Synthetic Data in Healthcare and Drug Development: Definitions, Regulatory Frameworks, Issues
ABSTRACT With the recent and evolving regulatory frameworks regarding the usage of Artificial Intelligence (AI) in both drug and medical device development, the differentiation between data derived from observed (‘true’ or ‘real’) sources and artificial data obtained using process‐driven and/or (dat...
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| Format: | Article |
| Language: | English |
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Wiley
2025-05-01
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| Series: | CPT: Pharmacometrics & Systems Pharmacology |
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| Online Access: | https://doi.org/10.1002/psp4.70021 |
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| author | Giuseppe Pasculli Marco Virgolin Puja Myles Anna Vidovszky Charles Fisher Elisabetta Biasin Miranda Mourby Francesco Pappalardo Saverio D'Amico Mario Torchia Alexander Chebykin Vincenzo Carbone Luca Emili Daniel Roeshammar |
| author_facet | Giuseppe Pasculli Marco Virgolin Puja Myles Anna Vidovszky Charles Fisher Elisabetta Biasin Miranda Mourby Francesco Pappalardo Saverio D'Amico Mario Torchia Alexander Chebykin Vincenzo Carbone Luca Emili Daniel Roeshammar |
| author_sort | Giuseppe Pasculli |
| collection | DOAJ |
| description | ABSTRACT With the recent and evolving regulatory frameworks regarding the usage of Artificial Intelligence (AI) in both drug and medical device development, the differentiation between data derived from observed (‘true’ or ‘real’) sources and artificial data obtained using process‐driven and/or (data‐driven) algorithmic processes is emerging as a critical consideration in clinical research and regulatory discourse. We conducted a critical literature review that revealed evidence of the current ambivalent usage of the term “synthetic” (along with derivative terms) to refer to “true/observed” data in the context of clinical trials and AI‐generated data (or “artificial” data). This paper, stemming from a critical evaluation of different perspectives captured from the scientific literature and recent regulatory endeavors, seeks to elucidate this distinction, exploring their respective utilities, regulatory stances, and upcoming needs, as well as the potential for both data types in advancing medical science and therapeutic development. |
| format | Article |
| id | doaj-art-2ea3772d92bc4a2ca05fa96a4f82e9eb |
| institution | OA Journals |
| issn | 2163-8306 |
| language | English |
| publishDate | 2025-05-01 |
| publisher | Wiley |
| record_format | Article |
| series | CPT: Pharmacometrics & Systems Pharmacology |
| spelling | doaj-art-2ea3772d92bc4a2ca05fa96a4f82e9eb2025-08-20T02:31:08ZengWileyCPT: Pharmacometrics & Systems Pharmacology2163-83062025-05-0114584085210.1002/psp4.70021Synthetic Data in Healthcare and Drug Development: Definitions, Regulatory Frameworks, IssuesGiuseppe Pasculli0Marco Virgolin1Puja Myles2Anna Vidovszky3Charles Fisher4Elisabetta Biasin5Miranda Mourby6Francesco Pappalardo7Saverio D'Amico8Mario Torchia9Alexander Chebykin10Vincenzo Carbone11Luca Emili12Daniel Roeshammar13InSilicoTrials Technologies S.p.A. Trieste ItalyInSilicoTrials Technologies B.V. s‐Hertogenbosch the NetherlandsMedicines and Healthcare products Regulatory Agency London UKUnlearn.AI San Francisco California USAUnlearn.AI San Francisco California USACentre for IT & IP Law (CiTiP), KU Leuven Leuven BelgiumCentre for Health, Law, and Emerging Technologies (HeLEX), Faculty of Law University of Oxford Oxford UKDepartment of Drug and Health Sciences University of Catania Catania ItalyHumanitas Clinical and Research Center‐IRCCS Milan ItalyInSilicoTrials Technologies S.p.A. Trieste ItalyInSilicoTrials Technologies B.V. s‐Hertogenbosch the NetherlandsInSilicoTrials Technologies S.p.A. Trieste ItalyInSilicoTrials Technologies S.p.A. Trieste ItalyInSilicoTrials Technologies S.p.A. Trieste ItalyABSTRACT With the recent and evolving regulatory frameworks regarding the usage of Artificial Intelligence (AI) in both drug and medical device development, the differentiation between data derived from observed (‘true’ or ‘real’) sources and artificial data obtained using process‐driven and/or (data‐driven) algorithmic processes is emerging as a critical consideration in clinical research and regulatory discourse. We conducted a critical literature review that revealed evidence of the current ambivalent usage of the term “synthetic” (along with derivative terms) to refer to “true/observed” data in the context of clinical trials and AI‐generated data (or “artificial” data). This paper, stemming from a critical evaluation of different perspectives captured from the scientific literature and recent regulatory endeavors, seeks to elucidate this distinction, exploring their respective utilities, regulatory stances, and upcoming needs, as well as the potential for both data types in advancing medical science and therapeutic development.https://doi.org/10.1002/psp4.70021drug developmentexternal control armsgenerative AImedical devicesreal‐world dataregulatory framework |
| spellingShingle | Giuseppe Pasculli Marco Virgolin Puja Myles Anna Vidovszky Charles Fisher Elisabetta Biasin Miranda Mourby Francesco Pappalardo Saverio D'Amico Mario Torchia Alexander Chebykin Vincenzo Carbone Luca Emili Daniel Roeshammar Synthetic Data in Healthcare and Drug Development: Definitions, Regulatory Frameworks, Issues CPT: Pharmacometrics & Systems Pharmacology drug development external control arms generative AI medical devices real‐world data regulatory framework |
| title | Synthetic Data in Healthcare and Drug Development: Definitions, Regulatory Frameworks, Issues |
| title_full | Synthetic Data in Healthcare and Drug Development: Definitions, Regulatory Frameworks, Issues |
| title_fullStr | Synthetic Data in Healthcare and Drug Development: Definitions, Regulatory Frameworks, Issues |
| title_full_unstemmed | Synthetic Data in Healthcare and Drug Development: Definitions, Regulatory Frameworks, Issues |
| title_short | Synthetic Data in Healthcare and Drug Development: Definitions, Regulatory Frameworks, Issues |
| title_sort | synthetic data in healthcare and drug development definitions regulatory frameworks issues |
| topic | drug development external control arms generative AI medical devices real‐world data regulatory framework |
| url | https://doi.org/10.1002/psp4.70021 |
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