Two Rochester Neurologists among Those Calling for Stronger Studies
Two neurologists at the University of Rochester Medical Center are part of an international team of scientists who call for greater rigor in the way that early-stage biomedical research is done and reported. The group, led by scientists at the National Institute of Neurological Disorders and Stroke, part of the National Institutes of Health, makes its recommendations in a paper published in Nature.
Among the authors are Robert A. Gross, M.D., Ph.D., and Richard T. Moxley, M.D. Gross took part through his position as editor in chief of Neurology, the world’s leading clinical neurology journal, while Moxley was asked to join the group because of his extensive experience working with patients and doing research on common neurological disorders.
The Nature publication is the result of a workshop organized by NINDS in June to discuss issues around preclinical research – research on which subsequent studies in people are based. Scientists, patients, and pharmaceutical companies use this early information to make crucial decisions about what compounds to pursue to try to develop better treatments for conditions like stroke, Alzheimer’s, and Parkinson’s disease, and which compounds or efforts to wind down because they lack promise.
The availability of new treatments that affect millions of people ultimately depends on these early studies, and the stakes are high not only for researchers but for patients. A new approach that might successfully treat many people down the road could be quashed too early if a poorly designed study errantly concludes that the approach is ineffective. Or, based on limited data, an approach might seem promising, raising the hopes of doctors and patients alike, only to subsequently fail.
“The last thing a physician wants to do is build up inappropriate expectations in patients and their families, and to have them devote time and emotional energy to a treatment that, in a perfect research environment, should not even have been entertained. And that is one outcome of poorly designed preclinical studies,” said Moxley. “Improving these studies is not going to be straightforward or easy, but it’s crucial.”
The effort brought together scientists from government, academia and the pharmaceutical industry, as well as the editors of scientific journals, representatives of patient advocacy groups, and representatives of organizations that fund research.
The team recommended that investigators doing preclinical research follow rules similar to those suggested by a group known as the Consolidated Standards of Reporting Trials, or CONSORT. That independent group of investigators has put forth minimum guidelines for investigators to follow when it comes to doing later-stage research, in people. The guidelines address areas such as statistical significance, transparency regarding the limitations of studies, the reporting of side effects, the importance of publishing “negative” studies about approaches that did not work, and a host of other issues.
“Clinical journals have long used checklists to assure proper reporting of clinical trials,” said Gross. “Adoption of similar measures for more basic studies would help others to pursue work pertinent to the development of clinical trials.”
In its Nature paper, the NINDS-led group also calls for several other changes, including more rigor on the part of scientists reviewing the work of their peers, improved training of investigators, and consideration of alternate ways to recognize and reward work by scientists who attempt to reproduce other groups’ findings – a crucial step in determining whether a new research avenue should be pursued or not.