ perceptions of treatment benefits that can be used to support medical product labeling claims. Such claims are statements or implications of treatment benefit that appear in any section of a medical product’s FDA approved label.

Learning Objectives:

This learning module will provide an overview of the importance of incorporating PROs in clinical trials to provide evidence of treatment effectiveness. The FDA PRO Guidance emphasizes the importance of conducting qualitative research throughout the process of instrument development to ensure that the measure is appropriate and comprehensive relative to the concept of interest, patient population, and context of use. When selecting an acceptable PRO measure for a clinical trial, it is important that the content of the measure is consistent with patients’ experiences and that the concepts measured by the instrument cover what patients consider most important about a condition and/or treatment. It is important to understand the documentation required for FDA review of a patient reported outcome instrument. In addition to providing evidence of content validity for the selected PRO instrument, it is also necessary to demonstrate the psychometric properties of the instrument in the target population. The FDA will review and evaluate the measurement properties of the PRO instrument such as evidence of validity, reliability, and the ability of the PRO instrument to detect change, within the specific context of use. Moreover, regulatory bodies hold PRO instruments to support labeling claims to the same regulatory and scientific standards as other measures used in clinical trials.

This module will discuss the evidence needed for PRO labeling claims (FDA requirements) and will also provide case studies of successful and unsuccessful label claims to demonstrate how to collect and present PRO evidence of treatment effectiveness.

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