New ways of obtaining valuable health data continue to emerge. “Big data,” including information from electronic health records and expanded patient registries, along with increased willingness of patients to participate and share health information, are generating useful data for large interventional studies and providing new opportunities for complementary use of multiple data sources to gain stronger evidence for action.
For example, although a randomized, controlled trial (RCT) may show the benefit of a drug, large observational studies can be conducted to refine dosages and identify rare adverse events. In addition, new strategies have been undertaken to increase the efficacy and efficiency of RCTs, including collaborative and adaptive trials to increase enrollment, reduce costs and time to completion, and better identify populations that benefit from treatments. Advances in genomic science may allow for better understanding of unique characteristics in patients that can affect outcomes of RCTs and other.
This introduction is an excerpt of the recent published article in the The New England Journal of Medicine (N Engl J Med 2017;377:465-75. DOI: 10.1056/NEJMra1614394 Copyright © 2017 Massachusetts Medical Society)