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ePRO Resource Center > FDA Guidance - Patient Reported Outcomes (PRO)
FDA's Guidance on Patient Reported Outcomes in Clinical Research
In December, 2009 the FDA issued its finalized Guidance on Patient Reported Outcomes (PRO) measures, capturing the FDA’s current thinking on PRO data capture in clinical research. The final Guidance 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 Guidance on PROs:
- Webinar Replay: "Implementing PROs in Clinical Trials"
Summary: This webinar is the third part of invivodata's 2010 PROficiency webinar series which focuses on the FDA's final PRO Guidance. In this session, Dr. Jean Paty will review specific considerations that biopharma researchers need to keep in mind when implementing PROs in clinical trials. The webinar will focus on specific elements of PRO trial design, including blinding and randomization, missing data, and clinical interpretation of the PRO data. This will be followed by a detailed discussion on methods of PRO administration in the context of trial execution.
- Webinar Replay: "How to Effectively Incorporate PROs into Clinical Trials"
Summary: In this webinar, invivodata's Dr. Jean Paty addressed specific implications of the final PRO Guidance and what it means for your clinical development program, including the design of your PRO measures.
- Read "The PROs of ePRO"; The Monitor, Feb., 2010; Waife, R.
Summary: In this article, industry consultant Ron Waife of Waife & Associates reviews the "pros" of ePRO in three ways: pros, pro's, and prose. The article highlights of capturing PRO data electronically, how professional ePRO providers deliver value, and the need for more information about when and how ePRO should be added to the clinical development program.
- Read "Recommendations on Evidence Needed to Support Measurement Equivalence between Electronic and Paper-Based Patient-Reported Outcome (PRO) Measures: ISPOR ePRO Good Research Practices Task Force Report"; Coons, S., Gwaltney, C., Hays, R., Lundy, J., Sloan, J., Revicki, D., Lenderking, W., Cella, D., Basch, E., on behalf of the ISPOR ePRO Task Force, Value in Health, November, 2008
Summary: The article presents recommendations from ISPOR’s ePRO Good Research Practices Task Force regarding the evidence necessary to support the comparability, or equivalence, of ePROs to the paper-based PRO measures from which they were adapted. It provides a general framework for decisions regarding the level of evidence needed to support modifications that are made to PRO measures when they are migrated from paper to ePRO devices in accordance with the current U.S FDA’s Draft Guidance on PROs in Clinical Research.
- Read, “Evaluating & Documenting Content Validity for the Use of Existing PRO Instruments & Their Modification”; Rothman, M., Burke, L., Pennifer, E., Kline-Leidy, N., Patrick, D.L., Petrie, C.D., on behalf of the ISPOR ePRO Task Force, Value in Health, November, 2009
Summary: The article presents recommendations from ISPOR’s ePRO Good Research Practices Task Force for evaluating and documenting content validity for the use of existing instruments and their modification. It discussed the key issues involved in assessing and documenting the content validity of an existing instrument, including concept clarification, instrument identification, and initial review, as well as qualitative methods as they might be used to evaluate the suitability of one or more existing instruments.
- Read, “Translation & Linguistic Validation of PRO Instruments”; Wilde, D., Eremenco, S., Mear, I., Martin, M., Houchin, C., Gawlicki, M., Hareendran, A., Wiklund, I., Chong, L., Von Maltzahn, R., Cohen, L., Molsen, E., ., on behalf of the ISPOR ePRO Task Force, Value in Health, November, 2009
Summary: The article presents recommendations from ISPOR’s ePRO Good Research Practices Task Force on the translations required and approaches to using the same language in different countries, and the approaches to support pooling the data. It is hoped that this report and the decision tools proposed will assist those involved with multinational trials to 1) decide on the translations required for each country; 2) choose the approach to use when the same language is spoken in more than one country; and 3) choose methods to gather evidence to support the pooling of data collected using different language versions of the same tool.
- Download Whitepaper "Answering Questions about FDA's Draft Guidance on Patient Reported Outcomes (PROs)"; Shields, December, 2008.
Summary: The FDA Draft Guidance provides structure for a more efficient, effective and appropriate use of PROs in clinical research. Challenges exist, however, in interpreting various aspects of this document. The goal of this White Paper is to provide answers to questions frequently asked by researchers as they develop their PRO strategies, based on information gathered from the Draft Guidance and FDA presentations and publications that have emerged following its issue.
- Read about the Process for Developing an Endpoint Model and other aspects of the FDA's Draft Guidance. CenterWatch Weekly, Sept. 15, 2008
Summary: In this industry profile, Dr. Jean Paty, Chief Scientist and Regulatory Advisor of PRO Consulting discusses the FDA's draft guidance on PROs in clinical research and the importance of measuring patient-reported endpoints from a regulatory perspective, including the process for developing an endpoint model to meet regulatory expectations.
- 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, April, 2008 (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, “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, March 2008 (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 “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).