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

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.

Artificial Intelligence in Pediatrics: Learning to Walk Together

“…This comprehensive approach not only enhances diagnostic accuracy but also facilitates the development of targeted interventions that consider an individual’s unique genetic and molecular profile, ultimately leading to more effective and tailored healthcare for pediatric patients. 46 However, when examining a singular kind of omics data, the scope is largely restricted to the detection of variances, or at most, correlations between 1 or 2 forms of biological entities. This limitation confines the results to reactive processes rather than causal occurrences.…”

Biomarker und Neuromonitoring zur Entwicklungsprognose nach perinataler Hirnschädigung

“…Omics-Technologien werden nicht nur in Studien zu Prognose und Verlauf von Erkrankungen eingesetzt, sondern sollen auch der Entwicklung von Therapiestrategien, die auf den einzelnen Patienten personalisiert sind, dienen [ 67 ]. Die Herausforderung besteht in der Verarbeitung großer Datenmengen, die mit klinischen Informationen zusammengebracht werden müssen.…”

Innovative methodologies in paediatric drug development: A conect4children (c4c) special issue

“…6 Neumann et al discuss the need for and benefits of Omics approaches in paediatric drug development. 7 A better understanding of paediatric disease, the impact of ontogeny on drug disposition and effect, as well as the interplay of drugs with the disease warrants the use of Omics techniques. Omics approaches discussed are epigenomics, genomics, transcriptomics, proteomics, microbiomics and metabolomics.…”