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Precision medicine in paediatrics: Progress and priorities

Nicola Husain1,2, Claire Booth1,3, Joseph F Standing1,4,5

  • 1Infection, Immunity and Inflammation Research & Teaching Department, UCL GOS Institute of Child Health, London, UK.

British Journal of Clinical Pharmacology
|May 20, 2026
PubMed

Insights

Paediatric precision medicine uses pharmacogenomics and pharmacometrics to tailor drug treatments for children, improving safety and efficacy. Advanced therapies offer new hope, but challenges in cost and implementation remain.

Area of Science:

  • Paediatric medicine
  • Pharmacogenomics
  • Pharmacometrics
  • Advanced therapy medicinal products

Background:

  • Precision medicine personalizes healthcare based on individual patient profiles.
  • Paediatric precision medicine leverages advances in genetics and quantitative pharmacology.
  • Traditional 'trial-and-error' prescribing in children leads to suboptimal outcomes and toxicity.

Purpose of the Study:

  • To review the evolving landscape of paediatric precision medicine.
  • To examine advances in pharmacogenomics, pharmacometrics, and advanced therapies.
  • To demonstrate how these innovations can optimize paediatric clinical care and therapeutic strategies.

Main Methods:

  • Review of pharmacogenomic principles and their application in paediatrics.
  • Analysis of pharmacometric techniques, including population pharmacokinetic-pharmacodynamic modeling.
  • Examination of advanced therapy medicinal products (e.g., gene therapies) in paediatric disease treatment.

Main Results:

  • Pharmacogenomics can reduce drug toxicity and accelerate the identification of effective paediatric treatments.
  • Pharmacometrics provides data-driven dosing guidelines for special paediatric populations.
  • Advanced therapies are advancing paediatric disease treatment, necessitating new funding models.

Conclusions:

  • Paediatric precision medicine holds significant potential for improving child healthcare outcomes.
  • Implementation challenges include cost, workforce readiness, governance, and equity.
  • Continued research is crucial for integrating novel precision medicine technologies into paediatric practice.

Keywords:
advanced therapy medicinal productsgene therapymodel‐informed precision dosingpersonalized medicinepharmacogenomicspharmacometrics

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