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ORIGINAL RESEARCH article

Front. Pharmacol.
Sec. Obstetric and Pediatric Pharmacology
Volume 15 - 2024 | doi: 10.3389/fphar.2024.1436554

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

Provisionally accepted
Juliane Münch Juliane Münch 1Anna L. Schwarzwälder Anna L. Schwarzwälder 1Carolin Kloft Carolin Kloft 1Hans M. Bosse Hans M. Bosse 1Manfred Wargenau Manfred Wargenau 2Sibylle Reidemeister Sibylle Reidemeister 3Ingrid Klingmann Ingrid Klingmann 4Viviane Klingmann Viviane Klingmann 1*
  • 1 Department of General Pediatrics, Neonatology and Pediatric Cardiology, University Hospital of Düsseldorf, Duesseldorf, Germany
  • 2 M.A.R.C.O. GmbH & Co. KG, Institute for Clinical Research and Statistics, Duesseldorf, Germany
  • 3 Novartis Pharma AG, Global Drug Development/Technical Research & Development, Novartis Campus, Basel, Switzerland
  • 4 Pharmaplex bv, Wezembeek‑Oppem, Belgium

The final, formatted version of the article will be published soon.

    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 3 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 4 sequences with 3 out of 4 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 3 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.

    Keywords: Acceptability, palatability, oral pediatric formulations, Swallowability, Drug administration

    Received: 22 May 2024; Accepted: 29 Jul 2024.

    Copyright: © 2024 Münch, Schwarzwälder, Kloft, Bosse, Wargenau, Reidemeister, Klingmann and Klingmann. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.

    * Correspondence: Viviane Klingmann, Department of General Pediatrics, Neonatology and Pediatric Cardiology, University Hospital of Düsseldorf, Duesseldorf, Germany

    Disclaimer: All claims expressed in this article are solely those of the authors and do not necessarily represent those of their affiliated organizations, or those of the publisher, the editors and the reviewers. Any product that may be evaluated in this article or claim that may be made by its manufacturer is not guaranteed or endorsed by the publisher.