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 The FDA has a draft guidance on this, but frankly it’s difficult to learn that much from that document in terms of details in how this plays out.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.

9 thoughts on “Huge clinical trial patient fees allowed by FDA at times, details often secret”

  1. Secrecy is antithetical to science.

    In the words of George Washington:
    “Truth will ultimately prevail where there is pains taken to bring it to light.”

    Science takes all pains to bring it to light…

    Who knew that GW was just another scientist at heart?

  2. @Kirk, good question. It’s hard to find answers on this kind of stuff due to secrecy built into the system. Paul

  3. This is a notable issue; kudos to you for airing the topic and pursing it further. In a similar vein, you might also discuss whatever you can glean on fees paid to hospitals and/or docs to run trials, and whether the fees are disclosed in all instances.

  4. One major point you are missing is the Stem Cell Clinics are charging patients under an IRB and not any IND. This in my opinion is wrong!

    1. Stem cells aren’t a drug, that’s why they’re not “FDA approved.” That’s like asking if snot is FDA approved. Therefore, IND (and FDA “approval”) aren’t relevant,

      1. Read-before-posting

        Drugs are any component having properties for treating or preventing disease in human beings. So basically what you just said is that stem cells that are classed as drugs don’t work in any diseases? What a good job the FDA and not you are regulating approval, or we wouldn’t have any stem cell therapies 😉

        And in your short rant you made almost as many mistakes as there are words, e.g.
        – some stem cells ARE defined as drugs, some are not
        – the FDA does not approve all drugs, so “FDA approval” IS NOT a definition of whether or not something is a drug
        – IND is an application to investigate whether a potential drug is safe and efficacious so anyone applying for one MUST BE testing a “drug”

        It amazes me that people churn out comments but apparently cannot read – or cannot be bothered. Back to school with you.

  5. Certainly it is less than ideal that patients would pay for a new type of treatment that may turn out to be useless or even harmful. It is also less than ideal when patients pay for established treatments that are useless or even harmful. Both these things sometimes happen.

    It also happens that some types of treatment may not have much profit-making potential for a drug company. In that case it becomes less than ideal if patients are not allowed to pay for trials. The comeback would be that patients can always make donations to this or that medical charity. I agree, much can be achieved that way. I also happen to think that even more can be achieved by broadening the funding window so that patients can very directly participate and decide where their money should go.

    The idea that there is an ideal system is an unrealistic idea. The advantage of “systems” is that they do the thinking for you. The disadvantage of “systems” is the same.

  6. If “it’s like a monopoly situation where patients have to pay thousands of dollars” then so are most other medical treatments. Altruism only goes so far. One way or another, someone usually has to pay in order to “pass GO” for any medical treatment. Sometimes it’s the patient directly, sometimes indirectly via insurance plans, be they private or public.

    The other definition of monopoly has to do with commercial exclusivity. For profit stem cell clinics are extending the number and types of treatment options — which is more like breaking down existing monopolies… The real issue is whether a stem cell treatment is better or worse than the other treatments for a given malady. From my own experience, sometimes yes they are.

    Stem cells or not, the an old medical aphorism still applies: Eighty percent of sick people will get better by themselves. Ten percent are helped by medical and surgical interventions. The other ten percent are made worse by “accepted” medical treatments…

    In his book, “How to survive your doctor”, Dr Paul Curson puts the matter more bluntly: “Doctors with all their optimism, media hype and high technology rarely cure anything.” But this reality check equally applies to all aspects of the medical industry, from academic researchers to the insurance industry (be it private or government).

    I think that the aphorism contains the essential fact that I have (belatedly) learned to apply when evaluating a treatment option: Does the treatment option build upon the essential fact that eighty percent of the time the body applies mechanisms of its own in order to cure itself? Some stem cell therapies clearly do. On the other hand, oxycodone does not!

    I’m a man of numbers and mechanics. I castigate or praise according to analysis of the numbers and the mechanics.

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