Clinical trials design best practices for nutraceuticals
Designing clinical trials is tricky business, especially when you’re limited to studying healthy populations. While the pharmaceutical industry has the advantage of conducting studies on diseased populations, where any improvement is easily noticeable, dietary supplement companies can only base claims on studies performed on healthy subjects. These and other constraints typically mean making changes to study parameters or recruiting a larger sample, both of which can be time-consuming and expensive to do.
So how can supplement companies design and conduct sound clinical trials to prove the health benefits of their products? Here’s what clinical trial experts recommend.
Tip 1: Choose Pre-Disease Risk Factors as Study Endpoints
While supplement companies can’t directly research diseased populations, they can examine certain pre-disease risk factors within healthy subjects. Jayesh Chaudhary, founder and CEO of contract research organization Vedic Lifesciences (Mumbai, India), says there are two main types of supplements: 1) lifestyle and wellness supplements, which keep consumers in peak physical and cognitive condition, and 2) condition-specific supplements, which address a condition that is likely to worsen over time.
“There are several early signs of [conditions] that don’t get reported to doctors and hence aren’t termed as ‘disease,’” Chaudhary explains. “We can use these as endpoints for supplement studies.” In India, he adds, nutritional researchers don’t need to file an Investigational New Drug (IND) application with the government—that is, India’s equivalent of the FDA—even if they are testing a condition-specific supplement.
Tip 2: Limit Studies to One or Two Variables
When researching the effects of supplements in healthy populations, clinical trials need to have the necessary statistical power to show a benefit. Chaudhary notes there are several methods of adequately powering studies, but one of the most effective ways is to be selective about dependent variables.
“Choose only the variables that are likely to move noticeably in a short period,” he advises. “Use sensitive equipment, tools, or questionnaires that can spot small changes quickly. Place your bets on only one, or at the most two, key variables, and recruit to sharply control these at baseline.”
By having only one primary endpoint, Chaudhary explains, researchers will have an easier time identifying changes. This endpoint should be carefully chosen, and any other endpoints should be designated as secondary.
Tip 3: Recruit from a Wide Base
There’s no shortage of challenges researchers encounter when designing and recruiting for nutritional supplement trials. Issues like confounding dietary factors, difficulty recruiting or retaining participants, and poor compliance with the study protocol can all derail a trial. Chaudhary says these problems make it necessary to invest heavily in recruitment.
“We need to recruit study participants to meet requirements of the primary variable in a way that leaves room for improvement with the supplement under study,” he notes. “We also need to keep standard deviation as low as possible. Tight recruitment strategies can increase screening failures and costs, but they pay off in the long run.”
Study location may also be an important consideration. Chaudhary says the urban and semi-urban areas of India have above-average recruitment rates and below-average study dropout rates, making India a favorable location for clinical research.
Tip 4: Plan to Run Two Trials
Clinical research can be expensive. While it may sound counterintuitive to invest in multiple studies at the same time, Chaudhary says that planning for two clinical trials instead of one is the most cost-effective approach. Even if local regulators don’t require multiple trials, having a pilot trial and a confirmatory trial makes economic sense.
“The first pilot study helps [create an effective] design, which saves on unnecessary costs in the second confirmatory study,” Chaudhary explains. “It may sound frivolous [to invest in two studies], but this is the cardinal rule of clinical development we recommend, especially if prior human data is sketchy or absent.”
Smart Trial Design Pays Off
While there are challenges involved in studying healthy populations, a robust library of clinical research is still any brand’s best bet for marketing its supplements. Effective trial design can overcome many of the limitations supplement brands face in conducting research, and the potential ROI of a well-done trial is a compelling reason to invest in clinical studies. Consumers ultimately care about efficacy, and no amount of marketing hype will make up for a dearth of clinical validation.