Compassionate Use of Gene Therapies in Pediatrics: An Ethical Analysis
CRISPR special issue; Seminars in Perinatology 42, no. 8 (2018): 508-514.
7 Pages Posted: 29 Sep 2020 Last revised: 22 Dec 2021
Date Written: December 1, 2018
In this commentary, we raise concerns about the compassionate use of CRISPR-mediated gene therapies in pediatric and perinatal patients. There is already a precedent for obtaining gene therapies for pediatric patients through compassionate use programs, and the recent passage of a federal Right to Try law raises the stakes as it may contribute to an increase in the number of patients who seek access to investigational products outside of a clinical trial. Clinicians, nurses, drug companies, and parents need support as they grapple with whether compassionate use of CRISPR-mediated gene therapies is the right thing to pursue for a child. We raise three issues to consider in that decision: (1) the effects of compassionate use on scientific research; (2) hype and harms of gene therapies; and (3) the limits and scope of parental authority.
Suggested Citation: Suggested Citation