Published Jul 2013
Matza LS, Patrick D, Riley AW, et al. Pediatric patient-reported outcome instruments for research to support medical product labeling: report of the ISPOR PRO Good Research Practices for the Assessment of Children and Adolescents Task Force. Value Health. 2013; 16(4):461-479.
Background: Patient-reported outcome (PRO) instruments for children
and adolescents are often included in clinical trials with the
intention of collecting data to support claims in a medical
product label. Objective: The purpose of the current task force report
is to recommend good practices for pediatric PRO research that is
conducted to inform regulatory decision making and support claims
made in medical product labeling. The recommendations are based
on the consensus of an interdisciplinary group of researchers who
were assembled for a task force associated with the International
Society for Pharmacoeconomics and Outcomes Research (ISPOR). In
those areas in which supporting evidence is limited or in which
general principles may not apply to every situation, this task force
report identifies factors to consider when making decisions about the
design and use of pediatric PRO instruments, while highlighting
issues that require further research.
Good Research Practices: Five good research practices are discussed: 1) Consider developmental differences and determine age-based criteria for PRO administration: Four age groups are discussed on the basis of previous research (<5 years old, 5–7 years, 8–11 years, and 12–18 years). These age groups are recommended as a starting point when making decisions, but they will not fit all PRO instruments or the developmental stage of every child. Specific age ranges should be determined individually for each population and PRO instrument. 2) Establish content validity of pediatric PRO instruments: This section discusses the advantages of using children as content experts, as well as strategies for concept elicitation and cognitive interviews with children. 3) Determine whether an informant-reported outcome instrument is necessary: The distinction between two types of informant-reported measures (proxy vs. observational) is discussed, and recommendations are provided. 4) Ensure that the instrument is designed and formatted appropriately for the target age group. Factors to consider include health-related vocabulary, reading level, response scales, recall period, length of instrument, pictorial representations, formatting details, administration approaches, and electronic data collection (ePRO). 5) Consider cross-cultural issues.
Conclusions: Additional research is needed to provide methodological guidance for future studies, especially for studies involving young children and parents’ observational reports. As PRO data are increasingly used to support pediatric labeling claims, there will be more information regarding the standards by which these instruments will be judged. The use of PRO instruments in clinical trials and regulatory submissions will help ensure that children’s experience of disease and treatment are accurately represented and considered in regulatory decisions.
Keywords: adolescents, children, ISPOR, medical product labeling, patient-reported outcomes, pediatrics, PRO, task force.
Copyright © 2017, International Society for Pharmacoeconomics and Outcomes Research (ISPOR). Published by Elsevier Inc.