TY - JOUR
T1 - Path towards efficient paediatric formulation development based on partnering with clinical pharmacologists and clinicians, a conect4children expert group white paper
AU - Walsh, Jennifer
AU - Schaufelberger, Daniel
AU - Iurian, Sonia
AU - Klein, Sandra
AU - Batchelor, Hannah
AU - Turner, Roy
AU - Gizurarson, Sveinbjörn
AU - Boltri, Luigi
AU - Alessandrini, Elisa
AU - Tuleu, Catherine
N1 - Funding Information:
Conect4children has received funding from the Innovative Medicines Initiative 2 Joint Undertaking under grant agreement No 777389. The Joint Undertaking receives support from the European Union's Horizon 2020 research and innovation programme and EFPIA. Conect4children has received funding from the Innovative Medicines Initiative 2 Joint Undertaking under grant agreement No 777389. The Joint Undertaking receives support from the European Union's Horizon 2020 research and innovation programme and EFPIA.
Funding Information:
Noteworthy, conect4children ( www.conect4children.org/ , c4c) is a public–private consortium funded by Innovative Medicines Initiative 2 aimed to create a sustainable infrastructure promoting innovation in the design and conduct of paediatric clinical trials. One of the c4c methodology expert groups (work package 4) focuses on formulation for children (present authorship) and provides advice on formulation aspects during children's drug development. The group works closely with other dedicated paediatric scientific initiatives, such as the European Paediatric Formulation Initiative ( www.eupfi.org/ ), the European Paediatric Translational Research Infrastructure (work package 8; www.eptri.eu/work-packages/wp8-thematic-platform-on-formulation-science/ ), and the International Consortium for Innovation and Quality in Pharmaceutical Development, Pediatric Working Group ( www.iqconsortium.org ). This work aims to combine expert opinions and recommendations of the c4c formulation group. In this paper certain key areas where the can be further improved are identified and ways for a closer collaboration between formulation scientists and their colleagues in Clin Pharm and Clinical R&D are suggested. path to better paediatric formulations
Publisher Copyright:
© 2021 British Pharmacological Society.
PY - 2022/12
Y1 - 2022/12
N2 - 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.
AB - 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.
KW - clinical pharmacology
KW - drug delivery
KW - drug development
KW - paediatrics
KW - pharmacy
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U2 - 10.1111/bcp.14989
DO - 10.1111/bcp.14989
M3 - Review article
C2 - 34265091
AN - SCOPUS:85112376886
SN - 0306-5251
VL - 88
SP - 5034
EP - 5051
JO - British Journal of Clinical Pharmacology
JF - British Journal of Clinical Pharmacology
IS - 12
ER -