Innovation in Funding Research

"It's a model of funding that I think we are going to see being proposed more and more in the coming years."
"It's co-opting the kind of scientific aura that we associate with clinical trials. [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."
Jonathan Kimmelman, bio-ethicist, McGill University, Montreal

"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."
"Patient-assisted clinical trials — where all parties bear a portion of the financial risk — are an ideal way for society to advance clinical trial initiatives … when no other external financial resources are available."
Dave McGuigan, vice president, Stemedica, San Diego

novastem.mxNovastem, a stem cell treatment clinic, is currently running 14 clinical trials partly funded by patients.

"Pay-to-participate" clinical trails have become a new formula for funding clinical trails in launching research investigations that range the gamut from impotence treatments to multiple sclerosis medication. Research start-ups can no longer count on funding support from government agencies or from conventional sources of funding, and some innovation was seen as a way to get projects off the ground that would otherwise never be initiated.

One example is a new bio-sciences startup, Regenastem Inc. with operating space at an incubator operated by a small Ontario university. The company had a vision of cell-based arthritis and spine treatments which they began developing with a research company based in Buffalo, New York. And that research into that treatment is being funded by the very patients who hope to be able to take advantage of the findings that they expect will help to manage their medical problems.

Stem-cell therapy, a new procedure that holds great promise for the future, is promoted by some bio-sciences groups on the basis of inviting patients to themselves help fund the trials. So patients who have the wherewithal are now placing their faith in direct funding of treatment that will be beneficial to them.

Since most trials require that there be two groups of subjects, one half of which will receive a placebo, the other the medication or treatment the trials are based on, in a blind test, it is anticipated that people who fund the program won't be thrilled to be selected at random not to receive the treatment, but a placebo.

Getty Creative Images  People who pay to be part of a study will naturally be unwilling to take the chance of receiving a placebo, one critic of patient-funded clinical trials says.

That, clearly, looms as one of the drawbacks of the self-funding regime. Critics of the self-funding model warn that ethical problems may arise which in and of themselves could produce harmful effects. The placebo problem is certainly one of them, but no tests can be run without the treatment in question being withheld from a blind [neither patient nor doctor knows which patients receive the drug and which do not] control group.

The new experimental pay-to-participate holds great promise, however, with investigation into the treatment of rare diseases that pharmaceutical companies don't want to invest funding in on their own, because the returns don't make financial sense to them. There is simply a too-narrow pool of patients to justify large research investments in finding a 'cure' or appropriate medication that will help cope with the symptoms of a rare disease.

Funding sources for clinical trials have always presented as a dilemma; without funding the trials can't be planned and pursued. Government funding bodies for health research have been less generous in their acceptance of research proposals as governments cut back expensive outlays in response to tighter economic realities. So this less-than-conventional model, of inviting the patients themselves to invest in their own future medical well-being has been launched.

Ageless Regenerative Institute based in Florida, for example, had authorization from the U.S Food and Drug Administration to treat erectile dysfunction with stem cells derived from body fat. Regenastem, at Brock University's Biolinc St.Catharines facility, isolates stem cells from fat tissue and has set up two trials, charging each patient $7,500 to be involved in the trials.

The two trials involve the removal of cells and blood from the patients' bodies to be processed and re-injected to treat arthritis and disc-related pain. Not too steep a price for many patients whose symptoms could be relieved and give them that proverbial new lease on life, potentially. At the same time, researchers gather scientific data and doctors provide vital care for the patients.

The charge per patient just covers operational costs. Which in and of itself constitutes a concern to Dr. Kimmelman, who observes that experimental treatments generally use as few people as necessary for safety reasons. With patient funding, however, he sees a financial incentive looming that the more patients enrolled the larger the funding pool, thus exposing an unnecessarily large number of people to possible side effects.

"This can tarnish a reputation of a new and emerging research area. There is some threat to actually undermining the progress in certain areas", Dr. Kimmelman cautions.