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September 15, 2015 – A special series of articles addressing the ethical and regulatory challenges to pragmatic clinical trials appears this week on the website of the journal Clinical Trials.
The 12 articles were sponsored by the NIH Health Care Systems Research Collaboratory with additional support from the Patient-Centered Outcomes Research Institute, and include authors drawn from the fields of clinical research and patient advocacy, as well as clinicians, bioethicists, and regulatory experts.
The articles can be found on the Clinical Trials website.
An overview of the series was written by the leaders of the project, bioethicist Jeremy Sugarman, professor of bioethics and medicine and deputy director of the Johns Hopkins Berman Institute of Bioethics, and cardiologist Robert Califf, former vice chancellor for clinical and translational research at Duke University and now the Deputy Commissioner for Medical Products and Tobacco at the Food and Drug Administration.
The remaining 11 articles focus on topics including defining and evaluating research risks, protection of vulnerable research participants, informed consent, privacy of research participants, and the appropriate interpretation and application of current federal regulations governing research.
Established by a 2013 grant from the National Institutes of Health Common Fund, the Collaboratory is dedicated to supporting investigations into new and better ways of conducting clinical research. In particular, the Collaboratory is focused on pragmatic clinical research, which aims to answer important research questions quickly and efficiently by applying novel research designs and tools.
However, these approaches are currently applied within an existing structure of rules, regulations, and ethical guidelines that was created to govern more traditional types of healthcare research. The result has been an increasing tension between the imperative to protect the rights and welfare of research participants while enabling the research needed to provide information that future patients and their healthcare providers will rely upon to make informed choices.
“We’re facing a serious gap in terms of the scientific evidence we need to better guide decisions about medical treatments,” said Califf, who together with Sugarman served as an editor for the issue. “The problem is ensuring that we can converge upon an ethical and regulatory framework that’s appropriate for the kinds of research that will be essential in the coming era of patient-centered precision medicine.”
“These articles address a number of areas that for years have been marked by uncertainty, even for experts,” Sugarman said. “What we have tried to do here is clarify some of the most pressing issues and point toward some possible solutions.”
September 10, 2015
For Immediate Release
Contact: Leah Ramsay, firstname.lastname@example.org, 202.642.9640
Human Genome Editing Research Should Move Forward, Says Influential Hinxton Group
Research involving editing the human genome, including research with human embryos, is essential to gain basic understanding of biology and germ cells and should be permitted, according to one of the first global meetings to debate the controversial new techniques.
The bold statement is published Thursday, September 10, by the Hinxton Group, a global network of stem cell researchers, bioethicists, and experts on policy and scientific publishing, who met in Manchester, England, September 3-4.
While firmly backing the need for gene editing research, the group makes a clear distinction between research and clinical application.
“We believe that while this technology has tremendous value to basic research and enormous potential for somatic clinical uses, it is not sufficiently developed to consider human genome editing for clinical reproductive purposes at this time,” the consensus statement reads.
According to Debra Mathews, a member of the Hinxton Group steering committee, discussions at the meeting focused on the use of gene editing in research and the most contentious aspects of these new technologies, primarily the implications for any children born with engineered genetic modifications, and also successive generations who would inherit those genetic changes; that is, the inheritable, or germline, nature of the modifications.
“While there is controversy and deep moral disagreement about human germline genetic modification, what is needed is not to stop all discussion, debate and research, but rather to engage with the public, policymakers and the broader scientific community, and to weigh together the potential benefits and harms of human genome editing for research and human health,” says Mathews, the Assistant Director for Science Programs at the Johns Hopkins Berman Institute of Bioethics.
The consensus statement addresses these ethical concerns, with the group agreeing that, “given all safety, efficacy and governance needs are met, there may be morally acceptable uses of this technology in human reproduction, though further substantial discussion and debate will be required”.
In the meantime, knowledge gained through basic science research is essential to human understanding of both ourselves and other life, the group says. “Much of our knowledge of early development comes from studies of mouse embryos, yet it is becoming clear that gene activity and even some cell types are very different in human embryos. Genome editing techniques could be used to ask how cell types are specified in the early embryo and the nature and importance of the genes involved,” says Robin Lovell-Badge, a member of the Hinxton Group steering committee and Group Leader, and head of the Laboratory of Stem Cell Biology and Developmental Genetics, The Francis Crick Institute.
The statement emphasizes the importance of “meaningful and substantial public engagement” to decision-making about genome editing. Policymakers are specifically addressed, stating that policy restraints on science should have justification that “that reaches beyond disagreements based solely on divergent moral convictions.”
“The relevant regulatory distinction should be not between using genome editing in somatic cells and using it in embryos, but between research and reproduction: whether those embryos are ever destined to be implanted, says Sarah Chan, another steering committee member and a Chancellor’s Fellow at the Usher Institute for Population Health Sciences and Informatics, University of Edinburgh.
“Restricting research because of concerns that reproductive application is premature and dangerous will ensure that it remains forever premature and dangerous, for want of better knowledge,” Chan says.
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Hinxton Group Consensus Statement on Genome Editing Technologies and Human Germline Genetic Modification, published online:http://www.hinxtongroup.org/Hinxton2015_Statement.pdf
About the Hinxton Group: http://hinxtongroup.org/
For more information or commentary from Debra Mathews, contact:
Leah Ramsay, Johns Hopkins Berman Institute of Bioethics
For commentary from experts who are NOT members of the Hinxton Group, contact: Robin Bisson, Genetic Expert News Service
email@example.com +1 (202) 833-4613
The Science Media Centre (UK)
firstname.lastname@example.org +44 (0)20 7611 8300
Professor Robin Lovell-Badge, Hinxton Group Steering Committee member
Office: +44 (0)20 8816 2126; Mobile: +44 (0)7785 366987
Or: The Francis Crick Institute media team:
Katie Matthews: +44 (0)20 7611 2197 or +44 (0)7734 955308
Peter Zarko-Flynn: +44 (0)7525 053795