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ePRO Resource Center > FDA Draft Guidance - Patient Reported Outcomes (PRO)
FDA Draft Guidance on Patient Reported Outcomes
In February, 2006 The FDA issued its Draft Guidance on Patient Reported Outcomes (PRO) measures, capturing the FDA’s current thinking on PRO data capture in clinical research. It is intended to help sponsors understand what the agency will look for when reviewing NDAs that include the collection of PRO data, including suggestions on how sponsors can best support claims in product labeling with study results measured by PRO instruments.
Click any link below to access related information about the FDA’s Draft Guidance.
- Download the FDA’s Draft Guidance
- Read, “Patient-Reported Outcomes to Support Medical Product labeling Claims: FDA Perspective”; Sloan, J., Halyard, M., Frost, M., Dueck, A., Teschendorf, B., Rothman, M, the Mayo/FDA Patient-Reported Outcomes Consensus Meeting Group (Online Article - Blackwell Synergy Download Fee Applies)
Summary: Patient-reported outcomes (PROs) have become increasingly prevalent in clinical research and practice. On February 2, 2006, the Food and Drug Administration (FDA) released a draft guidance document with respect to incorporating PROs into clinical research endeavors which include FDA involvement. Researchers at the Mayo Clinic worked with FDA personnel and experts from academia, industry, clinical research, and clinical practice to facilitate discussion, dissemination, and operationalization of the FDA guidance document. This article introduces a manuscript series that resulted from this collective effort. - Read “Equivalence of Electronic and Paper-and-Pencil Administration of Patient-Reported Outcome Measures: A Meta-Analytic Review”; Shields, A., Gwaltney, C., Shiffman, S., Value in Health, September 2007 (Online Early Article - Blackwell Synergy Download Fee Applies)
Summary: Patient-reported outcomes (PROs; self-report assessments) are increasingly important in evaluating medical care and treatment efficacy. Electronic administration of PROs via computer is becoming widespread. This article reviews the literature addressing whether computer-administered tests are equivalent to their paper-and-pencil forms. - Read “Grasping the FDA's PRO Guidance”; Shields, A., Gwaltney, C., Tiplady, B, Paty, J., Shiffman, S. Applied Clinical Trials, 2006
Summary: By setting ground rules for the use of Patient Reported Outcomes (PROs), the FDA has implicitly given credibility to PROs as the basis for evaluating drugs and biologics. However, some researchers perceive a few challenges in the guidance FDA has issued. The objective of this article is to give an overview of the PRO Guidance for clinical researchers implementing PROs, with specific focus on how to document the history and psychometric properties of their PRO instruments and how to determine the amount of revalidation, if any, might be necessary in light of the guidance. - Read “Documentation of PRO Instruments to Meet Contemporary FDA Standards”; Shields, A., Gwaltney, C., Paty, J., Shiffman, S., White Paper, 2006
Summary: The FDA draft guidance on PROs may make it difficult for researchers to understand the FDA's recommendations on how to justify and appropriately document the intended use of their PRO instruments. The goal of this paper is to describe what information clinical researchers should consider providing to the FDA to justify the use of a given PRO instrument in some future trial - Read “Conceptualizing Modifications & Identifying Validation Procedures for PRO Instruments”; Shields, A., Gwaltney, C., Paty, J., Shiffman, S., White Paper, 2006
Summary: Clinical researchers are challenged by the FDA's reference to evaluating modified PRO tools as if they were newly developed instruments (Draft PRO Guidance for Industry, Federal Register, Vol. 71, No. 23; Friday, Feb. 3, 2006; Lines 178-179). The primary goals of this paper are to assist researchers as they conceptualize the level of modification they make to their existing PRO instruments and identify the procedures that may be necessary to validate that change. - Read “Distinguishing among Symptom vs. Health Related Quality of Life PRO Concepts: Developing a Conceptual Framework”; Shields, A., Gwaltney, C., Paty, J., Shiffman, S., White Paper, 2006
Summary: The goal of this paper is to evaluate differences between symptom and health related quality of life PRO assessments and evaluate the implications of these differences in terms of what information clinical researchers should consider providing to the FDA to justify their use in some future trial. - Read “Equivalence of Computerized and Paper and Pencil PRO Measures White Paper”; Gwaltney, C., Shields, A., Shiffman, S., White Paper, 2006
Summary: The FDA draft guidance on PROs states that migrating from paper to computer will be considered a modification of the measure, which may require additional validation evidence. This paper is an overview of a meta-analysis of published studies that compared the two modes of administration. concluding that use of computerized measures in clinical trials should not be impeded by concerns about equivalence. - View Webinar Replay: Migration of PRO Measures from Paper to Electronic: Theoretical & Practical Perspectives
Summary: The FDA's Draft Guidance on Patient-Reported Outcome (PRO) assessments lays out principles for validating PRO instruments, and for ensuring valid migration from paper to electronic platforms (ePRO). We present a discussion of current scientific views on the validation activities required to migrate PRO measures from paper to electronic; a discussion on the recent meta-analysis on the impact of moving from paper to electronic, and practical strategies to successfully migrate from paper to electronic in the context of the FDA draft PRO Guidance. - View Webinar Replay: Practical Implications of the FDA's Draft Guidance
Summary: A Web Seminar discussing life after the FDA’s Draft PRO Guidance. Covered topics included: Meaning of “Conceptual Framework” as defined by the FDA; Best practices in defining a solid endpoint model; Necessary steps to determine the validity of a PRO measure; and, Practical steps to migrate from paper to electronic patient reported outcomes (PROs).