Traditionally, the use of patient-reported outcome (PRO) data has been an adjunct to primary data when it comes to clinical trials; however, in the current environment, reliance on PRO models has gained steam. As the FDA and research community continue to evolve in terms of how and what data gets captured during the COVID-19 pandemic, one of the areas coming to the forefront is data collection using electronic patient-reported outcome (ePRO) tools.
The FDA defines a PRO as “any report of the status of a patient’s health condition that comes directly from the patient, without interpretation of the patient’s response by a clinician or anyone else.” 1 In general, data from a PRO can be used to predict trends in a population or identify complications or difficulties associated with a particular study protocol or its procedures. Up to the point of the current pandemic, researchers typically utilized PROs to gather secondary endpoints or support a primary endpoint with additional real-world experience. However, we have managed several studies that have used these (including ePROs) as primary outcome measures as well.
Advantages of ePROs, Even Post-COVID-19
In these unprecedented times, when gathering data remotely is the method of choice, it’s worth making use of ePRO data in your clinical studies to help maintain the integrity of your clinical trials. It’s also important to keep in mind that, even beyond the context of COVID-19, ePROs can continue to be a valuable adjunct data collection tool for trials seeking to capture information about the patient experience. ePROs offer a number of potential advantages over paper-based PROs, including:
- Accuracy. ePROs enable input validation, which helps ensure that patients make valid and complete entries.
- Patient Safety. With certain ePRO systems, data is monitored, and messages can be sent directly to patients or clinicians if their responses meet a pre-determined critical level.
- Integrity. Electronic time stamps indicate when entries are made, which helps avoid the so-called “parking lot syndrome” where patients fill out the PRO right before their office visit. Generally, ePROs are also less time-consuming for patients than their paper counterparts.
- Security. Unlike paper which can be lost or damaged, ePRO data is stored on a server, with a backup.
- Compliance. ePROs have a better compliance rate than paper PROs, where compliance is a measurement of actual ePRO responses as a percentage of expected ePRO responses. Some ePRO devices can also be used to send automated reminders to patients, which help with both diary compliance and other study-related tasks.
- Speed. Assessment calculations are made in real-time, rather than requiring investigators to manually tally up responses and apply scores.
In addition, the FDA supports direct patient reporting because some treatment effects are only known to patients and may not be captured as predicted outcomes in the clinical trial. Furthermore, the National Institutes of Health (NIH) has an existing initiative, Patient-Reported Outcomes Measurement Information System (PROMIS®), to develop, validate, and standardize PROs across common medical conditions for studies that use PROs. Sponsors can seek additional information by referencing the FDA guidance for industry on patient-reported outcomes and the European Medical Agency (EMA) reflection paper on expectations for electronic source data [PDF].
Perhaps most compellingly, the FDA has stated its preference for ePROs over paper PROs. At Premier Research, we have developed our own in-house ePRO platform for use in our clinical studies. We also have extensive experience with other ePRO platforms and the implementation of such collection methods. Contact us to find out how your organization can best optimize such tools in your clinical programs.
1 U.S. Food and Drug Administration. Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims, December 2009. Available at https://www.fda.gov/media/77832/download.