By Chad Gwaltney, Ph.D., chief scientist and regulatory advisor, endpoints, ERT
Interest in the bring your own device (BYOD) approach to capturing electronic patient-reported outcomes (ePROs) is increasing dramatically among sponsors. With BYOD, patients use their own devices (smartphones, tablets, personal computers) to complete ePRO diary assessments, rather than separate, dedicated devices provided to them by the sponsor. BYOD approaches have notable strengths and can significantly reduce the time, effort, and costs associated with dedicated devices. However, while BYOD is emerging as a reliable and appropriate option for post-approval studies — where realworld data are critical, relatively large sample sizes may be used, and patients may be less likely to regularly visit a clinical site — there are a number of questions that sponsors should consider before using it in pre-approval (phases 2-3) research.
WHAT IMPACT WILL BYOD HAVE ON PATIENT RECRUITMENT? WILL IT LIMIT RECRUITMENT TO ONLY THOSE WHO OWN THESE PERSONAL DEVICES?
The seeming ubiquity of smartphones is a big reason for the appeal of BYOD. However, although smartphone penetration is increasing, many individuals still do not own them, and ownership is likely biased in ways that will impact a clinical trial (age, region, socioeconomic status).
WILL MIXING MODALITIES IN A SINGLE TRIAL AFFECT OUTCOMES?
Sponsors need to consider if/how enabling patients to use their own devices could affect their responses to PRO items. Also, there are potential scientific and regulatory concerns with the multiple interface differences that are certain to be present in a BYOD approach (e.g., device size, method of inputting responses, other functions available to user). BYOD approaches inherently introduce mixed modes of administration to a trial, and this could introduce unanticipated response biases. The nature of the modality used by patients may not be random; it may be tied to other factors that could influence responding. For example, in Web-based BYOD studies, some patients may be able to respond in any location at any time using a smartphone, while others using a desktop computer in their home may be limited in the timing and location of their entries. A unique feature of BYOD is that variability due to mixing modalities may not only cause response differences across patients using different devices, but also within the same patient, if they use different devices to respond to items in a single study.
WILL COMPLIANCE WITH THE ePRO PROTOCOL BE ADVERSELY IMPACTED BY BYOD APPROACHES?
Increased compliance is one of the key reasons why ePRO is essential in studies implementing patient diaries. The high rates of compliance with traditional ePRO (> 90 percent typically) are due, in part, to the alarm functionality that is included in most dedicated eDiary devices, typically via an audible alarm that cannot be muted. In a BYOD scenario that utilizes smartphones, ePRO apps can use reminders, but this alarm functionality can be limited by user preferences or through the operating system itself.
DO PATIENTS INTERPRET A PRO INSTRUMENT IMPLEMENTED VIA BYOD THE SAME WAY AS ITS INITIAL, PAPER-AND-PENCIL FORMAT?
The FDA’s PRO Guidance states, “When a PRO instrument is modified, sponsors generally should provide evidence to confirm the new instrument’s adequacy.” This includes changing an instrument from a paper to electronic format. The challenge with BYOD is that there are a number of possible electronic platforms that could be used by patients, each with different screen sizes and other interface features. It is difficult at the outset of a trial to ensure that patients will interpret the electronic administration the same way across all potential device options. This could lead to scientific and regulatory concerns about the integrity of the data collected through BYOD approaches.
A BYOD approach to ePRO data collection offers significant benefits in clinical research. Its use in post-approval studies — which have different logistic characteristics and scientific goals — can provide important data to sponsors in a more cost-effective manner than dedicated, handheld eDiaries. However, additional research is needed to demonstrate that patients comply with and interpret PRO instruments in the same way across different types of personal devices.