Over the last decade, 2 themes have emerged as major drivers for the future of health care: comparative effectiveness research and precision medicine. Both have relied heavily on data derived from clinical trials, particularly the randomized controlled clinical trial (RCT). Technological developments such as electronic health records (EHRs) will vastly increase the accumulation and availability of data related to outcomes of care, potentially transforming clinical research and reducing the reliance on the RCT. In fact, EHRs and prospective registries are now thought by some to provide information that could complement or replace information derived from RCTs. Whether RCT or registry is used, the analyses must be statistically rigorous and scientifically sound to best guide the evolution of practice so that 21st century neurosurgery will be based on reliable, scientific data, and analyses. The information provided by either RCT or registry may also lead to more effective use of medical resources and reduce expense of care.
The Launching Effectiveness Research to Guide Practice in Neurosurgery Workshop was held on February 5, 2015 in Bethesda, Maryland, to discuss clinical research in neurosurgery. The attendees included experts in neurosurgery, neurology, statistics, effectiveness research, and bioethics, along with representatives from federal agencies including National Institutes of Health (NIH), Food and Drug Administration (FDA), Center for Medicare and Medicaid Services (CMS), and Agency for Healthcare Research and Quality (AHRQ). The Research Committee for the Society for Neurological Surgery and National Institute Neurological Disorders and Stroke (NINDS) leadership worked together to ensure broad participation for a variety of stakeholders. For example, for the neurosurgery/neurology participation, selection of participants was based on current leadership in national neurosurgical societies, principal investigatorship on NIH, or other type of grants related to neurosurgical or neurological research in clinical trials, and participation in clinical trials related to neurosurgery. Similarly, representatives for federal agencies were selected based on their expertise and/or leadership in the matter under study. This report is a summary of the meeting agreed upon by the authors.
From Launching Effectiveness Research to Guide Practice in Neurosurgery: A National Institute Neurological Disorders and Stroke Workshop Report by Patricia Walicke, MD, PhD, Aviva Abosch, MD, Anthony Asher, MD, Fred G. Barker, II, MD, Zoher Ghogawala, MD, Robert Harbaugh, MD, Lara Jehi, MD, John Kestle, MD, Walter Koroshetz, MD, Roderick Little, PhD, Donald Rubin, PhD, Alex Valadka, MD, Stephen Wisniewski, PhD, E. Antonio Chiocca, MD, PhD