Clinical Research: Phase 1 - Phase 4

Mobile clinical research. There’s an app for that.

Visit the iTunes App Store or Google Play and you will find the smartphone app PoopMD+: A New Parent’s Guide to Baby Poop and Pediatric Liver Disease Study from Johns Hopkins.

PoopMD+ is an imaging and clinical survey-based app developed to help screen for a rare but life-threatening liver disorder in babies and collect feedback on what parents do with that information. It is the first of what be many existing mobile health (mHealth) apps initially developed for consumers that are later transformed into mobile clinical research study apps. To date, PoopMD+ is also only the second app to use Apple’s fledgling, yet promising open source ResearchKit to target a rare childhood disease.

There are now more than 150,000 mobile health apps available to hundreds of millions current smartphone and tablet users around the world. Yet, this impressive number still includes only a few hundred, clinical trial-related apps. Most of those are largely search/info-only mHealth apps aimed at patients or caregivers trying to locate clinical trials for specific diseases or clinical trials occurring within specific medical institutions.

For several years, the U.S. Food & Drug Administration (FDA) has actively encouraged the use of mHealth technology like smartphone apps to enable faster, more cost-effective clinical trials that help get better drugs to patients, sooner. Still, the FDA and an array of clinical trial research community interests continue to wrestle with standards and how best incorporate fast-moving digital innovation without negatively impacting the expected quality, efficacy and privacy protections associated with and expected of randomized clinical trial research. Over the past year, the FDA has collected feedback from some 40 leading organizations and individuals about “Using Technologies and Innovative Methods to Conduct FDA-Regulated Clinical Investigations of Investigational Drugs,” but not reported out any additional guidance.

Last year, a less open clinical research mobile trial model that does not rely on Apple or Google being developed at the University of California San Francisco (UCSF) received $9.75 million from the U.S. National Institutes for Health (NIH). The money is earmarked to help UCSF develop the platform like ResearchKit that can be used to conduct digital health studies. This platform, called Health ePeople, is supposed to provide researchers with access to a large group of volunteers that have agreed to participate in research and the infrastructure to collect participant health data through mobile and wireless technologies.

For now, though, existing FDA guidance around “Electronic Source Data in Clinical Investigations” and its more recent 2015 “Mobile Medical Applications” guidance are what is fueling and limiting the development of clinical trial-based mHealth apps.

That guidance says “mobile apps used for data collection in clinical studies (such as electronic Patient Reported Outcomes [ePRO] apps) are not considered on its own a mobile medical app.  However, manufacturers and users of this type of mobile app should see FDA’s draft guidance related to use of computers in clinical trials.”

The Association of Clinical Research Professionals (ACRP) and others remain concerned about exactly where the limits are and are not when it comes to mHealth applications, especially those that interconnect data from randomized clinical research, electronic health records and ongoing patient care. They have requested more direction from the FDA.

As discussions continue, mHealth apps for clinical trial awareness and patient recruitment for specific studies continue to grow in popularity.

Stanford Medicine’s MyHeart Counts app, one of the first five apps to use the ResearchKit framework last year, had a reported 30,000 people enroll in the mHealth app’s first month of availability. The ongoing clinical study, being done in partnership with the American Heart Association and Oxford University, aims to become the largest ongoing study of measured physical activity and cardiovascular health to date.

And it appears there is more to come. Over the past year, Apple accelerated the building of its health-focused team, including doctors, researchers and fitness experts. In October, Apple hired away Duke Medicine’s Dr. Ricky Bloomfield, Director of Mobile Technology Strategy and Assistant Professor in Internal Medicine & Pediatrics. Dr. Bloomfield helped develop Duke’s mobile video-supported Autism & Beyond study app and was one of the earliest proponents of both HealthKit and ResearchKit.