Michele Russell-Einhorn has spent 23 years working in the world of institutional review boards, which decide whether to green-light clinical trials. In all that time, she’s never seen quite so many proposals as she has in the past year that rely on an unusual — and controversial — funding mechanism.

Some plan to ask participants to pay $7,000 or so to enroll. Another wanted to ask for upward of $250,000.

In each case, there were “serious concerns about how ethical it was to charge people to participate in the research — and whether it was absolutely necessary,” said Russell-Einhorn, chief compliance officer for Advarra, the second-largest commercial IRB.

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These studies have caught the attention of federal regulators. The Food and Drug Administration recently asked a federal advisory committee to consider how the research community should think about such trials, an agency spokesperson confirmed. Members of that panel are now drafting recommendations on the issue. And the National Institutes of Health asked the committee to consider whether its existing resources to guide patients considering a clinical trial are adequate for scenarios in which they are being asked to pay, a spokesperson for that agency confirmed.

The new scrutiny reflects mounting concern about these trials at a time when they’re perceived to be increasing in volume — and when several have attracted media attention.

Last year, STAT reported on a plan in Florida to charge seniors a fee proposed as high as $285,000 to enroll in a clinical trial in which they would get young-blood transfusions to try to forestall aging. That proposal was part of the reason that the Health and Human Services Secretary’s Advisory Committee on Human Research Protections — or SACHRP — decided to take up the issue, according to Holly Fernandez Lynch, a University of Pennsylvania bioethicist who sits on a subcommittee that’s helping draft the recommendations.

In the same field, the startup Ambrosia recently ran a clinical trial that charged about 80 people $8,000 each to get an infusion of blood plasma from a young donor — before halting its business in response to a warning from the FDA. Meanwhile, a clinic in Panama enrolled 20 autistic children in a stem cell trial that charged parents $7,200, the science news site Spectrum reported last month

FDA regulations permit such trials in “extraordinary circumstances,” such as when the drug being tested has a price tag so high that the trial couldn’t otherwise be run. (Asked why FDA recommended that SACHRP take up the issue of pay-to-participate trials, an FDA spokesperson said the agency suggests topics that its believes “may be of interest to a wide range of stakeholders.”)

But experts say these trials are ethically fraught for a number of reasons: At worst, they run the risk of being a guise for an enterprise looking to profit from desperate patients. But even when intentions are sound, the trials are likely to only enroll those patients who can afford to participate, thereby skewing the results. There could also be a weak study design, without blinding or a control group, because patients would likely be reluctant to pay for a placebo. And they may reinforce the common misconception among patients that clinical trials are a route to accessing treatments that are guaranteed to help them.

Still, SACHRP doesn’t want to rule out the possibility that such trials can in rare cases be valuable, Fernandez Lynch said.

“The examples that we have, that get this kind of media attention, it’s obvious that people shouldn’t be asked to pay to participate in those trials,” Fernandez Lynch said. “But there are some where we think we don’t want to have a hard-line stance that says no, never, this could never be appropriate, because sometimes, we think, in rare circumstances, it could be ethically acceptable to ask for this kind of cost-sharing.”

Fernandez Lynch pointed to research around medical uses of illicit drugs, like medical marijuana and psychedelics, that might have a hard time getting funded through traditional channels, though she acknowledged that that field has increasingly been finding mainstream funding.

But Dr. Steven Joffe, a pediatric oncologist and bioethicist at the University of Pennsylvania, worries about making exceptions for even a few trials.

“If you open the door to these trials, you inevitably are going to get … exploitative trials, and bad trials, and trials that don’t lead to any useful information,” Joffe said.

Sure, Joffe said, a blanket policy against pay-to-participate trials would mean foregoing a few potentially valuable trials. “But you are also closing the door to a lot of potential abuse that is very likely to happen — and overall the harms of opening the door outweigh the benefits,” he said.

A draft of the recommendations discussed at a SACHRP meeting last week offers a hint of the balancing act that the panel is trying to strike. The recommendations urge IRBs to consider questions like, “Does the study meet relevant thresholds of scientific quality?” and “Is the risk-benefit balance acceptable?”

SACHRP doesn’t expect to finalize its recommendations on the subject until its next meeting in December or its subsequent one next March, said SACHRP committee member Nancy King, who’s also a bioethicist at Wake Forest School of Medicine. When finalized, SACHRP’s recommendations will be delivered to HHS Secretary Alex Azar, and they’ll be meant to offer general guidance for researchers, patients, IRBs, and regulatory agencies.

Good data are lacking on the volume of pay-to-participate trials, in part because the researchers running them don’t always disclose that information when they announce their results or list their studies on the government’s human study registry, clinicaltrials.gov.

“Whether it’s just that we are seeing more of it — there’s more attention, there’s more press, there’s more academic study of the phenomenon — or whether there’s actually truly growth in the phenomenon, I think is hard to say,” Joffe said.

The largest commercial institutional review board, WIRB-Copernicus Group, didn’t return STAT’s requests for comment on how many of these study protocols it’s seeing.

At Advarra, they’re still relatively rare. Russell-Einhorn estimated that, even with the surge this past year, they represent just a handful of the thousands of protocols that she and her team review annually.

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  • We have published a response to the original article on Spectrum News here: https://www.cellmedicine.com/stem-cell-institute-response-to-article-in-spectrumnews-org-experts-question-rationale-for-stem-cell-trial-for-autism/ It includes all 21 questions Ms. Furfaro asked Dr. Riordan with his complete, unedited answers. We also address various errors, omissions and differences of opinion, including that fact that Stem Cell Institute and Medistem lab did not charge subjects any fees. The fees charged were for outside services, not for harvesting, processing, storing or administering the stem cells, which have a retail cost of around $64,000 for the number of injections and dosage each child received.

  • There are a lot of omissions and errors in that SpectrumNews.org article about our autism clinical trial. Our rebuttal is here: https://www.cellmedicine.com/stem-cell-institute-response-to-article-in-spectrumnews-org-experts-question-rationale-for-stem-cell-trial-for-autism/

    Neither Medistem Panama (lab) nor Stem Cell Institute (treatments) profited from the autism trial. As stated before, the $7,200 covered services that were provided by companies outside of the lab and clinic. All lab and clinic services (cell harvesting, cell expansion, cell testing, cryogenic preservation, syringe prep, 16 IV infusions, doctor visits) were provided free of charge. The current retail price for 16 IV infusions at our clinic is approximately $64,000. While not common in well-funded clinical trials, charging subjects for outside services in smaller, less well-funded trials is not without precedent. For example, Duke and Northwestern have both charged clinical trial subjects in the past.

    We have also completed a clinical trial for MS in 2018 (https://www.clinicaltrials.gov/ct2/show/NCT02034188) Subjects in this trial were not charged. We are currently finishing a trial for rheumatoid arthritis (https://www.clinicaltrials.gov/ct2/show/NCT01985464) Participants in this trial are not being charged. – Jay Lenner, VP International Operations, Stem Cell Institute, Panama

  • I got one of that sort of thing in the mail yesterday. They said the thing (which I did not have anyway) was incurable but they wanted me to participate in the “free” clinical trials anyway, and buried in the fine print was that they had to charge a fee. Didn’t even say what the fee was.

  • The article doesn’t mention if Advarra and other commercial IRBs have agreed to serve as the IRB for these studies. Despite these ethical quandaries, it seems likely that Michelle Einhorn and other IRB for-profit entities will accept their business until the government (assuming it does, which isn’t necessarily likely) takes a position against them.

  • Short cut to manufactoring here is one for you. Dr friedman admits 80 Per cent of his trials were frauduant so is that why lupron has destroyed thousands of women,created disabled babies,and no one will speak up.A FDA agent said it should have never been marketed. Or is it due to Obama and Podests involvement in FDA upper management like FBI
    it is all there at lupronvictimshub.com even the witness for a young girl lies inder oath. Who is the Pharma. co paying off?

  • The public should be made aware that the FDA has been strategically underfunded for years. These pay to play clinic trials have already been used to market untested products to desperate people. Laws and regulations about health marketing are no longer enforced by the FDA or FTC.

    That explains why most American don’t know that most of their pharmaceuticals are manufactured in China and India. The FDA has not been inspecting these overseas factories, the positions that would have were cut or never filled at the request of pharma industry lobbyists. It was just cheaper and easier to manufacture these drugs overseas, and then spread lies and propaganda about it. It shows the unmitigated greed in the pharma industry.
    Even our national security interest was co opted by greedy corporate lobbyists, because the DOD has to rely on China for pharmaceuticals, that are produced in China.

    Pharma lobbyists and paid industry trolls attacked people who brought up fact based information in online forums. Healthcare lies and propaganda are not only deadly and dangerous, they are a serious public health issue.

    We are seeing the real damage with health outcomes in the US getting worse, and trust in the industry going downhill. Our news media did not explain how many drugs were manufactured overseas, the corporate media found it was more profitable to mislead the public.

    The FDA made false claims about importing cheaper pharmaceuticals from overseas, because they left out the fact that they were already manufactured overseas. Of course due to Regulatory Capture and industry pressure they were not allowed to bring up a serious public health and national security problem. It was a lie by omission.

    Most of the health related sites on the Internet are funded by big pharma and other industries. In order to please their advertisers, they offer limited information, and mislead vulnerable sick people. The paid clinical trial is just another iteration of for profit market based healthcare. No patient or physician has accesses to factual data about these companies, they have to read between the lines. Most of the information is proprietary, and institutions we used be able to trust for factual data are no longer reliable.

    The FDA has been co opted by big pharma, and other industries. They no longer protect the health and safety of Americans. They hid millions of adverse events on drugs and devices over the years to protect certain corporations. CMS the Center for Medicare Services, has been sidelines by these industries too. CMS is not allowed to collect certain data, anything that could lead to an unprofitable result for pharma and other industries.

    We are all being Gas Lighted by the corporate interests. We are paying dearly too. This costs us billions every year. A number of drugs have been secretly recalled or replaced, when they were found to have different ingredients, and even poison in them, like Valsartin. This drug was manufactured by several companies in China, and each one found a cheaper short cut in the manufacturing process. This led to a drug which contained poison. Back in 2007 the news was suppressed, when dirty manufacturing plants in China and India were found to be using drywall in the pills they manufactured for our market. Some of medications had no active ingredients, of course physicians were never informed. They were told that this could lead to liability so they should ignore it. For some patients this had deadly consequences but that story was suppressed.

    The greed and corruption in big pharma is proof that we need Medicare For All. Theses greedy corporations intruded on our democracy, when they paid our politicians to look the other way. They used their influence on media too, bringing us to a so called opioid epidemic, but even that did not bring any scrutiny or prosecutions. These corporations are above the law!

    • Short cut to manufactoring here is one for you. Dr friedman admits 80 Per cent of his trials were frauduant so is that why lupron has destroyed thousands of women,created disabled babies,and no one will speak up.A FDA agent said it should have never been marketed. Or is it due to Obama and Podests involvement in FDA upper management like FBI
      it is all there at lupronvictimshub.com even the witness for a young girl lies inder oath. Who is the Pharma. co paying off?

  • Combining my comments about this on Twitter. (For context, to me this isn’t a hypothetical: I’m someone who survived a near-fatal kidney cancer in 2007, by participating in a clinical trial. Having said that, a decade of advocating for patient interests around the globe, since then, has opened my eyes: I work WITH pharma companies and have also seen the worst of the industry’s behavior. It’s a life-saving industry struggling to maintain its margins in an era of profound change, and often harming people in the process. This is not a one-sided issue.)

    As usual for Twitter, these are a bit choppy:

    Repulsive. Greedy. The worst of pharma trying to find SOME way to stay rich now that blockbuster drugs are in the past.

    Cranking up insulin prices isn’t enough – some don’t sell it. Cranking up purchase prices is gone to get spanked down.

    Billing people for NON-PRODUCTS?? Ew!

    Of course there are the ethical / equity concerns: nobody poor would have access. But then someone’ll lobby to add it to taxpayer-funded things – again, for a product that doesn’t exist yet!

    I also strongly suspect they’d have real difficulty turning down someone who doesn’t really qualify but can write that $200k check to try anyway … skewing the trial. [<= as the article above says]

    BUT! Switch to the patient perspective – if my baby's dying and I WANT to pay to try something unproven, who's to say no? (My answer on that: fine, but don't call it a clinical trial.)

    All in all the short answer is "Sure! What could go wrong??" Um, everything.

  • The concept of making patients pay to participate in a clinical study dismantles the partnership that should exist between the clinical trial participants and the company running the study. The essential point of a clinical study is to see if the drug is safe enough and effective enough to be approved. And the gold standard for most pivotal studies (obviously with some exceptions) is that it is double blind and placebo or active controlled. So is the next step to start asking patients in these types of studies to pay for a 50/50 chance of getting the study drug? And what good are these studies going to be when the patient population is skewed toward only those who can afford to pay? That will affect the results.

    And the fact that these studies are being posted on clinicaltrials.gov, with no indication that the patient will have to pay to participate? Why aren’t they adding to the inclusion criteria “money to give us so that we can risk your life with our unapproved intervention”? Because that risk is the patient’s true “cost” in a clinical study. They are taking a risk in agreeing to participate in a clinical study. That is “payment” enough.

    Patients are the lifeblood of clinical trials. Drugs exist that cure diseases thanks to patients who risk their lives in these studies. Sometimes these cures are at the cost of the lives of some of those patients. My uncle had childhood leukemia. What essentially killed him (it was advanced, he wasn’t going to survive regardless) was the toxicity of the–at the time experimental–chemotherapy. Imagine the lives in the past 50+ years that have been saved, in part, because of patients like him. Imagine the treatments we would have lost if there was no one willing to take that risk. Asking people to pay dismisses the critical importance of clinical trial participants to the success of the study.

    I agree with Dr. Joffe. There should be a blanket policy against pay-to-participate trials. He is completely right. The risk of missing a few possibly valuable trials doesn’t compare to the likely abuse that would happen (and that apparently is already happening).

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