Clinical Studies: The Elephant in the Room

“This product is not intended to diagnose, treat, cure or prevent disease.” Have you ever asked yourself why this statement is on your supplement container? It’s a good question, with an answer that is somewhat complex, while also simple. The simple part of the answer is money; the more complex part of the answer is clinical trials, an integral part of obtaining government approval of a drug that is intended to “treat, cure or prevent disease.”

Recent estimates of the cost to bring a new drug to market range from just under $1 billion, to as much as $4 billion. These estimates include costs associated with failed trials and other expenses incurred for drugs that would never make it to market. A significant portion of this cost is consumed by the planning and execution of clinical trials, studies that are critical to receiving regulatory approval.

The typical drug development process includes human studies of three phases. Phase I studies are the first in-human trials conducted and are usually quite small, involving perhaps 20 – 80 participants. These trials are not designed to determine efficacy of a drug, rather they are designed to determine an initial safety profile and monitor for potential side effects. Phase II studies are a bit larger, usually with 100 – 300 participants. These studies further monitor the safety of the drug but also begin to look at efficacy.

Phase III studies are the largest, most complex, and most costly studies in the drug development process. Phase III studies are often considered “pivotal” studies, as the information gathered is critical to obtaining regulatory approval. These studies typically involve thousands of participants, unless the target indication is a rare disease for which participants would be difficult to find. The “gold standard” in Phase III studies is the randomized, double-blind, placebo-controlled design. Under this study design a “control” group is included. This control group often would be given a placebo, while other participants would be given the drug being studied. The purpose of having a control group is essentially to have a baseline against which to compare results from the drug-dosed group.

What does this have to do with nutritional supplements? As mentioned above, clinical studies are costly. A Phase III controlled study can cost tens of millions of dollars. For a company to invest upwards of a billion dollars to bring a new drug to market, they must be certain they can realize a significant return-on-investment. For every drug that makes it to market, many fail. Therefore, those that make it to market must bring in enough revenue to pay for all programs in the future, both successful and otherwise. If you have ever been prescribed a prescription drug by your doctor, you know just how much drugs can cost.

Nutritional supplements, on the other hand, are relatively affordable and in many cases are known quantities. For example, the roles of vitamins in physiological function are well known and the cost to produce a supplement containing them is reasonable. As a result, it is unlikely that complex and costly clinical studies will be executed on vitamins, or on most other supplements. The return on investment is simply too small, given the affordable nature of supplements.

So, if supplements aren’t studied in clinical trials, how can we know if they have benefits? The simple answer is to do your homework. The ingredients in many supplements have been studied retrospectively through meta-analyses. There is much evidence to support the benefits of most supplements, but discovering the information can take some work. Reading the blogs on the Seven Lakes website is a good start, as they are based on a significant amount of information available through research papers.

In summary, dive right in and educate yourself, reviewing many sources of information so you gain a solid base of knowledge to make decisions on what supplements make sense for you. And remember, just because a supplement has not been subjected to clinical studies, does not mean it can’t have benefit as part of your balanced and healthy lifestyle.