Clinical Trials vs. Mode of Action Studies - 101

Prior to introducing a new ingredient to market, it is important to verify its safety and effectiveness for use in humans. Well-designed research not only supplies this substantiation, but it provides the basis upon which Structure/Function claims can be made. Since conducting research is an expensive endeavor, ingredient developers must determine where to start in deciding what types of studies to undertake.

Safety studies are paramount and are necessary for a new dietary ingredient (NDI) or self-affirmed GRAS submission. These usually entail a series of in vitro and animal studies that can provide a sound basis for determining the upper dosage levels and safety for long term use.

For ingredients in which the safety in humans has already been confirmed, most companies will opt to conduct a human clinical trial (interventional study). The FDA and FTC prefer that these studies be designed as randomized, placebo-controlled trials (RCTs) to substantiate claims. Ideally, they should be double or tripled-blinded. However, observational studies can also provide valuable information regarding an ingredient’s effectiveness and can provide additional support for RCTs.

Another type of study that is critical for fully understanding an ingredient’s safety and functioning is a Mode of Action (MoA) study. MoA studies are usually in vitro cell culture or in vivo animal studies, and can give critical information regarding how an ingredient may be expected to function in the body as well as what body systems or types of cells are impacted. Sometimes these studies can give unexpected insight into how the ingredient works or possibly how it might interact with other ingredients or medications. MoA studies help answer the question of “How?”, after confirming that an ingredient does work, and are especially important in cases where there is an unexpected result in a clinical trial.

Hopefully, this provides some insight into the importance of research studies, and how each has its place in providing support for ingredient substantiation. Now happy researching!

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