Are Pediatric Trials Including More Patient-Centered Outcomes?
Author(s)
Nora Fayed, PhD1, Simrin Pardal, MScOT2, Colin MacArthur, PhD MD3;
1Queen's University, Associate Professor, Kingston, ON, Canada, 2Queen's University, Kingston, ON, Canada, 3The Hospital for Sick Children, Toronto, ON, Canada
1Queen's University, Associate Professor, Kingston, ON, Canada, 2Queen's University, Kingston, ON, Canada, 3The Hospital for Sick Children, Toronto, ON, Canada
OBJECTIVES: To describe child, family or caregiver-important outcomes in pediatric randomized controlled trials, from peer-reviewed literature, over a 10-year period.
METHODS: The search was conducted in Ovid MEDLINE from 2014 to June using a strategy to identify pediatric trials. Included trials: i) had clustered or individualized randomization, ii) focused on infants, children, adolescents or youth <25, or ii) were maternal-infant or parental trials including at least one endpoint of the infant/child. In phase 1 (2014-2019), all abstracts were screened and data extracted. In phase 2 (2019-2024), only 25% of abstract and full-text screening of the search was conducted by 2 reviewers.The data extracted were: i) report of patient engagement in trial endpoint selection or development; ii) inclusion of psychosocial, or perceived health or quality of life endpoints coded using standardized procedures, and iii) the tool, agent or respondent of the primary co-primary, and secondary outcomes. Trial characteristics such as: ID/registration #, inclusion age at eligibility, unit of randomization (individual or clustered), intervention type (pharmacological, surgical, medical-technology, other), trial region, and funding type (industry, NGO, government), were extracted.
RESULTS: 1100 randomized clinical trials demonstrated low rates of cited child, family or caregiver input or selection with trial endpoints <10% in each trial year. Specific child or youth engagement, (as opposed to parent/caregiver) increased from 3 to 8% over the 10-year period. Inclusion of psychosocial, perceived health or quality of life primary endpoints ranged from 31-40%, but increased for secondary endpoints from 15 to 30%.
CONCLUSIONS: Despite global initiatives to increase patient engagement in pediatric trials, child, parent, or caregiver engagement in selection of trial endpoint remains low. The doubling of psychosocial, perceived health or quality of life as secondary endpoints demonstrates potential for increasing demand for evidence based on patient-important outcomes.
METHODS: The search was conducted in Ovid MEDLINE from 2014 to June using a strategy to identify pediatric trials. Included trials: i) had clustered or individualized randomization, ii) focused on infants, children, adolescents or youth <25, or ii) were maternal-infant or parental trials including at least one endpoint of the infant/child. In phase 1 (2014-2019), all abstracts were screened and data extracted. In phase 2 (2019-2024), only 25% of abstract and full-text screening of the search was conducted by 2 reviewers.The data extracted were: i) report of patient engagement in trial endpoint selection or development; ii) inclusion of psychosocial, or perceived health or quality of life endpoints coded using standardized procedures, and iii) the tool, agent or respondent of the primary co-primary, and secondary outcomes. Trial characteristics such as: ID/registration #, inclusion age at eligibility, unit of randomization (individual or clustered), intervention type (pharmacological, surgical, medical-technology, other), trial region, and funding type (industry, NGO, government), were extracted.
RESULTS: 1100 randomized clinical trials demonstrated low rates of cited child, family or caregiver input or selection with trial endpoints <10% in each trial year. Specific child or youth engagement, (as opposed to parent/caregiver) increased from 3 to 8% over the 10-year period. Inclusion of psychosocial, perceived health or quality of life primary endpoints ranged from 31-40%, but increased for secondary endpoints from 15 to 30%.
CONCLUSIONS: Despite global initiatives to increase patient engagement in pediatric trials, child, parent, or caregiver engagement in selection of trial endpoint remains low. The doubling of psychosocial, perceived health or quality of life as secondary endpoints demonstrates potential for increasing demand for evidence based on patient-important outcomes.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
P34
Topic
Patient-Centered Research
Topic Subcategory
Patient-reported Outcomes & Quality of Life Outcomes
Disease
SDC: Pediatrics