REVIEW ARTICLE
Staunch protections: the ethics of haemophilia gene transfer research
J. KIMMELMAN,
J. KIMMELMAN
Department of Social Studies of Medicine Clinical Trials Research Group/Biomedical Ethics Unit Faculty of Medicine/McGill University, Montreal, QC, Canada
Search for more papers by this authorJ. KIMMELMAN,
J. KIMMELMAN
Department of Social Studies of Medicine Clinical Trials Research Group/Biomedical Ethics Unit Faculty of Medicine/McGill University, Montreal, QC, Canada
Search for more papers by this authorJonathan Kimmelman, PhD, Department of Social Studies of Medicine Clinical Trials Research Group/Biomedical Ethics Unit Faculty of Medicine/McGill University, 3647 Peel Street, Montreal, QC, Canada H3A 1X1.Tel.: (514) 482 8362; fax: (514) 398 8349; e-mail: [email protected]
The author serves in a non-remunerative capacity on the ethics advisory board of the American Society of Gene Therapy.
Abstract
Summary. Haemophilia has long been considered an ideal system for validating human gene transfer (GT). However, haemophilia GT trials present a particular ethical challenge because they involve subjects whose medical condition is stabilized by standard therapies. Below, I review the ethics and risks of haemophilia GT clinical research. I propose several conditions and practices that strengthen the ethical basis for such trials. These include consultation with haemophilia advocacy organizations as trials are designed and executed, high standards of supporting evidence before trials are initiated, pretrial publication of this evidence, and the offer of indemnification for participants. I further argue against the conduct of paediatric haemophilia GT studies at this time, and raise questions about the fairness of recruiting economically disadvantaged subjects into studies that are primarily directed towards the health needs of persons in the developed world.
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