Is ePRO more than just a questionnaire?

ePROs have the potential to provide patients with greater flexibility and input in their participation in clinical trials

Lindsay Hughes, Ph.D., M.S. – eCOA Principal Scientific Advisor at Clario

Patient-Reported Outcomes (PROs) are Clinical Outcome Assessments (COA) collected directly from the patient, based on their own interpretation of symptoms, quality of life, or other health-related outcome. The availability of modern technology, including smartphones and tablets in the home, has enabled electronic Patient Reported Outcomes (ePROs) to provide patients with greater flexibility and input in their participation in clinical trials.

So, is an ePRO just a questionnaire? While it may appear that way, these assessments vary in complexity and can do much more than a traditional survey. Within clinical trials, ePROs have the potential to not only capture patient compliance but also positively impact compliance by serving as a behavioral trigger. ePRO assessments should be carefully designed to drive high compliance and promote data quality. In this article, we’ll discuss the science behind the questionnaire, how it delivers value to sponsors and the difference it makes to the patient experience.

“Within clinical studies, ePROs should be carefully designed to drive high compliance and promote data quality”

Lindsay Hughes, PhD, MS – eCOA Principal Scientific Advisor

Adhering to protocol design through ePRO

With ePROs, the study workflow can be programmed to increase study compliance. For example, best practice states that PROs should be completed prior to other study activities to reduce any bias due to conversations during a study visit. While site staff and patients are trained on the Schedule of Assessments and order of deployment, ePRO can provide an additional layer of control by enforcing the order in which assessments must be completed before additional assessments become available.

Obtaining greater insights through ePRO

Collecting patient-reported outcomes electronically gives us access to useful data that’s not limited to the patients’ answers. Connected devices, for example, allow us to gather context. Metadata contains essential information to reference if an assessment is flagged for further investigation, such as if fraud is suspected. This data gives us more control over study management by confirming details surrounding the completion of the assessment, such as when and where it was completed. It also allows us to monitor patient progress; if progress is not optimal, alerts can be sent to patients and or sites.

Incorporating connected devices enables performance outcome measures (PerfO) to be collected. This powerful combination of ePRO plus connected devices is a real game-changer. Let’s take mobility loss as an example. Mitigating mobility loss is a major priority in health care since any change in mobility is a marker for change in health. Therefore, being able to monitor mobility through, for example, activity trackers, while placing no extra burden on the patient, is a great example of how ePRO is providing sponsors with the opportunity to collect valuable additional measures.

Collecting robust data in hybrid and decentralized trials

ePROs give us the potential to collect robust data through hybrid and decentralized trials. If we consider decentralization in the home, smartphones and tablets have given sponsors the ability to collect PRO and ObsRO (such as by giving the device to a caregiver who can fill out questionnaires of their observations). Virtual video visits allow for Clinician-reported outcomes (ClinRO) in the home.

Achieving patient compliance through ePRO

Patients using ePRO demonstrate significantly higher protocol compliance — as high as 94% compared to 11% with paper.² In many studies, compliance is two-fold. For example, when a patient receives an alert to fill out their eDiary and does so, they are compliant in their reporting. Filling out the e-diary then triggers the intervention associated with the PRO, such as taking a medication. When patients fill out their assessment, it creates a behavioral trigger that reminds them to take their pill. Filling out the assessment signifies compliance with taking the medication, and cases such as these reinforce overall study adherence.

Supporting data quality control through ePRO

When we design studies using our validated methods and many rounds of usability testing, there’s a lot less variability on the patient’s end. For example, offering answers in scroll wheel or radio button format allows patients to choose only what is listed, preventing them from writing in a new option. The automatic nature of data entry also helps control data quality. While paper requires manual data entry, data quality checks for inter-rater reliability, and maintenance of original files, ePRO incorporates front-end data quality control and reduces the opportunity for error. With eCOA, data goes into the system in a much more controlled fashion, which results in better quality data and less burden on the site.

Minimizing clinician and self-reported bias through ePRO

Study design order of operations minimizes bias. Let’s consider how clinicians might introduce bias and how ePROs can mitigate this risk. Sponsors can radically reduce the opportunity for clinician bias to influence the patient’s answers when we require PROs to be completed before the site visit. Completing assessments in private allows patients to more honestly report potentially sensitive information – avoiding the need to discuss it in-person with their clinician. We also minimize other biases that influence self-reported data like social desirability, in which patients answer based on what they believe is the “right answer.” With eCOA, answers go directly from the patient into the system without external influence, thus collecting more honest responses.

In summary, while questionnaires need to appear simple and be easy to complete, the science and operational expertise behind delivering them is complex. ePROs should be carefully designed to capture high-quality, rich data while also considering the patient experience.

References

¹ https://www.fda.gov/regulatory-information/search-fda-guidance-documents/electronic-source-data-clinical-investigations

² Stone, A. A., Shiffman, S., Schwartz, J.E., Broderick, J.E., Hufford, M.R. (2002). Patient non-compliance with paper diaries. British Medical Journal, 324, 1193 – 1194.