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Selecting Health Claims for Nutraceutical Clinical Trials


by Jayesh Chaudhary, BPharm, MS

Determining the primary question to be asked in a clinical trial of a dietary supplement is critical to its success. Ask a wrong question and you end up with the wrong answer, no answer or an answer that has no market or regulatory value. A complex human trial can be compared with a simpler scientific experiment consisting of a substrate (the patient or disease state), the reagent (the supplement or the intervention) and the endpoint (the outcome). Of course, clinical research has an added dimension of medical ethics that is absent from other research. In any study the scientist must smartly choose the correct substrate and watch for the mostly likely endpoint to prove a certain property of the reagent. In other words, selection of the wrong subject population and impractical clinical endpoints may result in failure of the clinical trial objectives.

How does one take these critical decisions regarding claim selection/definition and substantiation? First, look at what happens in conventional drug discovery and development, which provides lessons for planning effective clinical trials for dietary supplements. Next, consider all research decisions from the point of view of the investigator and, more importantly, the trial subject (the patient) in addition to the sponsor’s interests.

Prior Data Available for Trial Design

When a drug molecule enters Phase II clinical research, a trial phase that is comparable in its objective to typical nutraceutical trials, a lot is already known about the molecule from preclinical work. For most molecules entering Phase II, the target site of action, exact indication, disease sub-type, stage/severity of disease, probable safe and effective dose, expected duration of therapy, onset of action and half life of the drug in the body are known factors. The concept of the new drug already exists and needs to be proven in humans (Proof of Concept Study). The safety of the new chemical entity (NCE) in healthy humans in terms of maximum tolerable dose (MTD), likely adverse effects, etc., are also known through Phase I trials. The safety of the drug in patients is studied in further Phase II/III trials.

A systematic review of available data about folklore; identity, quality and strength of ingredients; and biological effects will help compile a respectable Investigators’ Brochure (IB) which will not only reassure IRBs, but also stimulate the imagination of investigators. They can suggest outcomes patients long for and value, which should therefore be measured or counted. For example, if a product facilitates epithelial healing (effect), and is shown to reduce the time required for re-epithelialization (efficacy), patients must find earlier or faster relief of symptoms (effectiveness).

For this discussion, let us consider only supplements that go beyond a nutritional benefit and are chosen to be studied for “hard” claims. While current regulations in most countries will allow only normal structure/function or quality-of-life type of claims, it is common practice to study the effect of nutraceuticals on a disease state. In the eyes of the regulators, even a “clinically proven” therapeutic product can not be labeled as a drug until it goes through the IND-NDA process consisting of several phases of human trials. Yet, it is often beneficial to have supplement studies done under an Investigational New Drug (IND) Application to FDA or local authorities to lend the study some credibility and improve quality of the research.

In comparison to NCEs, a typical herbal supplement that enters a human clinical study for the first time is not armed with much data. A lot can be dug out from folklore or traditional use of herbal remedies; but, a lot is often lost in translation from a traditional medical book to a modern clinical research protocol. Finding someone trained in both sciences—complementary and alternative medicine (CAM) and clinical pharmacology—can fill the void due to inadequate preclinical data. In the event that the supplement or ingredient is lacking in both ethnobotanical and in vitro/in vivo animal data, the task of choosing the correct label claim for the clinical trial becomes even more challenging. Such trials are just based on popular use without an established basis.

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