Articles sponsored by NIH Health Care Systems Research Collaboratory provide new standpoint on clinical research

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.

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 2012 cooperative agreement 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."