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Somatic Gene Therapy Research in Pediatric Populations: Ethical Issues and Guidance for Operationalizing Early Phase

Alison Bateman-House1, Lesha D Shah2, Rafael Escandon3

  • 1Division of Medical Ethics, Department of Population Health, NYU Grossman School of Medicine, New York, NY, USA. Alison.Bateman-House@nyulangone.org.

Pharmaceutical Medicine
|December 17, 2022
PubMed

Insights

Investigational gene therapies (GT) for children present unique ethical challenges due to diverse diseases and limited treatments. Proactive identification and discussion of these issues are crucial for ethical pediatric research.

Area of Science:

  • Pediatric Research Ethics
  • Gene Therapy Clinical Trials
  • Bioethics

Background:

  • Pediatric gene therapy research spans diverse indications, from rare to ultra-rare diseases, with varying treatment availability.
  • The heterogeneity of these conditions necessitates a nuanced approach to ethical considerations, as a universal framework is insufficient.

Purpose of the Study:

  • To review and identify key ethical issues in pediatric investigational gene therapy (GT) research.
  • To highlight the need for proactive ethical analysis and transparent stakeholder discussion in GT clinical trials involving children.

Main Methods:

  • Review of current ethical issues in pediatric gene therapy research.
  • Identification of specific concerns including immunogenicity, toxicity, consent, trial design, and equity.

Main Results:

  • Key ethical concerns include product-related immunogenicity, toxicity, duration, irreversibility, informed consent/assent, trial design, participant/caregiver burdens, and equity in access.
  • Anticipated issues like research uncertainty, risk, preclusion from other studies, long-term monitoring, and caregiver urgency require proactive management.

Conclusions:

  • Ethical challenges in pediatric gene therapy trials are multifaceted and context-dependent.
  • Proactive identification, adherence to best practices, and transparent communication are essential for addressing ethical issues in pediatric GT research.

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