Path towards efficient paediatric formulation development based on partnering with clinical pharmacologists and clinicians, a conect4children expert group white paper

Jennifer Walsh, Daniel Schaufelberger, Sonia Iurian, Sandra Klein, Hannah Batchelor, Roy Turner, Sveinbjörn Gizurarson, Luigi Boltri, Elisa Alessandrini, Catherine Tuleu

Research output: Contribution to journalReview articlepeer-review

Abstract

Improved global access to novel age-appropriate formulations for paediatric subsets, either of new chemical entities or existing drugs, is a priority to ensure that medicines meet the needs of these patients. However, despite regulatory incentives, the introduction to the market of paediatric formulations still lags behind adult products. This is mainly caused by additional complexities associated with the development of acceptable age-appropriate paediatric medicines. This position paper recommends the use of a paediatric Quality Target Product Profile as an efficient tool to facilitate early planning and decision making across all teams involved in paediatric formulation development during the children-centric formulation design for new chemical entities, or to repurpose/reformulate off-patent drugs. Essential key attributes of a paediatric formulation are suggested and described. Moreover, greater collaboration between formulation experts and clinical colleagues, including healthcare professionals, is advocated to lead to safe and effective, age-appropriate medicinal products. Acceptability testing should be a secondary endpoint in paediatric clinical trials to ensure postmarketing adherence is not compromised by a lack of acceptability. Not knowing the indications and the related age groups and potential dosing regimens early enough is still a major hurdle for efficient paediatric formulation development; however, the proposed paediatric Quality Target Product Profile could be a valuable collaborative tool for planning and decision making to expedite paediatric product development, particularly for those with limited experience in developing a paediatric product.

Original languageEnglish
Number of pages18
JournalBritish Journal of Clinical Pharmacology
Early online date14 Aug 2021
DOIs
Publication statusE-pub ahead of print - 14 Aug 2021

Keywords

  • clinical pharmacology
  • drug delivery
  • drug development
  • paediatrics
  • pharmacy

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