Comparative effectiveness and implementation research: Directions for Neurology

Abstract

There is an enormous unmet need for knowledge about how new insights from discovery and translational research can yield measurable, population-level improvements in health and reduction in mortality among those having or at risk for neurological disease. Once several, well-conducted randomized controlled trials establish the efficacy of a given therapy, implementation research can generate new knowledge about barriers to uptake of the therapy into widespread clinical care, and what strategies are effective in overcoming those barriers and in addressing health disparities. Comparative effectiveness research aims to elucidate the relative value (including clinical benefit, clinical harms, and/or costs) of alternative efficacious management approaches to a neurological disorder, generally through direct comparisons, and may include comparisons of methodologies for implementation. Congress has recently appropriated resources and established an institute to prioritize funding for such research. Neurologists and neuroscientists should understand the scope and objectives of comparative effectiveness and implementation research, their range of methodological approaches (formal literature syntheses, randomized trials, observational studies, modeling), and existing research resources (centers for literature synthesis, registries, practice networks) relevant to research for neurological conditions, in order to close the well-documented “evidence-to-practice gap.” Future directions include building this research resource capacity, producing scientists trained to conduct rigorous comparative effectiveness and implementation research, and embracing innovative strategies to set research priorities in these areas.

Priorities in pediatric epilepsy research

“…57 Reasons for and the impact of this variation on patient outcomes are unknown, but there is extensive literature on the role of implementation science in linking evidence to improved population outcomes. 58 CONCLUSIONS Early-onset pediatric epilepsy is a high-stakes condition. Early seizure control is of supreme importance to parents and is an overriding goal for clinicians.…”

A simple behavioral–developmental checklist versus formal screening for children in an epilepsy center

“…Formal screening, as recommended by the American Academy of Pediatrics [6,9], with a wellvalidated measure such as the ASQ-3 is ideal and should be done whenever feasible. There is a recognized gap between screening guidelines and implementation of those guidelines in clinical practice [14,15]. Such screening requires resources that may not always be available.…”

Treatment of infants with epilepsy: Common practices around the world

“…Efficacy is based on the outcome not of complete seizure control but 50% efficacy whereby seizure frequency is halved for most pharmaceutical company-driven studies. 17,18 Although the respondents did include phenobarbital in some of the responses even from resourceequipped settings, this was a less frequent selection compared to the other AEDs. Based on this global preference, it brings to question the common practice, even in resource- limited settings.…”