What started out as a quiet, yet firm, request for data transparency several years ago has now become a cacophonous call-to-action. In fact, according to the International Committee of Medical Journal Editors (ICMJE), who wrote an editorial discussing the issue in last week’s New England Journal of Medicine (NEJM), “many funders around the world—foundations, government agencies, and industry—now mandate data sharing. “
Things have evolved a great deal since November 2014, when the National Institutes of Health (NIH) proposed forcing pharma companies to put all data from clinical trials—successful and otherwise—online. This so-called “transparency initiative” was supported by more than 80,000 individuals and 500 organizations with a vested interest in clinical trial data transparency. Even earlier, in 2007, a U.S. law came into effect making failure to register clinical trials illegal.
Fast-forward to January 2016
By January 2016, the conversation about the value of clinical trial data registration and sharing had shifted dramatically. At his last State of the Union address, President Obama said, “Let’s make America the country that cures cancer once and for all,” and then Vice President Joe Biden outlined the plan for the ambitious “Cancer MoonShot 2020” initiative, spearheaded by Dr. Patrick Soon-Shiong, the well-known biotech entrepreneur, who heads up NantWorks in Los Angeles.
The goal of Cancer MoonShot 2020—to design and implement clinical trials of new immunotherapies for 20 tumor types in up to 20,000 patients by 2020—hinges on the implementation of effective big data analysis, a topic we covered in depth last week.
ICJME: “Data-sharing guidelines are needed”
The ICJME published a proposal in NEJM last week laying out a proposed framework for data-sharing.
In an editorial, Dr. Howard Bauchner, Editor-in-Chief of the Journal of the American Medical Association (JAMA), and other ICJME committee members introduced proposed guidelines with a bold statement reflecting a core belief: "The International Committee of Medical Journal Editors (ICMJE) believes that there is an ethical obligation to responsibly share data generated by interventional clinical trials because participants have put themselves at risk," the wrote.
They also provided a working definition of “clinical trial,” which indirectly posits what type of data should be shared and available to the larger research community. According to Bauchner et al, “The ICMJE defines a clinical trial as any research project that prospectively assigns people or a group of people to an intervention, with or without concurrent comparison or control groups, to study the cause-and-effect relationship between a health-related intervention and a health outcome. “
The power of individual de-identified patient data
The broad outline of the ICJME’s proposal includes a requirement that researcher-authors share individual de-identified patient data (IDP) with others, including tables, figures, appendices and other supplementary materials no later than six months post-publication.
In addition, the proposal would require authors to include a plan for data-sharing as a core part of clinical-trial registration. The plan should include specific information about where the data will be stored and how it can be accessed by other researchers.
No data-sharing plan, no publication
Although the ICJME has published its proposal with the understanding that there will be a period of feedback prior to implementation, according to the plan, eventually an author who does not have a documented data-sharing plan will be unable to publish clinical trial results in an ICJME journal.
What of the implications of this? Essentially, no data-sharing plan equals losing access to important publication opportunities and losing professional and research-related traction. It means that journals, such as NEJM, JAMA, the British Medical Journal, the Lancer, Annals of Internal Medicine, the Chinese Medical Journal, the New Zealand Medical Journal, the Ethiopian Journal of Health Sciences—and many other top-tier, high-impact factor, peer-reviewed clinical journals—will be unavailable to them.
In other words, if you don’t sign up for data-sharing and support the broader big-data initiative you risk not having your work published, recognized or funded.
Concerns about research parasites
In a separate editorial in the same issue of NEJM, Drs. Dan Longo and Jeffrey Drazen weighed in on the data-sharing issue. Although they endorse data-sharing, Longo and Drazen also recognize the specter of what could happen in the worst-case scenario if data-sharing is not properly handled.
"The aerial view of the concept of data sharing is beautiful," they wrote. "What could be better than having high-quality information carefully reexamined for the possibility that new nuggets of useful data are lying there, previously unseen?
"The potential for leveraging existing results for even more benefit pays appropriate increased tribute to the patients who put themselves at risk to generate the data… However, many of us who have actually conducted clinical research, managed clinical studies and data collection and analysis, and curated data sets have concerns about the details. The first concern is that someone not involved in the generation and collection of the data may not understand the choices made in defining the parameters…”
In addition, Longo and Drazen speak to a very prominent concern—the concern about ‘research parasites.’ Research parasites are those who have nothing to do with clinical trial study design or execution, but come along and use other groups’ work to further their own goals, without providing proper attribution. In some cases, they may even subvert or compromise the work of the original researchers.
These individuals may think of themselves as ‘thought leaders’ or consider themselves too smart or clever to do the real on-the-ground work—and completely destroy the integrity of medical research.
Dialing back the paranoia
While the threat of research vampires would be very real without a well-vetted set of guidelines for data-sharing, the ICJME’s plan is focused on being ‘symbiotic,’ according to Longo and Drazen. It assumes safety checks along the way and an unprecedented level of collaboration.
In fact, this week’s issue of NEJM unabashedly comes out on the side of data-sharing. It is clearly understood that the complexity of cancer and the emerging understanding of the primacy of immunotherapy depends on large amounts of data—and that eventually that data will lead to tangible discoveries and pathways and practices that actually bring the goals of Cancer MoonShot 2020 to fruition.