Volunteer for a clinical trial and your body will be poked, prodded, scanned, and analyzed. But you’re unlikely to get any of the results.
A small but influential band of activists has been pushing hard to change that — and they’re starting to get traction.
The research establishment has long opposed giving volunteers access to their data, even though that’s supposed to be part of the arrangement. Some worry that it’s too easy for laypeople to misinterpret test results, while others maintain that it’s a waste of resources to organize data for individual review.
But in a striking shift, two global drug makers recently agreed to let clinical trial participants review data generated by their own bodies. In July, the federal office that oversees research on human subjects recommended that data sharing be the rule, not the exception.
And many of the million people expected to sign up for the big federal research project known as the Precision Medicine Initiative will get access to everything scientists learn about them, including DNA readouts and data on the billions of bacteria living in their guts.
“It’s a power shift,” said Dr. Eric Topol, a cardiologist and director of the Scripps Translational Science Institute.
But it remains to be seen how the average volunteer will make use of the information — or if they will even want to see it.
In two clinical trials for diabetes in 2013, Pfizer enabled US volunteers to set up online accounts where they could download data such as heart rhythm charts, lab reports, and vital sign measurements after the study’s end. Less than 15 percent did so.
“What we found is patients expressing that they wanted access, but not a high volume of clicks for access,” said Craig Lipset, head of clinical innovation at Pfizer. “I’m not sure they felt there were enough things to do with the data that were truly meaningful.”
Lipset suspected the company can boost interest by tweaking when and how volunteers get the data, and what they learn.
Janssen Pharmaceutical is now trying out something similar with a study of an experimental drug for rheumatoid arthritis. Volunteers can get real-time lab data and upload it into their own electronic medical records. At participating study sites, 24 percent of volunteers opted in — and 38 percent of them used the system regularly.
It’s a minority, admitted Dr. Andreas Koester, a vice president of research and development operations at Janssen. But he’s not deterred: The aim was to get feedback on making such a system easier to use.
“The message from industry is: ‘This is a black box. You give us everything, and we can’t tell you anything, and that’s just the way it is,’” Koester said. “This is not sustainable.”
A matter of life and death
Proponents make the case that returning data to volunteers could improve public trust in research and increase participation in studies. It might also reduce duplicate medical tests, help participants manage their own health, and encourage more data sharing between scientists.
Jack Whelan, 67, has less abstract goals in mind: He considers access to his own data crucial to his survival.
In the past eight years, he has joined seven clinical trials of drugs for his blood cancer. This month he’s starting his eighth, for a new diagnosis of advanced prostate cancer.
Right from the start of his first trial, Whelan told the study doctor: I want that data you’re collecting. He plugged it into a spreadsheet, and tracked his body’s response to each experimental drug. Although Whelan doesn’t have a background in medicine, as a former research analyst, he’s good with data.
He quickly learned to figure out whether an experimental drug was effective or not, just by observing changes in creatinine, bilirubin, and other factors in his blood. Participants in clinical trials aren’t supposed to know whether a drug is working. But the way Whelan saw it, he couldn’t afford not to know. “If after two or three [drug] infusions, I could see that it wasn’t going the right way, I’d say: ‘I want to stop the trial, I want to get off this bus,’” he said.
Such stories make researchers wary; it can hurt the reliability of clinical trials if volunteers drop out when they think a drug isn’t working. That matters to Whelan, too, but he’s understandably more worried about his own life. He’s convinced that analyzing his data is what’s kept him alive so long with such a deadly disease.
To help others do the same, Whelan and collaborators built an app and information service for patients to manage their health records (including clinical trial data, if they can get it). The team sold it to the clinical trial software company ERT, which is integrating it with its own system. (Whelan, who lives in Andover, Mass., also gets speaking fees from various drug companies as a patient representative.)
Others argue that giving volunteers their data is just the right thing to do. “You’re asking me to spend time, to literally devote my body and possibly compromise my health to help advance a therapy, and yet I tend to be the invisible partner,” said Laura Kolaczkowski, 62, a patient advocate in Ohio who is now the co-principal investigator for iConquerMS, which lets people with multiple sclerosis share their data for research.
Kolaczkowski volunteered for a study of baclofen, a muscle relaxant, but was never even told whether the trial succeeded or failed. “At the end of the day, when that study concludes,” she said, “the people who’ve participated are owed that by the sponsors.”
Is a little knowledge dangerous?
In the US, federal rules don’t forbid giving volunteers their test results, as long as the tests are well-established as legitimate.
In fact, the Secretary’s Advisory Committee on Human Research Protections at the Department of Health and Human Services recently recommended a “rebuttable presumption” in favor of returning individual results after a study is over, at least for conventional tests like viral loads for hepatitis — or even IQ. The information doesn’t need to be medically important. Pure intellectual curiosity is reason enough, the guidance says.
Access to data is usually written into study protocols and spelled out in the paperwork that participants sign. It just typically doesn’t happen.
Only 9 percent of research volunteers surveyed in 2014 got even so much as a summary of the study results. Just 4 percent found out whether they got an active drug or a placebo, according to the survey of 1,072 volunteers by the nonprofit Center for Information and Study on Clinical Research Participation.
Some scientists — and even some study volunteers — support keeping clinical trial participants partly or largely in the dark.
“I used to think patients need all data, but that’s not what I believe nowadays,” said breast cancer survivor Deborah Collyar, president of Patient Advocates in Research and a member of many research advisory committees. “Data is not knowledge. You need to understand the data, and then be able to interpret it.”
Without proper context, she said, the information isn’t useful.
Another concern: Giving volunteers their data sets up the expectation that they’ll get something valuable for participating in a clinical trial, rather than agreeing to do it purely out of altruism, said Dr. Ellen Wright Clayton, a professor at Vanderbilt University’s Center for Biomedical Ethics and Society. The practice blurs the lines between research and health care, she said. It’s a poor use of limited research funds. And volunteers might misinterpret test results and seek unnecessary treatments or further tests.
So Clayton argues for strict limits on data return: Research participants should get only results that uncover a potentially serious condition, like a suspicious shadow on an MRI.
A narrow focus on curing disease
On the other end of the spectrum, some researchers are trying to make it easier for study participants to get broad access to their data — and then share it, if they’d like, with scientists.
One recent project lets families with rare undiagnosed genetic diseases join a research study through the University of Washington Center for Mendelian Genomics. They get their genomes sequenced and receive the raw data — along with a report and tools to explore it — for just $825, instead of the $5,000 to $7,000 it could cost through a doctor. If they choose, they can publicly share that data, along with their own medical records.
Putting data directly into people’s hands is step toward reengineering the system. The goal is a research process that’s faster, more equitable, and more squarely aimed at curing disease, said Jason Bobe, an associate professor of genetics at the Icahn School of Medicine at Mount Sinai in New York City.
Academic researchers, companies, and nonprofits have incentives to keep data locked up, whether to protect intellectual property, grants, or publications, Bobe pointed out. Study volunteers are mostly in it just to improve their own health or further medical science.
“They don’t care as much about tenure, stock price, those kinds of things,” Bobe said. “They care about outcomes — their health and the health of their family.”
Correction: A previous version of this story misstated the first name of Ellen Wright Clayton of the Center for Biomedical Ethics and Society.