Let’s liberate clinical trial data

The Gutenberg press disseminated ideas to a wider society. But in the clinical world, much information is still on "lockdown." (Wikimedia Commons)

The best things in life are free: friends, sunny days, beautiful vistas. Wouldn’t it be nice if knowledge were also free? Historically, libraries promulgated knowledge sharing because it was for the public good. We see this spirit increasingly embraced on the Internet – take the recent announcement of a collaboration between Harvard and MIT to make their courses freely available to users around the world via the edX platform.

But have we made all useful knowledge available in a way that allows for the greatest societal advancement? Not really. According to Ken Mandl, MD, MPH, director of the Intelligent Health Laboratory at the Children’s Hospital Informatics Program (CHIP), one important source of information still on lockdown is clinical trial data. In an article called, “Learning from Hackers: Open-Source Clinical Trials” published this month in Science Translational Medicine (not currently available in full text), Mandl and his coauthors call for making raw, de-identified clinical trial data free to the public.

Expanding the definition of “free” to include not just “gratis” (zero cost) but also “libre” (no restrictions), the article makes the case that releasing these data sets could revolutionize our understanding of disease. The authors note that the open-source software community has demonstrated its ability to rapidly collaborate to solve problems through the free exchange of information. Success, they write, “is driven by the developers’ active engagement with users in the software creation and testing phases, and the rapid filling of gaps in functionality by a decentralized cadre of developers, a process supported by online communities formed around repositories of code.”

What is so compelling about liberated information? Just as the printing press and increased literacy allowed societies to tap the creative power of the many, making clinical trial data available to new analysts will introduce a new population of problem-solvers to answered medical questions. It’s likely these new analysts will be motivated for the same reasons they are in software: more data provide more opportunity to make discoveries, develop careers and gain clout in the field.

“Learning from Hackers” cites cases where releasing clinical data to a wider group of researchers led to rapid advances in understanding of medical conditions. For example, the number of publications using the Framingham Heart Study data increased by 19 percent in the four years after data were released to a greater audience of researchers. And, as a market analyst in a technology transfer office, I think crowd-sourcing clinical data could help the pharmaceutical industry replenish its diminishing pipeline. How we’re doing things now – proprietary datasets, redundant research efforts — just isn’t cost-effective.

At least one group of patients ought to have a strong opinion about sharing clinical knowledge: those with rare diseases. At the recent Bio-IT World conference, Alex Sherman, MSc, from Mass General spoke of setting up the IT infrastructure for a centralized data repository for patients with Lou Gehrig’s disease, and advocated for doing the same for other diseases, especially rare ones. Conditions with the most data, he argued, will be in the best position to entice pharmaceutical companies to develop new therapies.

Pharma and biotech will want to invest significant capital only where they’re most likely to meet the required clinical end points and get FDA approval. Rather than walking blindfolded on a tightrope over crocodile-infested waters, they could have a bridge – built on centralized, freely accessible data on the mechanisms and pathology of the disease and the ability of biomarkers and other measures to assess whether treatments are working.

That’s not to say there won’t be problems to solve. We still lack universal standards and quality controls that would allow researchers to build a solid data bank for meta-studies. De-identified data aren’t entirely protected from malevolent attacks on patient privacy. And, as Sherman noted, stakeholders often have incentives contrary to information-sharing: Researchers incentivized to “publish or perish” fear losing their competitive edge, while industry is motivated to keep some information private to avoid possible scandals or legal concerns.

These hurdles need to be addressed before we’ll see widespread adoption of the “libre” mantra, but we in health care should start brainstorming about how to get us there. Mandl and others engaged in both medicine and information technology are helping pave the way through their brilliant connection to the hacker (open source) community. Cross-industry innovation can help us think outside the (pill) box.

Keeley Wray (@Market_Spy) is senior market research analyst in the Technology and Innovation Development Office at Boston Children’s Hospital.