Researchers Increasingly Conduct ‘Real World’ Clinical Trials To Discover How Drugs Work in Patients With Complex Health Problems

The New York Times on Tuesday, in an occasional series called "The Evidence Gap," examined the "growing movement" among researchers to conduct "pragmatic," or "real world," clinical trials to gather evidence "that will fill some of the biggest gaps in medical science: What treatment is best for typical patients with complex symptoms?" 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."

The Times profiles a current pragmatic study focused on nesiritide, a heart drug approved by FDA to ease breathing. The study, paid for by the drug's maker Johnson & Johnson, is the largest of its kind, and patients unable to breathe from heart failure who seek care at one of 450 medical centers around the world are eligible to enroll.

According to the Times, "a move to conduct many more pragmatic trials would involve nothing less than a rethinking of how medical research is financed and managed." 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).