By Tom Ulrich
Ed. note: This is the second in a two-part series on making clinical trial data more transparent. Click here for part 1.
To grossly oversimplify, there are two kinds of people in the world: those who want to see data from clinical trials made widely and freely available, and those who would rather have the data restricted for privacy or business reasons. And as we noted in our last post, there are valid arguments to be made on both sides.
But is there a way to balance the benefits of openness and the safety of confidentiality?
Regulators, academics and companies are already working on several possible ways of answering that question. The European Medicines Agency, for instance, is moving forward with a new policy requiring full access to clinical trial data; how they will implement it is still under discussion. The U.S. Institute of Medicine has had a working group looking into possible frameworks for open data sharing for several months now. And Johnson & Johnson recently announced a new partnership with Yale University to open the doors to their vaults of raw trial data.
Others are weighing in as well. Harvard School of Public Health policy researcher Michelle Mello, JD, PhD, and her colleagues proposed in the New England Journal of Medicine four models for making clinical trial data available, aimed at encouraging openness while mitigating risks:
- Full availability of all data
- A query model where companies or a third-party intermediary retain control of data and investigators submit questions they’d like to have answered
- A sponsor-reviewed model where companies decide what data to make available and how
- A learned intermediary model where an independent third party would evaluate and oversee data requests and access
Ellis Neufeld, MD, PhD, who runs Boston Children’s Clinical and Translational Study Unit, notes that some companies have already adopted something akin to the sponsor-reviewed model described by Mello and her collaborators by inviting select scientists to take an unfettered look at data from particular trials.
"We have coming of age a generation that is used to sharing everything about themselves openly for everyone to see."
"Companies take this seriously, and want to learn from the data they have," he says. "I’ve collaborated with a couple of companies on review of their study safety and post-approval surveillance data, and they’ve been very forthcoming in allowing us to see and analyze all of the data they have. "
An issue of consent
Boston Children’s Informatics Program director Isaac Kohane, MD, PhD, for one, questions whether patient privacy is as much of a barrier as it’s been made out to be and wonders if the informed consent process might serve as both a barrier and a solution.
"Patients by and large are happy to share the data from the trials they participate in. It’s a question of being more robust about obtaining patient consent," he says. "Institutions need to say up front in their consents that the data will be shared, and say it in plain English."
He also proposes an answer that balances access with patient and corporate protection. "Every publication, clinical trial or patient cohort should have the data behind it available. But it has to be done responsibly, like with dbGap," referring to a publicly available NIH database of genotype/phenotype studies. Researchers can gain total access to dbGap data as long as they have a protocol that’s been approved by an institutional review board.
"That way," he says, "you commit researchers to good behavior and create a record of responsible use, rather than opening the data up to just anyone."
A moot point?
Kohane’s colleague, ClinicalTrials.gov founder Alexa McCray, PhD, agrees that more openness is needed, but also believes the question will sort itself out over the next 10 to 15 years, regardless of the solutions attempted today.
"We have coming of age a generation that is used to sharing everything about themselves openly for everyone to see," she says. "And patients with life-threatening disease and the families of children with chronic illness are often quite willing to make any information available for the purpose of solving disease.
"You’ll still need controls and regulatory oversight," she adds, "but just as data is becoming more available in other areas of science and of our lives, making trial data open is going to get a whole lot easier."