How paediatric drug development and use could benefit from OMICs: A c4c expert group white paper

Eva Neumann, Filippa Schreeck, Jethro Herberg, Evelyne Jacqz Aigrain, Anke H. Maitland-van der Zee, Antonio Pérez-Martínez, Daniel B. Hawcutt, Elke Schaeffeler, Anders Rane, Saskia N. de Wildt, Matthias Schwab*

*Corresponding author for this work

Research output: Contribution to journalReview articleAcademicpeer-review

4 Citations (Scopus)
28 Downloads (Pure)


The safety and efficacy of pharmacotherapy in children, particularly preterms, neonates and infants, is limited by a paucity of good-quality data from prospective clinical drug trials. A specific challenge is the establishment of valid biomarkers. OMICs technologies may support these efforts by complementary information about targeted and nontargeted molecules through systematic characterization and quantitation of biological samples. OMICs technologies comprise at least genomics, epigenomics, transcriptomics, proteomics, metabolomics and microbiomics in addition to the patient's phenotype. OMICs technologies are in part hypothesis-generating, allowing an in depth understanding of disease pathophysiology and pharmacological mechanisms. Application of OMICs technologies in paediatrics faces major challenges before routine adoption. First, developmental processes need to be considered, including a subdivision into specific age groups as developmental changes clearly impact OMICs data. Second, compared to the adult population, the number of patients is limited as are the type and amount of necessary biomaterial, especially in neonates and preterms. Thus, advanced trial designs and biostatistical methods, noninvasive biomarkers, innovative biobanking concepts including data and samples from healthy children, as well as analytical approaches (eg liquid biopsies) should be addressed to overcome these obstacles. The ultimate goal is to link OMICs technologies with innovative analysis tools, such as artificial intelligence at an early stage. The use of OMICs data based on a feasible approach will contribute to the identification complex phenotypes and subpopulations of patients to improve the development of medicines for children with potential economic advantages.

Original languageEnglish
Pages (from-to)5017-5033
Number of pages17
JournalBritish Journal of Clinical Pharmacology
Issue number12
Publication statusPublished - Dec 2022

Bibliographical note

Funding Information:
The conect4children (c4c) project has received funding from the Innovative Medicines Initiative (IMI) 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. E.N., E.S., F.S. and M.S. are supported by Robert Bosch Stiftung, Stuttgart, Germany.

Funding information
Horizon 2020 Framework Programme, Grant/
Award Numbers: 777389, Innovative
Medicines Initiative 2 Joint Undertakin; Robert
Bosch Stiftung

Publisher Copyright:
© 2022 The Authors. British Journal of Clinical Pharmacology published by John Wiley & Sons Ltd on behalf of British Pharmacological Society.


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