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The opportunity seemed too good to pass up. It was 2020, and the CEO of a blood-test company was addressing fibromyalgia patients through their television screens. His firm, EpicGenetics, wasn’t exactly a household name, but its product could help get you in the door at Massachusetts General Hospital. “With a positive test,” Bruce Gillis, the CEO, said, “you can volunteer for an FDA-approved clinical trial for an investigational new treatment to reverse the disease and eliminate your symptoms.”

The hospital, though, wasn’t so sure about that pronouncement. In 2017, Gillis had approached a researcher there, offering to bankroll a study to see whether an old tuberculosis vaccine could be repurposed to treat fibromyalgia. The researcher agreed, and money started coming in. But then, in 2018, once the project got regulatory approval, the payments stopped before patient recruitment could begin. The hospital sent reminders, but still no checks appeared. So the researcher was surprised to learn, in 2021, that EpicGenetics had been promoting its FM/a Test as a gateway to her study for years.

The CEO-philanthropist says he paused funding because of concerns about the study design and because of Covid-19; that doesn’t change the researcher’s sense that she and fibromyalgia patients were misled. It’s an unusual scenario. Among the many philanthropically funded research endeavors, this one’s hardly a textbook case. But the questions it raises are broadly applicable: Who should be allowed to bankroll science, and how? What safeguards should be in place? And how should trials be presented to potential volunteers?

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How is the trial framed for patients?

To Ana Iltis, director of Wake Forest University’s Center for Bioethics, Health and Society, one of the biggest issues is that clinical trials are so often billed as a way to access treatment. “That isn’t the primary purpose of research. The primary purpose of research is to produce generalizable knowledge,” she said.

The practice isn’t limited to privately funded trials. Companies do it. Hospitals do it. Even government-funded scientists do it. Often, they’re all in it together. The hospital has name recognition among patients. That attracts trial funders, who need volunteers for research — and the hospital uses that to bolster its own image as the locus of the latest, most cutting-edge experimental treatments. Ideally, it’s all in service of a worthwhile goal — uncovering information that will help people — but it’s easy for the messaging about the trial to advertise more than it can reliably deliver.

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“There’s sort of a symbiotic relationship there,” Iltis said. “Sponsors get the prestige of being associated with well-known medical institutions that come with lots of trust and respect — and access to patients. And institutions are able to present themselves as the place to be, right? ‘If you are a patient, we are your answer.’”

The trouble is that the benefit of clinical research is societal, not necessarily individual.

Often, the patient is not personally better off for having participated. They might’ve received a placebo, or a treatment that will turn out to be ineffective, or an effective treatment at an ineffective dose; they might have suffered some sort of side effect.

That doesn’t mean patients should not see a sliver of possibility in experimental treatments. Rather, it means companies, scientists, and hospitals should avoid inflating the chances of a good outcome. “It is perfectly understandable, and sometimes true, that this is in fact the only possible treatment that you might be able to access. And yet that is not the primary purpose” — nor is it even likely that an investigational treatment will work, Iltis said. “I think it is possible to have hope, while also being well-informed.”

How should research projects be chosen for funding?

The idea that research happens in order to produce generalizable knowledge also carries with it a whiff of social responsibility. As Jonathan Kimmelman, a bioethicist at McGill University, points out, science is a public good, its practitioners often partially trained or funded at taxpayers’ expense. It’s also a relatively scarce resource: Not everyone has the expertise to run a clinical trial for diabetes, say, or pancreatic cancer. There’s often an opportunity cost. Pursuing one hypothesis might entail shelving another one, at least temporarily. “Just because you’re rich doesn’t necessarily give you any stronger moral claim on being able to direct which lines of inquiry we pursue,” he said.

He puts the emphasis on establishing a process to maximize impact. Whether families have banded together to support research on an ultra-rare disease or a company is trying to figure out its approach to a therapy, it matters how you direct your funds within a chosen area. Kimmelman is very clear that this isn’t about limiting scientists’ freedom to look into the ideas that intrigue them. Instead, it’s about making sure that a privileged few don’t steer the research agenda away from projects backed by a scientific rationale.

Some think that market forces help with that: A biotech that keeps running disastrous trials would, you would think, eventually go bust. Alternatively, a foundation could put out a call for proposals, with a panel of experts reviewing submissions, trying to ensure that money is going to the most promising ideas. To Kimmelman, that makes more sense than having a tech billionaire personally call up a researcher to say: Hey, want a few million to look into my pet theory?

A Mass. General representative said its standard vetting process for gifts involves the development office reviewing the source of the funding and the reliability of the donor; extra layers of verification occur when a company is involved in a research donation. In the case of the fibromyalgia trial, the CEO of EpicGenetics offered the gift in his personal capacity, and not through his company.

“The MGH followed its standard process for reviewing a gift in support of research based on the information available at the time. Through this process, we did not identify concerns that would prevent us from accepting the gift,” wrote Harry Orf, the hospital’s senior vice president for research, in an email to STAT.

Who controls the data?

The terms of the funding agreement matter. In 1990, a pharmacist at the University of California, San Francisco, found that the generic versions of a thyroid disorder drug were just as good as a brand-name medication called Synthroid, and could cut American health care costs by $356 million annually. But the research was backed by the maker of Synthroid, and for seven years, the company invoked part of the funding contract to keep the results from being published.

It’s one of the most famous cases in the annals of academic conflict-of-interest, and it helped spur a sea change: Now, many contracts don’t allow for the sponsor to have that degree of control. “What I think is often not seen externally, because it’s an internal discussion, is how that institution has decided — if it makes sense for the research to go forward — what kind of mitigation and safeguards are in place,” said Heather Pierce, a senior director for science policy and regulatory counsel at the Association of American Medical Colleges. If a researcher has been a paid consultant for a pharmaceutical company, for instance, the data analysis might be performed by someone else.

“The ideal is to make it a truly independent trial, so the company hasn’t any influence over the design, how the trial is conducted, how it’s analyzed or reported,” said Andreas Lundh, a senior researcher at the University of Southern Denmark’s Center for Evidence-Based Medicine, in Odense. “But in a lot of cases, that’s not possible. The funder won’t fund the study unless they have some kind of influence.”

Even in seemingly independent trials, he has often seen instances in which the contract allows the funder to review the final manuscript before it’s published.

Who controls when the trial is over?

There are plenty of legitimate reasons for a trial to end before it was supposed to. If the data show that a treatment is so beneficial, for instance, that it’s unethical to continue giving some patients a placebo. Or, conversely, if a drug seems so dangerous that it isn’t right to continue giving it even in a research context. Other times, funding simply dries up. “This happens, sadly, with some frequency in the startup biotech context, where these companies run out of money,” said Jacob Sherkow, a professor of law at the University of Illinois at Urbana-Champaign.

To Lundh, though, one of the most important stipulations when drawing up a funding contract is to make sure that a study funder cannot terminate the project if the results seem commercially inconvenient. After all, data showing that a drug doesn’t work, for instance, can be clinically important, too.

It’s easy to zero in on the potential pitfalls of privately funded studies. But ethicists and legal scholars point out that plenty of them are done in good faith, and end up contributing to the public good. As Iltis put it, “I think all sources of funding have conflicts of interest, or pose potential conflicts of interest. Government-funded research is not a panacea.” In her view, the very nature of research is full of conflicting interests, and there will always be conflicts. “It’s not sort of enough to say, ‘Oh, there’s a conflict.’ The question is: Why does that pose a concern? What worries does that raise? And then what can we do about it?”

STAT+ subscribers can read the full investigation here of how a biomedical CEO and his testing company benefitted from a study that never took place.

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