More Prescription Drug Researchers Turning to ‘Real World’ Trials
There is a "growing movement" among pharmaceutical researchers to conduct "pragmatic," or "real world," clinical trials to gather evidence aimed at discovering how treatments will affect typical patients with complex conditions, the New York Times reports.
According to the Times, "thousands of medical studies are completed every year," but "most have relatively limited goals." The Times reports that while such limited studies "can have value," they "may no longer be enough, particularly when care has become so expensive and real evidence more crucial."
Sean Tunis, director of the Center for Medical Technology Policy, said, "There's this gulf between what questions researchers have found interesting to study and what questions industry and the NIH have chosen to fund and what users of information most want to know."
Robert Temple, director for medical policy at FDA's Center for Drug Evaluation and Research, said there are practical concerns with using large pragmatic trials because researchers must account for a wide range of possible effects when testing a drug. He said, "If you do a large outcome study in 10,000 people in the same way you do short-term studies, you'll never finish."
Steven Goodman, oncologist and biostatistician at Johns Hopkins University School of Medicine, said that pragmatic studies "sound wonderful" when they are first described but that they often require researchers to give up the "why." For example, a pragmatic trial will show that a surgery is not working, but it will not show why because it may not be aiming to answer that specific question.
A group of advocates -- including medical researchers, medical societies and insurers -- is lobbying Congress to provide funds for an Institute for Comparative Effectiveness Research, which would assess treatments and identify evidence gaps. The center also would initiate pragmatic studies (Kolata, New York Times, 11/25).This is part of the California Healthline Daily Edition, a summary of health policy coverage from major news organizations. Sign up for an email subscription.