Ethical challenges for a new generation of early-phase pediatric gene therapy trials
pediatrics; ethics; human; review; clinical trial (topic); patient selection; patient engagement; risk benefit analysis; gene therapy; giant axonal neuropathy
After decades of setbacks, gene therapy (GT) is experiencing major breakthroughs. Five GTs have received US regulatory approval since 2017, and over 900 others are currently in development. Many of these GTs target rare pediatric diseases that are severely life-limiting, given a lack of effective treatments. As these GTs enter early-phase clinical trials, specific ethical challenges remain unresolved in three domains: evaluating risks and potential benefits, selecting participants fairly, and engaging with patient communities. Drawing on our experience as clinical investigators, basic scientists, and bioethicists involved in a first-in-human GT trial for an ultrarare pediatric disease, we analyze these ethical challenges and offer points to consider for future GT trials. Copyright © 2021, This is a U.S. government work and not under copyright protection in the U.S.; foreign copyright protection may apply.
Iyer AA; Saade D; Bharucha-Goebel D; Foley AR; Averion GM; Paredes E; Gray S; Bonnemann CG; Grady C; Hendriks S; Rid A
Genetics in Medicine
2021
Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).
<a href="http://doi.org/10.1038/s41436-021-01245-3" target="_blank" rel="noreferrer noopener">10.1038/s41436-021-01245-3</a>
Views of adolescents and parents on pediatric research without the potential for clinical benefit
Female; Humans; Male; Adolescent Psychology; Adult; Attitude to Health; Questionnaires; Motivation; Altruism; adolescent; Risk-Taking; Parents/psychology; Nontherapeutic Human Experimentation; Research Subjects/psychology; Benefits of PPC
Wendler D; Abdoler E; Wiener L; Grady C
Pediatrics
2012
Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).
Journal Article
<a href="http://doi.org/10.1542/peds.2012-0068" target="_blank" rel="noreferrer">10.1542/peds.2012-0068</a>