PRO Task Force: Changing Mode of Administration of Instruments/ePRO

Goal:
Development of good research practices for changing the mode of administration (e.g. paper to computer, supervised to self questionnaire) and use of electronic PRO instruments.

Task Force Chair:
Stephen Joel Coons, PhD, Professor of Pharmacy and Public Health, University of Arizona, Tucson, AZ

Leadership Team:
Ethan Basch, MD, MSc, Health Outcomes Research Group, Departments of Biostatistics and Medicine Memorial Sloan Kettering Cancer Center, New York, NY
Laurie B. Burke, RPh, MPH, Director, Study Endpoints and Label Development Office of New Drugs, CDER, FDA Washington, DC
Don Bushnell, MA, Associate Director, Health Research Associates, Seattle, WA
David Cella, PhD, Professor, MED-Psych & Behavioral Science, Northwestern University, Evanston, IL
Paula Funk Orsini, PhD, Principal, Funk & Orsini Associates Scarsdale, NY
Chad J. Gwaltney, PhD, Center for Alcohol and Addiction Studies, Brown University, Providence, RI and ePRO Consultant, Invivodata
Ron D. Hays, PhD, Professor, UCLA Department of Medicine, Division of General Internal Medicine & Health Services Research, Los Angeles, CA
William Lenderking, PhD, Director/Team Leader, Outcomes Research Group, Pfizer Inc., Groton, CT
Dennis Revicki, PhD, Director, Center for Health Outcomes Research The MEDTAP Institute at UBC Bethesda, MD
James W. Shaw, PhD, PharmD, MPH, Assistant Professor, Department of Pharmacy Administration, University of Illinois at Chicago, Chicago, IL
Saul Shiffman, PhD, Senior Scientific Advisor, Pinney Associates, Bethesda, MD and Professor, Psychology & Pharmaceutical Science, University of Pittsburgh, Pittsburgh, PA
Jeff Sloan, PhD, Professor, Division of Biostatistics, Mayo Clinic College of Medicine, Rochester, MN
Theron Taber, BS, President, Assist Technologies Scottsdale, AZ
Brian Tiplady, PhD, Senior Clinical Scientist, Invivodata, Edinburgh, Scotland
Keith Wenzel, BS, ePRO Principal, ClinPhone, Oregon, WI
Arthur Zbrozek, MSc, MBA, Senior Director, Wyeth Research, Philadelphia, PA

Meeting Presentation
Third Plenary:  Patient Reported Outcomes: Implementing Good Research Practices
Tuesday, May 6 3:45 – 4:45 PM in the Grand Ballroom Center (Lower Concourse)
http://www.ispor.org/meetings/toronto0508/p050608.asp#Plenary3

Background:
Concurrent with the increased use and significance of PROs in clinical trials has been the steady growth in electronic data capture (EDC) or eSourcing—the electronic collection of clinical trial data at the source (e.g., case report forms and PROs). EDC refers to the use of electronic systems for capturing clinical data in trials, and usually refers to systems where data are entered by research staff. ePRO refers to systems where electronic devices (e.g., computers) are used to capture data directly from the patient.

In February 2006, the U.S. Food and Drug Administration issued a draft version of Guidance for Industry titled “Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims.”

Draft FDA Guidance for Industry - Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims Draft Guidance

In Chapter IV: Evaluating PRO Instruments, Part D: Modification of an Existing Instrument, Section 4 addresses Changed Mode of Administration:

An instrument’s data collection mode is altered.  For example:

• An interviewer-administered or supervised questionnaire is modified for self-administration (skip patterns can be a problem in this situation)
• Paper-and pencil self-administered PRO is modified to be administered by computer or other electronic device (e.g., computer adaptive testing, interactive voice response systems, Web-based questionnaire administration, computer)
• Instructions or procedures for administration within a trial differ from those used in validation studies (can alter the meaning of the responses from that of the original version)

The development of new PRO measures, specifically for EDC platforms, and the migration of existing paper-based PRO measures to electronic formats, present unique opportunities and challenges in outcomes research. 

Adaptation of clinical trial documents to eSource formats, including ePROs, must be accomplished in ways that assure the equivalence of data collected via paper and electronic methods.  Many PRO measures were originally developed to be administered via paper-based questionnaires; however, as with EDC in general, there is growing recognition of the many advantages of ePROs, including less administrative and subject burden, avoidance of secondary data entry errors, easier implementation of skip patterns, and more accurate and complete data.

The purpose of this task force is to set forth recommendations as to the evidence necessary to support the comparability or measurement equivalence of ePROs to the original paper-based PRO questionnaire from which they were adapted.

Migrating from paper-based modes of administration to electronic data collection devices is one of the most significant movements in the PRO measurement field.  Hence, in addressing the issue of changing modes of administration for PRO measures, ePRO will be the exemplar used by the task force.  Nevertheless, the good research practices developed are intended to be relevant to other administration mode changes.

Task Force Actions:
The Task Force document titled, "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" has been accepted for publication in Value in Health

The report considers the following issues, which begin the process of identifying relevant and unresolved issues that must be considered in establishing standards for the adaptation, testing, and application of ePRO measures:

• What are the advantages and disadvantages of electronic capture of PRO data (e.g., better data quality, less missing data, easier implementation of skip patterns, immediate creation of electronic dataset, the ability to see real-time data reporting, avoidance of secondary data entry errors vs. sample selection bias [computer literacy], patient burden, training, expense, validation requirements, technology/ communication requirements specifically in developing countries)?
• When does the increased functionality of electronic data capture outweigh the potential disadvantages?
• What are the subpopulations for which ePROs may be less suited (e.g., older adult, visually impaired, or pediatric patients) and how could ePRO overcome these limitations?
• What are the challenges (e.g., technological, legal/regulatory, financial) in converting a paper PRO to an ePRO?
• Who should be involved in the process of converting paper PROs to ePROs (e.g., what is the role of PRO developer, clinical trial sponsor, eVendor)?
• What are the best practices for converting a paper PRO to an ePRO?
• What serves as the “gold standard” if ePRO measures result in responses that are different from the corresponding paper-based PRO measure (e.g., due to more complete and less variable ePRO data)?