Recent PRO Publications

Are you looking for the most current information on Patient Reported Outcomes data capture? Click any link below to access some of the most recently published literature available.

• Read “The Evidence for Endpoints:  PROs and Beyond”; Paty, J., Life Science Leader; January 2012
  Summary:  In October 2011 FDA hosted a workshop that outlined their expectations for the development and qualification to establish that Outcome Assessments (OAs) – including Patient Reported Outcomess (PROs), Clinician Reported Outcomes (ClinROs), and Observer Reported Outcomes (ObsROs) – are fit for purpose and appropriate for their context of use. This article reviews the key take-aways of the workshop and its implications to researchers using OAs in new medical product development.

• Read “Content Validity—Establishing and Reporting the Evidence in Newly Developed Patient-Reported Outcomes (PRO) Instruments for Medical Product Evaluation: ISPOR PRO Good Research Practices Task Force Report: Part 1—Eliciting Concepts for a New PRO Instrument”; Patrick, B., Burke, L., Gwaltney, C., Kline-Leidy, N., Martin, M., Molsen, E., Ring, L.  Value in Health; October 2011.
  Summary: In this first of a 2-part ISPOR PRO Good Research Practices Task Force Report, the authors review the process for developing content and assessing respondent understanding of newly developed PRO instruments for medical product evaluation, specifically the elicitation of key concepts using qualitative focus groups and/or interviews to inform content and structure of a new PRO instrument.

• Read “Content Validity—Establishing and Reporting the Evidence in Newly Developed Patient-Reported Outcomes (PRO) Instruments for Medical Product Evaluation: ISPOR PRO Good Research Practices Task Force Report: Part II—Assessing Respondent Understanding”; Patrick, B., Burke, L., Gwaltney, C., Kline-Leidy, N., Martin, M., Molsen, E., Ring, L.  Value in Health; October 2011.
  Summary:  In this second of a 2-part ISPOR PRO Good Research Practices Task Force Report, the authors review the process for developing content and assessing respondent understanding of newly developed PRO instruments for medical product evaluation, specifically, the methods for conducting cognitive interviews that address patient understanding of items, instructions, and response options; and the methods for tracking item development through the various stages of research and preparing this tracking for submission to regulatory agencies.

• Read “The Patient Matters: Thanks FDA”. Paty, J., Applied Clinical Trials, February 2011
  Summary:  In this ‘Closing Thought’ article, Dr. Paty reviews the impact of the FDA’s Final Guidance on Patient Reported Outcomes (2009) in terms of its effect in driving the biopharmaceutical industry to take note of the patient – to listen, understand, and document the patient’s perspective during medical product development.

• Read “Real-Time Patient Reporting Could Open The Door To FDA Inspection”. Dubin, C., Life Science Leader, February 2011
  Summary: invivodata’s successful DiaryPRO and SitePRO study with Sucampo is highlighted in the February issue of Life Science Leader. In this article by Cindy Dubin, invivodata’s Gretchen Craig and Sucampo’s Dr. Raymond Panas discuss ePRO and the FDA inspections that resulted in the approval of Sucampo’s drug, Amitiza.

• Read “ePRO:  Electronic Solutions for Patient-Reported Data“; Edited by Bill Byrom and Brian Tiplady, Gower Publishing, 2010.
  Summary:  Recently, there has been much open debate with regulators around the use of ePRO in clinical drug submissions. US and European agencies have approved new drugs that have included ePRO data in the submission dossier, but there are many questions around the adoption of the technology that concern the community.  In this, the industry’s first textbook dedicated solely to ePRO, Bill Byrom and Brian Tiplady address all these issues, review  the new FDA guidance on PROs in Clinical Research, and provide a very contemporary view on this important subject.

Download a sample chapter, “Diary Design Considerations: Interface Issues and Patient Acceptability” or order the text directly through the publisher.

• Read “Electronic Patient Reported Outcomes (ePRO): Focus on Site-Based Assessments“; Tiplady, B., October 2010
  Summary:  In this White Paper, Dr. Brian Tiplady reviews the extensive literature on electronic forms of site-based PRO data collection, addresses issues regarding patient acceptance and whether ePRO can be used interchangeably with paper questionnaires, and offers recommendations for obtaining the best value when collecting patient assessments electronically at the investigative site.

• Read “Measurement is Strategic” (registration required); Paty, J., Applied Clinical Trials, October 2010
  Summary: In this article, PRO Consulting Chief Scientist and Regulatory Advisor, Dr. Jean Paty poses the question, “When should researchers think about how to best measure their products’ efficacy?” and provides a framework for ensuring that all PRO instruments used to evaluate a product’s efficacy and support its registration and labeling are “fit for purpose”.

• Read ”ePRO Regulatory Inspections: Best Practices for Smooth and Successful Outcomes“, Craig, G., September 2010
  Summary: In this White Paper, members of invivodata’s ePRO Inspection Team review our experiences in supporting and participating in ePRO regulatory inspections by the U.S. Food and Drug Administration (FDA), European Medicines Agency (EMA), specifically BfArM (German Federal Institute for Drugs and Medical Devices), and the Japanese Pharmaceutical and Medical Devices Agency (PMDA), and outline best practices for ensuring smooth regulatory inspections of ePRO data collection systems.

• Read “Increasing Study Sensitivity Early in Trials”; Applied Clinical Trials, April 2010; Shiffman, S.
  Summary: This peer-reviewed article demonstrates why eDiaries are important in Phase II clinical trials, delivering a greater ability to detect treatment effects and reducing the size and costs of drug development programs.  Multiple case studies are presented, outlining how researchers who have adopted ePRO methods early in clinical development have gained greater insight into the full therapeutic potential of their candidate drugs.

• 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.