Chair:
Louis Matza PhD, Research Scientist, Center for Health Outcomes Research, United BioSource Corporation

Leadership Team:
John J. Alexander MD MPH, Medical Team Leader, Division of Anti-Infective and Ophthalmology Products, FDA, Silver Spring, MD, USA
Monika Bullinger MD, PhD, Professor & Vice Chief, Institute and Polyclinic for Medical Psychology, University Clinics-Eppendorf, University of Hamburg, Hamburg, Germany
Donald L. Patrick PhD, MSPH, Professor, Department of Health Services, University of Washington, Seattle, WA, USA
Andreas M. Pleil PhD, Senior Director / Lead, Ophthalmology and Endocrinology Outcomes Research, Clinical Development & Medical Affairs, Specialty Care Business Unit, Pfizer Global Pharmaceuticals, Pfizer, Inc,
San Diego, CA, USA
Luis Rajmil MD, PhD, MPH, Senior Researcher, Catalan Agency for Health Technology Assessment & Research (CAHTA), & Collaborator, Health Services Research Unit, Municipal Institute of Medical Research (IMIM-Hospital del Mar), Barcelona, Spain
Anne W. Riley PhD, MS, Professor, Dept of Population, Family & Reproductive Health, Bloomberg School of Public Health, Johns Hopkins University, Baltimore, MD, USA 

Goal:
The task force will first identify the key challenges of pediatric PRO research.  Then the group will work individually and collaboratively to provide specific recommendations for addressing these challenges in light of the current regulatory environment.  Some of the recommendations are likely to focus on the following issues:

• Challenges related to developmental differences and age-based cutoffs:
• The youngest age at which PRO measures can be used: This lower age limit is likely to differ depending on the construct being assessed.  Younger children may be able to reliably report their own experiences of more concrete domains (e.g., symptoms), while more abstract domains (e.g., health-related quality of life) may only be appropriate for older children or adolescents.
• The age at which most adolescents can be expected to report their own health and functioning as reliably as adults.
• Determination of appropriate age groupings to account for developmental differences.
• The question of parent report: Are there constructs or particular types of instruments for which the FDA might accept the reports of parents?  For observable behaviors or symptoms, parent-report measures may be preferable to child-report measures.  For other constructs, such as feelings, are parent reports ever valid in application to specific child disorders?
• Methods for ensuring that PROs use age-appropriate vocabulary and recall periods.
• Strategies for instrument administration and formatting.

Background / Overview:
A patient-reported outcomes (PRO) measure is any report coming directly from patients about how they feel or function in relation to their health and treatment.  In recent years, clinical trials evaluating medical product effectiveness have increasingly incorporated PROs because of the unique information these measures can provide.  Many aspects of medical conditions are known only by the patients themselves, and direct assessment of the patient perspective is necessary to thoroughly understand patients’ experiences of disease and treatment.

The FDA draft guidance on the use of PROs has shaped much of the industry-funded PRO research during the past two years (Food & Drug Administration 2006, http://www.ispor.org/workpaper/FDA%20PRO%20Guidance.pdf ).  This document provides detailed a description of PROs and the reasons for their use, as well as guidance for developing and evaluating these instruments.  In a brief section of the draft guidance (Section IV.E.1), it is acknowledged that development and validation of PROs for use in pediatric samples introduces unique challenges not faced in research with adults. 

Several challenges are mentioned, including age-related vocabulary, comprehension of the health concept measured, and the need to determine the lower age limit at which children can provide reliable and valid responses.  However, no specific guidance is provided for addressing these challenges.  The purpose of this task force is to produce a manuscript that will provide specific recommendations for the assessment of patient-reported outcomes in children and adolescents.

The challenges of choosing, developing, and implementing PRO measures in children have been reviewed and discussed in several previously published articles.  This manuscript will differ from this previous work because it will discuss these issues within the regulatory context and provide specific recommendations on methods designed to supplement the FDA guidance document.

Timeline effective 3/31/2010:

Task Forces Index