The Randomized Registry Trial — The Next Disruptive Technology in Clinical Research?

NEJM: 9/1/13

The randomized trial is one of the most powerful tools clinical researchers possess, a tool that enables them to evaluate the effectiveness of new (or established) therapies while accounting for the effects of unmeasured confounders and selection bias by indication. Randomized trials, especially huge megatrials, have transformed medical practice. Thanks to randomized trials, we no longer, for example, treat acute myocardial infarction with lidocaine and nitrates. Instead we use rapid revascularization, anticoagulants, and antiplatelet agents, and during long-term follow-up we routinely prescribe statins, beta-blockers, and angiotensin-converting–enzyme inhibitors. But the reputation of randomized trials has suffered of late,1 owing to reasonable concern about excess complexity, expense, and time required to recruit study participants, as well as inadequate representativeness. What good are trials if the results aren’t applicable to real-world patients and if, because of excessive expense, they can be used to answer only a tiny fraction of our important clinical questions? Read more