Digital twins, synthetic patient data, and in-silico trials: can they empower paediatric clinical trials?
Summary: Randomised controlled trials are the gold standard to assess the effectiveness and safety of clinical interventions; however, many paediatric trials are discontinued early due to challenges in patient enrolment. Hence, most paediatric clinical trials suffer from lack of adequate power. Addi...
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| Format: | Article |
| Language: | English |
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Elsevier
2025-05-01
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| Series: | The Lancet: Digital Health |
| Online Access: | http://www.sciencedirect.com/science/article/pii/S258975002500007X |
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| author | ProfMD Mohan Pammi Prakesh S Shah, MD Liu K Yang, PhD Joseph Hagan, ScD Nima Aghaeepour, PhD ProfMD Josef Neu, PhD |
| author_facet | ProfMD Mohan Pammi Prakesh S Shah, MD Liu K Yang, PhD Joseph Hagan, ScD Nima Aghaeepour, PhD ProfMD Josef Neu, PhD |
| author_sort | ProfMD Mohan Pammi |
| collection | DOAJ |
| description | Summary: Randomised controlled trials are the gold standard to assess the effectiveness and safety of clinical interventions; however, many paediatric trials are discontinued early due to challenges in patient enrolment. Hence, most paediatric clinical trials suffer from lack of adequate power. Additionally, trials are expensive and might expose patients to unproven therapies. Alternatives to overcome these issues using virtual patient data—namely, digital twins, synthetic patient data, and in-silico trials—are now possible due to rapid advances in digital health-care tools and interventions. However, such digital innovations have been rarely used in paediatric trials. In this Viewpoint, we propose using virtual patient data to empower paediatric trials. The use of virtual patient data has the advantages of decreased exposure of children to potentially ineffective or risky interventions, shorter trial durations leading to more rapid ascertainment of safety and effectiveness of interventions, and faster drug approvals. Use of virtual patient data could lead to more personalised treatment options with low costs and could result in faster clinical implementation of interventions in children. However, ethical and regulatory concerns, including replacing humans with digital data, data privacy, and security should be addressed and the safety and sustainability of digital data innovation ensured before virtual patient data are adopted widely. |
| format | Article |
| id | doaj-art-d7a26bdc1572460dbd3903d3cc33925e |
| institution | OA Journals |
| issn | 2589-7500 |
| language | English |
| publishDate | 2025-05-01 |
| publisher | Elsevier |
| record_format | Article |
| series | The Lancet: Digital Health |
| spelling | doaj-art-d7a26bdc1572460dbd3903d3cc33925e2025-08-20T02:37:42ZengElsevierThe Lancet: Digital Health2589-75002025-05-017510085110.1016/j.landig.2025.01.007Digital twins, synthetic patient data, and in-silico trials: can they empower paediatric clinical trials?ProfMD Mohan Pammi0Prakesh S Shah, MD1Liu K Yang, PhD2Joseph Hagan, ScD3Nima Aghaeepour, PhD4ProfMD Josef Neu, PhD5Department of Pediatrics, Baylor College of Medicine and Texas Children’s Hospital, Houston, TX, USA; Correspondence to: Prof Mohan Pammi, Department of Pediatrics, Baylor College of Medicine and Texas Children's Hospital, Houston, TX 77030, USADepartment of Paediatrics, Mount Sinai Hospital, University of Toronto, Toronto, ON, CanadaStanford University School of Medicine, San Francisco, CA, USADepartment of Pediatrics, Baylor College of Medicine and Texas Children’s Hospital, Houston, TX, USAStanford University School of Medicine, San Francisco, CA, USAUniversity of Florida, Gainesville, FL, USASummary: Randomised controlled trials are the gold standard to assess the effectiveness and safety of clinical interventions; however, many paediatric trials are discontinued early due to challenges in patient enrolment. Hence, most paediatric clinical trials suffer from lack of adequate power. Additionally, trials are expensive and might expose patients to unproven therapies. Alternatives to overcome these issues using virtual patient data—namely, digital twins, synthetic patient data, and in-silico trials—are now possible due to rapid advances in digital health-care tools and interventions. However, such digital innovations have been rarely used in paediatric trials. In this Viewpoint, we propose using virtual patient data to empower paediatric trials. The use of virtual patient data has the advantages of decreased exposure of children to potentially ineffective or risky interventions, shorter trial durations leading to more rapid ascertainment of safety and effectiveness of interventions, and faster drug approvals. Use of virtual patient data could lead to more personalised treatment options with low costs and could result in faster clinical implementation of interventions in children. However, ethical and regulatory concerns, including replacing humans with digital data, data privacy, and security should be addressed and the safety and sustainability of digital data innovation ensured before virtual patient data are adopted widely.http://www.sciencedirect.com/science/article/pii/S258975002500007X |
| spellingShingle | ProfMD Mohan Pammi Prakesh S Shah, MD Liu K Yang, PhD Joseph Hagan, ScD Nima Aghaeepour, PhD ProfMD Josef Neu, PhD Digital twins, synthetic patient data, and in-silico trials: can they empower paediatric clinical trials? The Lancet: Digital Health |
| title | Digital twins, synthetic patient data, and in-silico trials: can they empower paediatric clinical trials? |
| title_full | Digital twins, synthetic patient data, and in-silico trials: can they empower paediatric clinical trials? |
| title_fullStr | Digital twins, synthetic patient data, and in-silico trials: can they empower paediatric clinical trials? |
| title_full_unstemmed | Digital twins, synthetic patient data, and in-silico trials: can they empower paediatric clinical trials? |
| title_short | Digital twins, synthetic patient data, and in-silico trials: can they empower paediatric clinical trials? |
| title_sort | digital twins synthetic patient data and in silico trials can they empower paediatric clinical trials |
| url | http://www.sciencedirect.com/science/article/pii/S258975002500007X |
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