Ethical and Epistemic Considerations for the Use of Artificial Intelligence in Ultra-Rare Disease Clinical Trials.

This article examines the ethical and epistemic implications of using artificial intelligence (AI) in research on ultra-rare diseases, where patient populations are extremely small and conventional clinical trials are often infeasible. It highlights AI’s role as an infrastructure of visibility that...

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Publicado en:American Journal of Bioethics Vol. 26; no. 8; pp. 37 - 40
Autores principales: Owens, Kellie, Kearns, Lisa, Bateman-House, Alison
Formato: commentary Journal Article
Publicado: Taylor & Francis Ltd Aug2026
Acceso en línea:Ver este registro en EBSCOhost
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        atl: Ethical and Epistemic Considerations for the Use of Artificial Intelligence in Ultra-Rare Disease Clinical Trials.
      aug:
        au:
          Owens, Kellie
          Kearns, Lisa
          Bateman-House, Alison
        affil: NYU Grossman School of Medicine
      sug:
        subj:
          Artificial Intelligence Ethical Issues
          Clinical Trials Ethical Issues
          Epistemology
          Rare Diseases
          Implementation Science
          Patient Selection
          United States
      ab: This article examines the ethical and epistemic implications of using artificial intelligence (AI) in research on ultra-rare diseases, where patient populations are extremely small and conventional clinical trials are often infeasible. It highlights AI’s role as an infrastructure of visibility that can identify eligible patients across fragmented data sources, potentially improving diagnosis and access to experimental therapies, but also risks reinforcing existing inequities due to uneven data representation. The article discusses AI-generated synthetic evidence, such as digital twins and synthetic control arms, as emerging tools for knowledge generation that raise questions about reliability and standards of evidence in ultra-rare disease research. It emphasizes the importance of patient and community involvement in governing AI use, advocating for transparency and stewardship to ensure equitable distribution of research opportunities and trust in AI-enabled systems.
      pubtype: Academic Journal
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        commentary
        Journal Article
      ougenre: Article
    language: English
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