Walsh, J;
Schaufelberger, D;
Iurian, S;
Klein, S;
Batchelor, H;
Turner, R;
Gizurarson, S;
... Tuleu, C; + view all
(2021)
Path towards efficient paediatric formulation development based on partnering with clinical pharmacologists and clinicians, a c4c expert group White paper.
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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.
| Type: | Working / discussion paper |
|---|---|
| Title: | Path towards efficient paediatric formulation development based on partnering with clinical pharmacologists and clinicians, a c4c expert group White paper |
| Open access status: | An open access version is available from UCL Discovery |
| DOI: | 10.1111/bcp.14989 |
| Publisher version: | https://doi.org/10.22541/au.161911326.60007239/v1 |
| Language: | English |
| Additional information: | This version is the submitted manuscript. For information on re-use, please refer to the publisher’s terms and conditions. Research Funding Innovative Medicines Initiative 2, European Union’s Horizon 2020 research and innovation programme and EFPIA. Grant Number: grant agreement No 777389. Horizon 2020 European Union Innovative Medicines Initiative |
| Keywords: | Innovative Medicines Initiative 2 Joint Undertaking; European Union (EU); Horizon 2020; EFPIA; conect4children; c4c; 777389; clinical pharmacology; drug delivery; drug development; paediatrics; pharmacy |
| UCL classification: | UCL UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Life Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Life Sciences > UCL School of Pharmacy UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Life Sciences > UCL School of Pharmacy > Pharmaceutics |
| URI: | https://discovery.ucl.ac.uk/id/eprint/10131023 |
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