Clinical trial success: Design and recruitment

Speaking with NutraIngredients, Dr Jay Udani, CEO of Medicus Research, a California-based clinical research organisation (CRO) with expertise in natural products, explained that when it came to dietary supplement and nutraceutical clinical trials, the biggest challenge and most important factor for success is designing the study correctly from the beginning.

“Getting the recruitment criteria right, so that you have a population that will serve your regulatory and marketing needs – and so that you can make solid claims about these products from that – is crucial,”​ said Udani.

The right planning​

Dr Udani said that when designing a trial, it is important to take time to truly understand the correct populations and end points to study. He said that one of the biggest challenges relating to clinical trials of natural healthcare products is that products cannot be tested in a diseased population, or tested on what could be considered disease end points.

“Designing and running a study on a disease population requires an IND ​[Investigational New Drug status], even if you plan to make no sort of health claim based on that trail. In the eyes of the FDA, and many other authorities, to work on a diseased population something must be a drug,”​ said Udani.

Because of these regulations, it is important to differentiate a diseased population from a non-diseased population, he said. For example in terms of joint health, running a clinical trial for a supplement on people with osteoarthritis would be impossible, as it is a disease – whereas people join pain or decreased mobility with function may be an appropriate population to begin a study with.

“Another good example is diabetes, you cannot run a supplement trial on people with diabetes, however you can look at effects in people with impaired glucose tolerance – which is essentially pre-diabetes,”​ said Udani.

The right people​

On the flip side, an increased recognition of natural alternatives in the market place, plus the economics associated with health care – in the U.S at least – means a lot of people are driven towards self care and natural supplements, said Dr Udani, and this makes recruitment easier.

“It’s not very difficult to recruit populations because there are people out there looking for it. They buy into the supplement trials because they already buy the supplements. As a research organization we can tap into that very easily,”​ he said.

“We also find that recruitment tends to mirror closely the types of people who are more active in the self care area,”​ he added.

According to Udani, the recruitment areas that most people are interested in are: heart health, joint health, cognitive functioning, energy supplements and beverages, and weight loss.

“Everybody is looking for something in one of those categories really,”​ said Udani.

“I think people are also much more interested about performing studies in the natural healthcare products arena because – rightfully so – they believe that the risk profiles of the products are much lower than would generally be seen in traditional studies,”​ he added.

Udani explained that once the issues surrounding recruiting the right population – with the right end points – have been understood and planned for, success will usually follow.

Nutra vs Pharma ​

Udani highlighted that one key difference between a pharmaceutical and a natural healthcare trial is that there is a great deal more pre-knowledge on herbal and natural supplements.

“When you’re performing a pharmaceutical study there are more strict exclusion criteria, because of the known and perhaps unknown potential interactions of these drugs,” ​he said.

“The interesting thing is that with natural supplements, even if the formulation combination is new, and you don’t know a priori what interactions are going to take place. In general there will be historical data on each individual ingredient.”​

He said that this perceived risk, and pre-knowledge is an important factor to many people when being recruited for clinical trials.

“In pharmaceuticals, when you design a novel single chemical drug for example, and do a phase one clinical study, then this is literally the first time that drug has ever been placed in a person, and so there are more unknowns. There is less pre-knowledge,” ​

“On the other hand, if you take a herbal formulation and perhaps do an extraction or reformulation that combines it with other products. You have so much pre-existing data on what that formulation will do, or at least is likely to do from human data, and that is completely different,”​ said Udani.