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Measuring What Matters for Children: A Systematic Review of Frequently Used Pediatric Generic PRO Instruments

Tasneem Arsiwala1, Nuzhat Afroz2, Kattayoun Kordy3

  • 1Novartis Pharma AG, Basel, Switzerland. tasneem.arsiwala@novartis.com.

Therapeutic Innovation & Regulatory Science
|June 18, 2021
PubMed

Insights

No current patient-reported outcome measures (PROMs) fully meet ISPOR standards for assessing pediatric health-related quality of life (HRQoL). Further research is needed to refine age-appropriate domains for children and adolescents.

Area of Science:

  • Pediatric Health Outcomes Research
  • Quality of Life Measurement
  • Clinical Assessment Tools

Background:

  • Health-related quality of life (HRQoL) in children is increasingly assessed using patient-reported outcome measures (PROMs).
  • Generic PROMs are frequently used in pediatric clinical studies, necessitating an evaluation of their quality.
  • The ISPOR task force provides good research practice guidelines specifically for pediatric populations.

Purpose of the Study:

  • To assess the quality of frequently used generic PROMs for measuring pediatric HRQoL.
  • To evaluate these PROMs against ISPOR's good research practice recommendations for children.
  • To identify gaps in current PROMs for pediatric populations.

Main Methods:

  • A literature search identified generic pediatric PROMs used in over ten clinical studies.
  • Six PROMs were descriptively evaluated: PedsQL, CHQ, KIDSCREEN, KINDL, DISABKIDS, and CHIP.
  • Evaluation criteria included age-appropriateness, domain coverage, and developmental input.

Main Results:

  • Six generic PROMs were evaluated, with most offering age-specific versions.
  • Common domains included physical, social, and emotional health; however, family, independence, and self-esteem domains varied.
  • Children's input was incorporated during development, and Likert scales were common, with visual aids for younger children.

Conclusions:

  • None of the evaluated generic PROMs fully adhere to all ISPOR good research practices for pediatric populations.
  • Significant differences exist in age-specific versions of PROMs.
  • Further research is required to establish age-appropriate domains, particularly for older children and adolescents.
Abstract

Keywords:
AdolescentsChildrenHealth-related quality of lifeISPORPatient-reported outcomesPediatrics

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