May 29, 2020

The Niche

Knoepfler lab stem cell blog

Huge clinical trial patient fees allowed by FDA at times, details often secret

stem cell monopolyI am often critical of for-profit stem cell clinics on this blog for numerous reasons. For instance, one thing that concerns me greatly about these clinics is that they charge patients to get experimental “treatments” that have not been proven to be safe or effective.

But as some patients have pointed out to me over the years, certain FDA-approved stem cell clinical trials also charge patients to receive unproven stem cell therapies.

Should someone (even if that someone is an academic clinical researcher) be able to charge a patient a large access fee to be in a clinical trial in which that patient will be subject to an experimental therapy that could ultimately be proven unsafe and ineffective?

I have serious doubts about this.stem cell monopoly quote

You might be surprised to learn that the FDA in some instances allows patients to be charged to be in trials including stem cell trials and others. Those running the trials can request permission from the FDA to charge patients. The agency then has to evaluate the request. The FDA has a draft guidance on this, but frankly it’s difficult to learn that much from that document in terms of details in how this plays out.

One major consideration seems to be whether the clinical trial needs that extra money drawn from patients for the trial to even be done at all and another is whether there are existing data to at least preliminarily support the clinical trial hypothesis.

I’ve tried to talk with the FDA about the details of how it decides on whether to allow clinical trials to charge and asked about specific trials that appear to charge, but no luck so far from the FDA. On the latter point, it is literally a secret as to the details of how the FDA has decided on particular cases. If there is still an investigational new drug (IND) involved or even if there was an IND involved in the past, but the trial was cancelled, then the FDA stays mum on the charging issue. Because INDs are by definition considered to contain “trade secrets” the FDA believes that it cannot comment on them or release information on them. While I respect the need for confidentiality on stem cell biologics in development, this situation also means the public is kept in the dark.

Of course if patients who had paid an FDA-approved clinical trial fee, which apparently can range from $5,000 to above $125,000, felt so inclined they could publicly talk about their experience having to pay to be in a trial and provide details, right?

Not always as non-disclosure agreements are pretty common. Patients also may not realize that the fee had an official FDA OK in some cases. Some patients have also shared with me that they would be uncomfortable talking about fees out in the open.

Why do patients go ahead with essentially paying to be experimented upon?

They may feel that they have no other choice but to get a stem cell therapy from a clinical trial that charges a big fee because such trials may seem like the only hope to the patients. It’s like a monopoly situation but where patients have to pay thousands of dollars to pass Go to get to the stem cells instead of getting $200 themselves (i.e. the trial paying the patient as used to be more common).

I’m working to learn more about a possible trend whereby even what are considered to be legit trials possibly are increasingly going to require patients to pay often large sums of money to participate. In part I’m doing this because there is a move underway led by the Bipartisan Policy Center to mainstream the idea of patients more often having to pay access fees to trials.

In a non-scientific poll here on the blog, about 70% of respondents opposed this so-called “pay-for-play” stem cell trial idea. While the way I did this survey using the term “pay-to-play” likely biased the results, they were still fairly strong in the trend that was apparent.

Whether it is a stem cell clinic or an FDA-approved clinical trial using an experimental stem cell intervention, if patients have to pay to get access, then I believe such situations should be closely examined, have the light of day shone on them through greater transparency and require consultation with bioethicists. Note that the focus of this post is on “pay-to-participate” trials, which are most often distinct from patient-funded research. This distinction and the latter kind of research will be the focus of a future post.

Other resources (HT to Leigh Turner)

Doug Sipp, “Pay-to participate funding schemes in human cell and tissue clinical studies,” Regenerative Medicine 2012; 7: 105-111.

Danielle Marie Wenner, Jonathan Kimmelman, and Alex John London. Patient-Funded Trials: Opportunity or Liability? Cell Stem Cell 2015; 17: 135-137.

Ezekiel J. Emanuel, Steven Joffe, Christine Grady, David Wendler, Govind Persad, “Clinical research: Should patients pay to play? Science Translational Medicine 2015; 7: 298ps16.

Stuart E. Lind. “Fee-For-Service Research,” New England Journal of Medicine 1986; 314 (5): 312-315.

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