At some point in the development of every health innovation, technology, or device there comes a time when it needs to be evaluated and tested to see if it can really achieve the purported health outcomes. This is frequently a critical and often expensive step in the development process and so needs to be given careful thought. When there is an obvious predicate device of specific regulatory requirements from the FDA, many of the parameters of such a study may be out of the hands of the innovator or researcher; however, often the choices and options are nearly limitless, each of which may represent an important trade off.
In the past, the approaches to clinical testing tended to be much more uniform. New techniques or devices were typically tested against placebo or sham in highly selected, homogeneous populations selected to minimize any noise in the measurement of outcome under conditions designed to maximize the apparent effect of the intervention. This may still be appropriate when testing something for which there is no real alternative available and the goal is to demonstrate baseline safety and efficacy.The key step is always to attain clarity on the specific question being asked Click To Tweet
Currently, there is an increasing need not just to show that a new product works, but that it adds value compared to available alternatives. Frequently, modern clinical studies of new innovations must consider the important comparative effectiveness questions and options. One such consideration may include a pragmatic clinical trial in which randomization is used to create equal groups at baseline, but the population, intervention protocol, and comparator treatment are designed to mirror as closely as possible the intended use in clinical practice.
“The Pros and Cons of Pragmatic Clinical Trials” (J Comp Eff Res. 2013 Jan;2(1):53-8. doi: 10.2217/cer.12.74.) discusses the background and details related to deciding upon particular design features for a pragmatic clinical trial.
The key step is always to attain clarity on the specific question being asked which typically follows directly from the value proposition for the device or intervention in question.
Written by Jonathan D. Lurie, MD, MS