Validating a composite endpoint for acceptability evaluation of oral drug formulations in the pediatric population: a randomized, open-label, single dose, cross-over study

Abstract

Objective: This study aimed to validate the newly developed composite acceptability endpoint to investigate acceptability of oral pediatric drug formulations that integrates swallowability and palatability assessments.

Methods: In this open-label study acceptability of oral formulations was tested in three age groups (1-<6 months, 6-<12 years, and 12-<18 years) with a 2-way cross-over design in children aged 1-<6 months (syrup and mini-tablets), and with an incomplete block design of four sequences with three out of four formulations (syrup, mini-tablets, oblong tablet, and round tablet) each in children aged 6-<18 years. The primary endpoint was acceptability derived from the composite acceptability endpoint. Secondary endpoints were palatability and acceptability derived from swallowability.

Results: A total of 320 children were stratified into three age groups (80 children aged 1-<6 months, 120 children aged 6-<12 years, and 120 children aged 12-<18 years). All participants completed the study. Age-specific differences were observed in acceptability derived from the composite acceptability endpoint. Mini-tablets had the highest acceptability in participants aged 1-<6 months and 6-<12 years while the oblong tablet was leading in adolescent participants (12-<18 years).

Conclusion: This study demonstrated that the composite acceptability endpoint method integrating both swallowability and palatability assessments is a sensitive method to assess acceptability of drug formulations in children of different age.

Clinical trial registration: https://drks.de/search/de, identifier DRKS00027948.

Keywords: acceptability; drug administration; oral pediatric formulations; palatability; swallowability.