Siblings and Discordant Eligibility for Gene Therapy Research: Considering Parental Requests for Non-Trial "Compassionate Use". (December 2021)

• Record Type: Journal Article
• Authors: Webb, Jamie; Shah, Lesha D; Bateman-House, Alison
• Is Part Of: Clinical ethics. Volume 16:Number 4(2021)
• Page Start: 359
• Page End: 363
• Publication Date: 2021-12
• Subjects: Bioethics and medical ethics -- pediatrics -- biomedical research -- justice -- gene therapy -- expanded access; Medical ethics -- Periodicals; 174.205
• DOI: 10.1177/1477750920983571
• Languages: English
• ISSNs: 1477-7509

Abstract

Deciding whether to grant an expanded access request for a child whose sibling is enrolled in a gene therapy trial involves a number of complex factors: considering the best interests of the child, the psychosocial and economic impact on the family, and the concerns and obligations of researchers. Despite the challenges in coming to a substantively fair outcome in cases of discordant eligibility, creating a procedurally fair decision-making process to adjudicate requests is essential.