Placebo response in pediatric clinical trials dilutes drug-placebo differences and severely hinders drug development. However, the factors that are associated with placebo effects are poorly understood, with most evidence coming from post-hoc analyses of single trial data or single indication meta-analyses. The FDA pooled pediatric clinical trial data using randomized placebo-controlled trials would afford the possibility to evaluate predictors of the magnitude of placebo response across pediatric therapeutic areas.
Is there a difference in placebo response when comparing clinician-rated vs. caregiver-rated outcome measures?
Is there a difference in placebo response in outcomes measures using different settings to elicit clinical information, e.g. based on observation vs. interview vs. report?
How are placebo response rates associated with child (age, sex) or study characteristics (i.e. number of sites involved in a trial, ratio of participants in control vs. drug arms)?
Understanding commonalities of the placebo response in pediatric trials would help researchers understand their data better, power trials more accurately, and improve future trial design - therefore bringing effective medications to pediatric populations quicker and more efficiently.