Transforming Pediatric Rare Disease Drug Development: Enhancing Clinical Trials and Regulatory Evidence With Virtual Patients.

Journal: CPT: pharmacometrics & systems pharmacology
Published Date: Aug 4, 2025

Abstract

Drug development in pediatric rare diseases is complicated by practical and ethical constraints on clinical trial design, stemming from small, highly heterogeneous, and vulnerable patient populations. Virtual patients (VPs) created with machine-learning (ML), mechanistically driven computational approaches, or hybrids thereof, have the potential to expedite and maximize the impact of trials. We discuss the potential of VPs to transform the efficiency and impact of clinical trials in pediatric rare diseases, based on adult and pediatric examples.

Authors

  • Fianne Sips
    InSilicoTrials Technologies BV, 's-Hertogenbosch, the Netherlands.
  • Marco Virgolin
    InSilicoTrials Technologies B.V., s-Hertogenbosch, the Netherlands.
  • Giuseppe Pasculli
    Nephrology, Dialysis, and Transplantation Unit, Department of Emergency and Organ Transplantation, University of Bari Aldo Moro, Bari, Italy.
  • Federico Reali
    Fondazione The Microsoft Research-University of Trento Centre for Computational and Systems Biology (COSBI), Rovereto, Trento, Italy.
  • Alessio Paris
    Fondazione the Microsoft Research - University of Trento Centre for Computational and Systems Biology (COSBI), Rovereto, Italy.
  • Annette Janus
    Axoltis Pharma, Bioparc Laennec, Lyon, France.
  • Yann Godfrin
    Axoltis Pharma, Bioparc Laennec, Lyon, France.
  • Daniel Röshammar
    InSilicoTrials Technologies Spa, Trieste, Italy.
  • Luca Marchetti
    Department of Clinical Oncology, Policlinico Umberto I, School of Medicine and Psychology, University of Rome La Sapienza, Rome, Italy.
  • Jane Knöchel
    InSilicoTrials Technologies Spa, Trieste, Italy.

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