Abstract
ObjectiveTo provide an assessment of the quality of the most frequently used self-reported, generic patient-reported outcome measures (PROMs) that measure health-related quality of life (HRQoL) in children against the good research practices recommended by ISPOR task force for the pediatric population.MethodLiterature search was conducted on OvidSP database to identify the generic pediatric PROMs used in published clinical studies. The quality of PROMs used in more than ten clinical studies were descriptively evaluated against the ISPOR task force’s good research practices.ResultsSix PROMs were evaluated, namely Pediatric Quality-of-Life inventory 4.0 (PedsQL), Child Health Questionnaire (CHQ), KIDSCREEN, KINDL, DISABKIDS and Child Health and Illness Profile (CHIP). All PROMs, except KIDSCREEN, had versions for different age ranges. Domains of physical, social, emotional health and school activities were common across all the instruments, while domains of family activities, parent relations, independence, and self-esteem were not present in all. Children’s input was sought during the development process of PROMs. Likert scales were used in all the instruments, supplemented with faces (smileys) in instruments for children under 8 years. KIDSCREEN and DISABKIDS were developed in a European collaboration project considering the cross-cultural impact during development.ConclusionThe comparison of the instruments highlights differences in the versions for different pediatric age groups. None of the PROMs fulfill all the good research practices recommended by the ISPOR task force. Further research is needed to define which age-appropriate domains are important for older children and adolescents.
Highlights
As with the adult population, establishing the health-related quality of life (HRQoL) for the pediatric population is essential
Of the generic HRQoL measures identified, we evaluated the quality of Patient-reported outcome measures (PROMs) used in more than ten clinical trials against the “Good Research Practices” recommended by Matza et al (Supplementary Table 2)
The International Society for Pharmacoeconomics and Outcomes Research (ISPOR) task force paper recommends using different age ranges within the pediatric population that serve as a starting point to determine developmental differences and age-based content requirements for patient-reported outcomes (PRO) assessment
Summary
As with the adult population, establishing the health-related quality of life (HRQoL) for the pediatric population is essential. It is increasingly incorporated as outcome endpoints in pediatric clinical trials [1]. Patient-reported outcome measures (PROMs) are used in USA clinical trials to assess a child’s functioning and evaluate health status improvement by medical intervention. The patient-reported outcomes (PRO) Guidance to Industry, laid out by the U.S Food and Drug Administration (FDA), supports the development of validated PROMs to collect PRO data. The FDA PRO guidance states that for pediatric PROMs, the vocabulary, comprehension, and minimum age at which the children can provide valid and reliable responses must be considered.
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