Would You Pay to Participate in a Clinical Trial?

Would you be willing to pay in order to participate in a clinical trial? Not exactly the thing that many people expect when they are considering becoming a participant. In fact, most clinical trial facilities offer stipends for people who volunteer for their clinical studies.

So why would we ask this question? There’s a controversial new trend amongst bio-science start-ups where their studies are being funded by the patients who take part in them.

Unable to secure funding from traditional sources like the government or other pharmaceutical companies, some small drug developers are looking to leverage a crowdsourcing tactic to be able to conduct their clinical trials. This “pay-to-participate” methodology isn’t being met with the sound of crickets either. Startups like Regenastem Inc. have been able to launch studies on everything from multiple sclerosis medications to new impotence treatments.

“It’s a model of funding that I think we are going to see being proposed more and more in the coming years,” stated Jonathan Kimmelman, a McGill University bio-ethicist.

At the moment, the “pay-to-participate” model is being used to fund trials for cutting-edge, cell-based treatments like stem-cell therapy and developing treatments for rare diseases that don’t get as much media attention. These are much more affordable for bio-science start-ups to conduct.

The drug development industry works to bring about some huge changes, but it still can reel at a change in philosophy like this.

What Do the Critics Have to Say?

There are already many critics who have not wasted any time making their concerns known. McGill’s Kimmelman is one of them. In a recent journal article, he and other colleagues warned that these “pay to participate” studies pose several ethical dilemmas that could put patients at harm and produce questionable results.

So what is his main concern? Patients who have paid to be enrolled will want to be given the actual drug, not the placebo. There are two key factors in obtaining clinically actionable data from a study:

• Make use of a placebo “control” group
• Make sure both patients and doctors are “blind” to who is in the control group

In fact, critics question whether these “pay-to-participate” trials can even be called true research studies. Kimmelman argues that they could easily be used to market unproven treatments.

“It’s co-opting the kind of scientific aura that we associate with clinical trials,” says Kimmelman. “(But) if your business model is to sell the trial itself, as opposed to the product, you don’t have strong incentives to design rigorous clinical trials.”

Benefits of “Pay-to-Participate” Clinical Trials

You may have already guessed what the supporters are saying about this new approach. They argue that it’ll help launch many studies that the big boys are overlooking— opening up new doorways into more controversial avenues that could show real promise for the human race.

“As funding becomes harder and harder to secure for all the good work that legitimate clinical trials seek to accomplish, society needs to be open to this evolving creative process,” says Dave McGuigan, the vice president of Stemedica.

His San Diego based drug development firm garnered a lot of press following a stem-cell clinical trial they conducted in Mexico. What had drawn the media’s eye was the fact that Gordie Howe, a hockey legend who set many scoring records in his time, was undergoing stem-cell therapy.

Plugging a Gap Left by Bigger Pharmaceutical Companies

As mentioned before, there are certain research avenues that pharmaceutical companies are less likely to fund. These patient-funded studies could play a significant role in developing stem-cell therapies and other treatments which are harder for these companies to patent and sell.

This is an area where Kimmelman and other critics must concede. In fact, he’s actually not totally against the idea, but he believes it needs to be tweaked. Kimmelman and two colleagues have already proposed a series of alterations that would fix the ethical issues with “pay-to-participate” clinical trials.

Getting the necessary funding to conduct a study has always posed a real challenge. Congress has made budget cuts that significantly affected medical research in the past, and the leash could be tightened at any time.

This has created an environment where more and more clinical trials are being financed in part or entirely by the participants.

So there are studies that are only partially financed by patients? These are called patient-assisted clinical trials. In this model, everyone involved will bear some of the financial risk of the research study.

According to Rafael Carrillo, the CEO of Stemedica’s Mexican partner, Novastem, these types of studies don’t make use of a control (what Kimmelman was concerned about). He has stated that you can’t ask person to pay these sums of money and then expect them to take a placebo. However, Carrillo says that promising results could lead to more rigorous, double-blind clinical trials.

Examples of the patient-assisted or sponsored model can be seen around the country. Ageless Regenerative Institute, a firm based in Florida, has received FDA approval for a new stem-cell clinical trial that will be patient-sponsored. Their research team will be test stem cells derived from body fat as a potential treatment for erectile dysfunction.

Conclusion

Kimmelman has raised one other important concern that he has about these patient-sponsored studies. Patient enrollment numbers are limited because there are risks to testing experimental drugs. The “pay-to-participate” model could incentivize these firms to enroll more patients. This puts more people at risk for side effects and could include some who are just not suitable for the study to begin with.

So given the positives and negatives, what do you think about the emerging “pay-to-participate” clinical trial model? Leave us a comment below.