The promise and perils of app-based clinical trials

Researchers use smartphones to study breast cancer, Parkinson's, and other diseases

Researchers and study participants using Apple's recently released ResearchKit platform say smartphone-based clinical studies are potentially revolutionary, but some worry the approach brings new challenges, Ron Winslow reports for the Wall Street Journal.

Apple's ResearchKit is an open-source software framework that allows medical researchers to collect medical data through consenting iPhone users. It was announced in March, and so far five disease-related apps are using the software to funnel clinical data securely to researchers for further study.

How it works

One app, called mPower, which was developed by researchers at the University of Rochester, is designed to study Parkinson's disease. Ray Dorsey, the lead researcher on the project, says using a phone to collect data provides new insights. "Patients say symptoms fluctuate over the course of a day, but we've never had a way of measuring that," he says.

Is Apple already changing health care? Thousands sign up for trials through ResearchKit

Every day, users of mPower are asked to perform a finger-tapping test to track the speed of their movements, evaluate their balance with the phone's accelerometer, record the strength of their voice, and test their memory. In addition, the phone's sensors measure a user's hours of sleep, minutes of exercise, and overall steps taken.

Steven DeMello, who has Parkinson's and uses mPower, says the app is not just about helping researchers. "If I know more about my condition, the better and smarter I will be in managing my own care," he says.

Stanley Shaw—co-director of the Center for Assessment Technology and Continuous Health at Massachusetts General Hospital, which has developed an app to track diabetes patients—agrees that ResearchKit may change what it means to be a study participant. "The phone can be a powerful way to engage participants and make participating in a clinical study more of a two-way information exchange," he says.

Related: Will Apple Watch revolutionize health care? Three reasons to be cautious.

New research opportunities

ResearchKit also makes launching certain types of clinical studies drastically easier. Since the apps were launched on March 9, they have been downloaded about 60,000 times. Eric Topol, director of the Scripps Translational Science Institute, says, "This is a whole new way of going forward with medical research that makes it quick, scalable, and efficient."

For instance, Kathryn Schmitz, an epidemiologist at University of Pennsylvania, says it took her team three years and 60,000 written solicitations to recruit 351 patients for a study on the impact of exercise on breast cancer survivors. In March, her team released a ResearchKit app called Share the Journey, which examines the same subject with less stringent enrollment criteria. In just one month, 2,000 patients had signed up.

New challenges

However, some worry that ResearchKit-based studies present new challenges. For instance, only those who can afford an iPhone are able to participate. Art Caplan, renowned bioethicist and founding director of NYU Langone Medical Center's population health department, cautions that smartphone-based studies are "not necessarily representative of the problems of the poor, or the health habits of the uninsured or underemployed."

Apple notes that it plans to open source ResearchKit, which means it could eventually be available on lower-cost devices.

Privacy is also a concern. Apple stresses that users have complete control over who they share their health data with and that the data are never shared with Apple directly. Even so, Topol notes a single, significant privacy breach could be a "serious hit" to the use of smartphones for clinical research (Winslow, Wall Street Journal, 4/13).

The takeaway: Researchers and study participants using Apple's recently released ResearchKit platform say smartphone-based clinical studies are potentially revolutionary, but some worry the approach brings new challenges.


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