“PROactively” Pursuing mHealth in Clinical Trials

The concept of patient-reported outcomes (PROs) in #clinicaltrials is certainly not new, but advances in mobile tech are helping to position PROs as a game-changing factor. In just the past few years, a whole new mHealth industry has arisen, spurring the development and adoption of wearable and sensor-based devices loaded with applications that monitor and transmit patient data in real time. Now, instead of relying on patients (or sometimes caregivers) filling out questionnaires to collect one-time assessments of drug efficacy or tolerability, investigators and other trial personnel can employ #mHealth technologies to monitor every second of a patient’s biochemical and behavioral response, potentially yielding a more accurate and reliable patient report.

Investigators and trial sponsors increasingly realize that clinical trial success will eventually go beyond standard measurements of drug safety and efficacy, and will need to include more objective assessments of the patient experience. Already we see usage of electronic clinical outcome assessments (eCOAs), which include patient-, physician-, and observer-rated outcomes, growing at about 15% per year. Unsurprisingly, pharma companies are scurrying to figure out how to use evolving tech products to gather massive amounts of real-world #patientdata, while adhering to the requisite structure and rigor of a #clinicaltrial.

The value of continuous patient monitoring is well illustrated, as one example, by ambulatory blood pressure monitoring (ABPM). These systems allow cardiologists to identify individuals with “masked” hypertension as well as so-called “dippers,” in whom blood pressure drops at night. Such fluctuations generally can’t be measured in the clinic, but are now readily obtainable through the use of portable, wearable ABPM technologies.

However, despite their transformative ramifications for trial design, reporting, and regulatory submissions, mHealth-enabled PROs are not without their potential pitfalls. Human nature being what it is, the possibility remains that use of mHealth devices will not prevent certain patients from self-monitoring and changing behaviors that may impact study endpoints. Even when patients are conscientious about wearing their devices and behaving as they should, some PROs may yield misleading or incomplete information. Consider when fatigue is a measurable adverse event in a clinical trial of an anticancer medication; in addition to being a common side effect, fatigue can sometimes be a sign of depression, or a cause of non-adherence to a therapeutic regimen. Consequently, designers of mHealth apps and trial protocols will need to fully understand these dynamics to enable a level of accuracy that will pass regulatory muster.

Additionally, designers will need to grapple with the question of standardization, as trials that offer patients a “BYOD” option – the ability to use one’s own FitBit, AppleWatch, iPhone, Android, or other hand-held device – will need to ensure that PRO data are generated, collected, and analyzed the same way across devices.

Moreover, taking outcomes measurement out of the clinic may have privacy implications, heightening the importance of HIPAA compliance. Companies operating in the mHealth space are also under the watchful eye of the FDA, which, through its Guidance for Industry on use of PROs to support labeling claims, its report on the value and use of PROs in assessing effects of medical devices, and the joint FDA/NIH Patient-Reported Outcomes Measurement Information System (PROMIS®) initiative, is shining an ever-brighter spotlight on PROs and the means to measure them.

Phesi: Staying Ahead of the PRO Curve

As clinical trial protocols become more patient-centric through the inclusion of PROs, trial sponsors and investigators will need to make the concept of patient-centricity a core value. At Phesi, with our broad and deep experience in synthesizing #bigdata and predictive analytics, we recognize the need to extend these capabilities beyond an investigator-based platform to encompass PROs as well. That will mean staying abreast of regulatory guidance/requirements as well as leveraging publicly available info on eCOAs as a means to enhance the dimensionality of clinical trials. At the same time, we will be watching the evolution of mHealth and eCOA, and our expertise in trial design and implementation will help us evaluate which companies are effectively integrating their technologies into clinical databases.

One likely result of the advent and adoption of mHealth technologies is that PROs will someday be as much a part of the clinical trial lexicon as p-values, objective response rates, and adverse event reports. We’ll be watching to see which companies lead, follow, or get sidelined.

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