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

See all articles by Carolyn Neuhaus

Carolyn Neuhaus

The Hastings Center

Rachel Zacharias

University of Pennsylvania Law School

Date Written: December 1, 2018

Abstract

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

Neuhaus, Carolyn and Zacharias, Rachel, Compassionate Use of Gene Therapies in Pediatrics: An Ethical Analysis (December 1, 2018). CRISPR special issue; Seminars in Perinatology 42, no. 8 (2018): 508-514. , Available at SSRN: https://ssrn.com/abstract=3672685 or http://dx.doi.org/10.2139/ssrn.3672685

Carolyn Neuhaus

The Hastings Center ( email )

Garrison, NY 10524
United States

Rachel Zacharias (Contact Author)

University of Pennsylvania Law School ( email )

3501 Sansom St
Philadelphia, PA 19104
United States

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