Transforming Pediatric Rare Disease Drug Development: Enhancing Clinical Trials and Regulatory Evidence With Virtual Patients
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 L. P. Sips
- Marco Virgolin (ORCID: https://orcid.org/0000-0001-8905-9313)
- Luca Marchetti (ORCID: https://orcid.org/0000-0001-9043-7705)
- Yann Godfrin
- Federico Reali (ORCID: https://orcid.org/0000-0002-7891-5695)
- Daniel Röshammar
- Jane Knöchel (ORCID: https://orcid.org/0000-0001-9839-2433)
- Giuseppe Pasculli (ORCID: https://orcid.org/0000-0002-0499-2292)
- Alessio Paris (ORCID: https://orcid.org/0000-0002-6667-9330)
- Anita Janus
Institutions
- University of Trento (IT)
- The Microsoft Research - University of Trento Centre for Computational and Systems Biology (IT)
- International School of Trieste (IT)
- ENPICOM (Netherlands) (NL)
- Hôpital Laennec (FR)
Publication Details
- Journal
- CPT Pharmacometrics & Systems Pharmacology
- Published
- 2025-08-04
- DOI
- https://doi.org/10.1002/psp4.70096
- Primary Topic
- Genomics and Rare Diseases
- Type
- article
- Field-Weighted Citation Impact
- 0.00