COMMENTARY

A New Mobile Model of Medical Research

Disclosures

March 18, 2015

An Easier and Cost-Effective Medical Research Model?

While most Apple aficionados were looking for more information about the company's ballyhooed new watch last week, an announcement of a new platform for medical research was unveiled by surprise. By shifting data collection to consumers in their real world, on a massive scale, this potentially represents the biggest change since the origins of clinical trials—such as with citrus fruits for scurvy in 1747 or streptomycin for tuberculosis in 1946.

Until now, nearly all medical research has been conducted in clinics and hospitals. It has required a willing individual—typically called a "subject"—to provide written informed consent after a nurse or doctor reviewed the research project details. A form, often comprising several pages, had to be signed to acknowledge the risks and potential benefits.

The measurements taken, including vital signs and labs, represented one-off data points in the unnatural, contrived environment of a doctor's office or hospital setting. This is a laborious process that takes considerable time and dedicated personnel, resulting in the impaired ability to recruit individuals to participate in research projects. A major sequela has been that clinical trials are remarkably expensive and painstakingly slow. For example, when my colleagues and I performed a large heart attack trial that enrolled over 41,000 patients in the early 1990s, it took nearly 3 years and cost more than $50 million.[1] Today such a trial would probably take even longer and cost at least four to five times as much.

Enter a mobile medical research platform such as Apple's ResearchKit, with the ability to recruit patients from the public via smartphones. Some have called this a "clinical trial in your pocket." Not only does this immediately access millions of individuals (iPhones were purchased by more than 70 million individuals in the fourth quarter of 2014), but the consent process is very similar to downloading and accepting an app.

Moreover, instead of the data being entered by a nurse or research coordinator, the patient enters his or her own information or generates the data via a sensor or app. And rather than the artificial measurement obtained in a medical center, the context is the individual's real world—whether it be at work, home, or on the go. Because smartphone users look at their phones an average of 100-150 times per day, we move from one-off data capture to the potential of "all in" engagement. For example, the University of Rochester app for Parkinson disease[2] tracks tremors, voice, and gait. Participating patients would conceivably be able to have previously unobtainable information, now in real-time, about the effectiveness of their treatment throughout the day with these multiple metrics, all assayed via their smartphone.

Patient Excitement

In the first 24 hours after the announcement, more than 11,000 patients enrolled in Stanford University's cardiovascular program and 3500 joined Mount Sinai's asthma research initiative.[3] Apple's CEO Tim Cook touted the ResearchKit platform as "perhaps the most profound change and positive impact" regarding the company's new offerings that day, but it could also be applied to the conduct of medical research.

Although this is not the first time that mobile apps have been used to conduct clinical studies, the immediate global reach of Apple to millions of people, representing all ancestries with all of the common diseases of man, is formidable. However, there are certainly some drawbacks to the platform that are noteworthy. There is a clear-cut skewing of the sample of patients who could participate: those who can afford iPhones (and only models 5 and 6, which are compatible with the new ResearchKit apps). Although the platform has been characterized as "open source," it is restricted to the Apple iOS software despite the fact that Android is the one most used globally. Furthermore, it is not "open" to all researchers but only to those selected by Apple to participate. And with the mobile device openness to patients, there is no assurance of the veracity of the data that are being provided. Or that a real patient is participating.

While such a mobile research platform may not be conceived as suitable across all medical conditions, there is the opportunity going forward to provide hardware to participants that will broaden the capabilities. For example, a microfluidic attachment to a smartphone could be capable of assaying many labs or components of the physical exam, or wearable sensors could be supplied to willing patients. Indeed, many consumer surveys show that over 80% of individuals are happy to participate in such research and share their results, provided that their anonymity is maintained.

Until now, Apple has done well to protect the privacy of consumers' information. Going forward, this will be one of the most important determinants of the success of such a platform. Another is the durable commitment of individuals who initially sign on to stay with the research initiative. This will be dependent on the user-friendliness of the apps, the utility of the information being provided to individuals, and whether researchers will be able to incentivize, gamify, and organize managed competitions to allure and sustain consumers' interest.

Notwithstanding the concerns of privacy and many other caveats outlined here, the new mobile medical research platform may turn out to be the most important advance in the history of clinical research. Capturing patient-generated real-world data at scale for minimal cost represents a complete rebooting of how clinical research is done today. This new model, which needs to be fully validated and expanded upon, builds on the extraordinary potential to tap into the most unused entity in healthcare—the patient. This "bottoms-up" approach also recognizes the power of smartphones, our digital infrastructure, and the democratization of medical information. Let a new, progressive way of medical research begin.

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