New opportunities and proven approaches in complementary and alternative medicine research at the National Institutes of Health.

This presentation describes some of the issues that arise when applying the clinical-trial approach of conventional medicine to complementary and alternative medicine (CAM) modalities. Conventional medicine has been making the evolution to using an evidence base and to making recommendations only when the evidence is strong. The National Center for Complementary Medicine (NCCAM), one of twenty-five Institutes or Centers of the National Institutes of Health (NIH), is working to hold CAM to the same high standards, not by rejecting previous CAM research, but by building on that strong evidence base of what works and what is safe. The process for conventional drug and device development follows an orderly process of preclinical studies (usually on animals), phase I, phase II, and phase III studies (with the large human clinical trial phase taking place in phase III). Today, the randomized controlled trial is recognized as providing the highest level of scientific evidence. This conventional medicine approach to development is now being used to develop complementary and alternative therapies. For instance, the discovery and development of Taxol (Bristol-Meyers Squibb, New York, NY), an extract from the bark of the Pacific yew tree that is now a widely used chemotherapeutic agent, followed the conventional pathway to approval and marketing. But for most CAM products, the pathway is not so straightforward. Most CAM therapies are traditional therapies or new products that are already available to the public. Most of what is known about these therapies is of an anecdotal nature. There has been little isolation of the active principals from the crude product and there has usually been no preclinical testing. This presentation details various approaches and programs that address how to plan and conduct a rigorous clinical trial of a CAM product. And, while it takes a good deal of persistence and a strong focus on what are the critical principals in a trial, I conclude that it is possible to apply randomized controlled trials to most of the CAM modalities.