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



Neumann, Eva, Schreeck, Filippa, Herberg, Jethro, Aigrain, Evelyne Jacqz, Maitland-van der Zee, Anke H, Perez-Martinez, Antonio, Hawcutt, Daniel B ORCID: 0000-0002-8120-6507, Schaeffeler, Elke, Rane, Anders, de Wildt, Saskia N
et al (show 1 more authors) (2022) How paediatric drug development and use could benefit from OMICs: A c4c expert group white paper. BRITISH JOURNAL OF CLINICAL PHARMACOLOGY, 88 (12). pp. 5017-5033.

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Abstract

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.

Item Type: Article
Uncontrolled Keywords: clinical trials, epigenomics, OMICS technology, paediatrics, pharmacogenomics
Divisions: Faculty of Health and Life Sciences
Faculty of Health and Life Sciences > Institute of Life Courses and Medical Sciences
Depositing User: Symplectic Admin
Date Deposited: 25 Mar 2022 08:26
Last Modified: 18 Jan 2023 21:06
DOI: 10.1111/bcp.15216
Open Access URL: https://bpspubs.onlinelibrary.wiley.com/doi/10.111...
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URI: https://livrepository.liverpool.ac.uk/id/eprint/3151418