ASHINGTON — Scientists are warning that a new proposal by the Obama administration could stifle medical research and undermine major initiatives being pursued by the White House.
The controversial provision would require researchers to obtain consent from patients to use almost all biospecimens — blood samples, tumor biopsies, and organ tissue — even when those samples do not include information that could be used to identify the patients.
The proposal would be part of the first significant changes to the federal requirements for human research in the United States in 25 years, and patient groups and government officials see it as necessary to protect patient privacy at a time when more and more can be discerned about patients from their genetic profile.
Stories like that of Henrietta Lacks, a black tobacco farmer whose tissue was taken in the 1950s without her permission, have heightened the desire for such safeguards.
But scientists are increasingly lobbying against the proposal, arguing that it could inhibit exactly the kind of effort that Vice President Joe Biden has been championing — the collection of vast quantities of tumor samples and analysis of genetic data to accelerate cancer research.
If the rule went into effect, critics say, and if clinicians didn’t obtain consent when tissues were taken, researchers would have to attempt to belatedly track down patients and seek permission to use the samples. Failure to do so would mean the specimens generally couldn’t be used, which researchers say would narrow the universe of samples and thereby endanger good science.
The fight over the issue has largely been hidden from public view, but the questions it raises are fundamental to the administration’s cancer “moonshot” and precision medicine initiatives, as well as the future of all medical research, which increasingly focuses on patients’ unique genes and pooling together huge databases to speed the pace of discovery.
“With the excitement we have going on with sharing information, to have this be, in effect, a setback to all that — to make it more difficult to do that — it’s strange timing,” said Jon Retzlaff, who oversees policy and government affairs at the American Association for Cancer Research.
Currently, if a patient’s tumor tissue is collected at a hospital, and then stored, a researcher who later wants to use it doesn’t have to obtain consent to do so, as long as the hospital has erased any information in its records that tie the sample to the patient.
“But if you’re sitting in the patient’s seat, you’re more concerned about how your data might be used and shared and shared for things you might not agree with.”
Mark Fleury, American Cancer Society's Cancer Action Network
That would change under the administration’s proposal, part of a broader revision of the research regulations known as the Common Rule.
“The bottom line is there is a significant body of research done today that’s clearly not covered under the current rules and would become covered with various requirements needed to be met,” said Michael Carome, a former senior official in the US Department of Health and Human Services’ human research office who is now with Public Citizen, an advocacy group generally supportive of the proposal.
Researchers say they depend on anonymous specimens for their work. And they say that it would be costly and burdensome to obtain patients’ consent as soon as biological samples are taken and then keep track of each sample’s consent status.
Dr. Ellen Wright Clayton, a bioethics and law professor at Vanderbilt University Medical Center, said instituting a similar system to manage the university’s DNA database has cost the school “millions and millions of dollars” and required substantial training for its staff.
Government officials working on the proposal acknowledge that there will be a cost for researchers. But they think it’s a worthwhile trade-off to protect patient rights. Recent research has shown how easy it could be to identify someone with as little as an otherwise anonymous genome profile and a public genealogy database.
Kathy Hudson, deputy director for science, outreach, and policy at the National Institutes of Health, said the agency believes establishing the new rule is “the right thing to do because it is respectful.”
The administration has the support of major patient groups including the American Cancer Society’s Cancer Action Network.
“If you’re sitting in the researcher’s seat, you may be concerned because of the extra burden,” said Mark Fleury, who works on emerging science policy for the cancer organization. “But if you’re sitting in the patient’s seat, you’re more concerned about how your data might be used and shared and shared for things you might not agree with.”
The proposal’s supporters point to troublesome real-life stories to bolster their argument, like the notorious case of Lacks, the subject of a popular 2011 book. Her specimens have been instrumental in major medical breakthroughs — including the development of a polio vaccine — but were taken without her consent. Fortunes were made on the progress aided by the samples while her family, cut out of any financial gains, lived in poverty.
In 2004, the Havasupai tribe in Arizona filed a lawsuit because researchers who had originally taken blood samples to track diabetes risk among the tribe later used those samples for studies on schizophrenia and inbreeding, to which the tribe had never agreed. The Havasupai eventually received a $700,000 out-of-court settlement.
Fleury noted that medical ethics are often updated only after a crisis. “What we’re trying to do here is not wait for that bad thing to happen,” he said.
The government is trying to mitigate researchers’ concerns by stipulating that they can obtain broad consent from patients at the time samples are taken. That consent could be used to authorize a wide range of unspecified research.
“The idea that this informed consent will be in any way meaningful I think is illusory.”
Dr. Ellen Wright Clayton, Vanderbilt University Medical Center
“There’s just no question that’s going to help research,” Hudson said. “There’s no debating it, and anybody who argues the other way is just plain wrong.”
But even that isn’t satisfactory to some of the proposal’s critics, who argue that such broad consent is actually too vague to mean anything. “The idea that this informed consent will be in any way meaningful I think is illusory,” Clayton said.
There have been hints that the government could help pay for institutions to set up the infrastructure necessary to receive and keep track of consent. Major research centers would likely be able to manage either way, but without financial support, smaller clinics that don’t focus on research might decide to stop collecting samples altogether. That would shrink and skew the universe of specimens available for research, critics say.
Researchers could also request waivers from the consent requirements under the proposed rule if they showed that their studies could not be completed without non-consented specimens.
Skeptics of the proposal include not only many researchers but also the medical device industry and nonprofit groups such as the March of Dimes, which has said it is concerned the requirement of consent could limit the availability of samples used for newborn screening research. The HHS secretary’s own advisory committee on human research also worried the proposal could “substantially hamper scientific progress.”
The clock is ticking. HHS released a revised version of the proposal in September, four years after the initial announcement. The public comment period ended 90 days later, though the lobbying push continues. The end of the Obama administration in January is the most obvious deadline for the department to finalize the rules, but some actually believe that the administration could withdraw the proposal altogether after the backlash. Everyone is waiting and wondering.
“If it goes into effect, it’s really going to be problematic for a lot of people,” said Retzlaff of AACR, which has been one of the biggest supporters of Biden’s moonshot initiative. “I think it is going to be a big, big issue.”