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Staunch protections: the ethics of haemophilia gene transfer research
Author(s) -
KIMMELMAN J.
Publication year - 2008
Publication title -
haemophilia
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.213
H-Index - 92
eISSN - 1365-2516
pISSN - 1351-8216
DOI - 10.1111/j.1365-2516.2007.01567.x
Subject(s) - haemophilia , medicine , clinical trial , disadvantaged , gene transfer , law , pediatrics , political science , pathology , biochemistry , chemistry , gene
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.