Patient Reported Outcomes (PRO) Overview

Patient Reported Outcomes (PRO) data represent an increasingly important component of new drug, biologics, and medical device development. As such, a wealth of information has been published on the merits of PRO data and the appropriate methods for capturing PRO data to support a trial’s primary efficacy endpoints.

To access any of the patient reported outcomes articles listed below, select the corresponding link.

• “Measurement is Strategic“; 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 instrumentsused to evaluate a product’s efficacy and support its registration and labeling are “fit for purpose”.

• “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.

• “Two Decades of Change for PROs: How patient-reported data collection has been transformed since 1987“; Shiffman, S., Applied Clinical Trials, 2008
  Summary: In this commentary, Dr. Shiffman reflects on how Patient Reported Outcomes (PROs) have changed since he started using electronic diaries in clinical research twenty years ago, including examples of how PROs have grown more important to drug development, more scientifically robust, and more technologically sophisticated.

• “The Past, Present, and Future of Capturing Subject’s Experience in Clinical Trials”; Paty, J., Engfer, D., Good Clinical Practice Journal, 2003
  Summary:A growing trend in clinical research is to directly ask subjects to report on their experience with the drug. The primary shortcomings of this approach are that most data is captured only at the clinical research site, meaning we only obtain a brief snapshot of the drug’s effect on the subject. This brief article discusses how technology will enable clinical research to move beyond the brief snapshot view of drug impact.

• “Methodological Issues Affecting the Value of Patient Reported Outcomes Data”; Hufford, M., Shiffman, S., Expert Rev. Pharmacoeconomics Outcomes Res., 2002
  Summary:This review examines the impact of a variety of methodological issues on the value of patient reported outcomes data. In particular, when patients are asked to self-monitor their experiences over time, a variety of methodological issues must be addressed if the data are to be considered a reliable and valid reflection of their daily lives.

• “Subject Experience Diaries in Clinical Research Parts I & II”; Shiffman, S., Hufford, M., Paty, J., Applied Clinical Trials, 2001
  Summary: The first of this two-part article explores the meaning and importance to clinical research of subject experience data. It also reviews the methods used to collect the data, focusing in particular on paper and electronic subject experience diaries. The second part discusses the scientific principles underlying ecological momentary assessment and how these principles can enhance protocol design and measurement sensitivity.

The patient reported outcomes articles above are just a small sample of our ePRO resources available.  Check back regularly for new information on patient reported outcome data collection and other PRO news.