Where possible, investigators are encouraged to use measures with adequate support for validity that are in the public domain. When choosing a PRO measure for an ePCT, investigators should ask the following questions:

• Are there core outcome sets or other widely used measures that are appropriate for my research question?
• Is the PRO data already being collected? If so, how and where are the data collected? Are the data available in the EHR or through other mechanisms?
• If the measure is not in the EHR system, what is the process for adding it? Who will be responsible for the cost? Are there alternative data capture platforms (e.g., REDCap)? Will the measure be acceptable and not burdensome to my patient population?
• Does the measure have support for validity in this use case (i.e., in this target population, setting, etc)?
  • If not, is there a similar measure with existing support for validity in this population?
    • If not, how will you gather necessary data to support validity?
• Is the measure in the public domain?
  • If not, is there a similar measure in the public domain that would be acceptable?

Investigators may want to consider NIH-sponsored measures (see Additional Resources) or the Core Outcome Sets (Section 3). If challenges arise in accessing appropriate PROs, funders may be open to troubleshooting or adapting plans to give the project the best chance of success.

Availability of Data

If PRO measures are already being collected as part of clinical care or quality assurance, then the investigator's priority is to determine how consistently the measures are collected across sites and in what format. In some cases, PRO data may be extracted from the EHR for research, but investigators should not assume that all sites are collecting PRO outcome data in the same way, if at all. For example, PPACT enrolled a patient population who had chronic pain and were on long-term opioid therapy. Although they initially were assured routine PRO measures for pain were collected (at each primary care clinic visit and at least quarterly) as part of required opioid treatment plan contracts and that the PRO measures would be in the EHR, collection of PRO measures was far less systematic than anticipated. Investigators from PPACT stated, “Despite the recognition of the potential benefits of using pain-related PROs, their systematic use in everyday clinical care is rare. In general, the use of pain-related PROs is not embedded into routine clinical practice in health care systems or coordinated with electronic health record (EHR) systems" (Owen-Smith et al. 2018). The investigators worked closely with clinical stakeholders at the participating health care systems to establish an acceptable assessment (DeBar et al. 2018).

Acceptability and Burden

Acceptability of the measure and perception of burden are important considerations for PROs, as lengthy questionnaires can lead to burnout for both the clinician and the patient. In general, PROs are more acceptable to patients if they provide value, such as informing treatment decisions, fostering meaningful communication between the provider and the patient, or helping with triage. Investigators should also ensure that the measure is not too burdensome: brief forms have generally been found to be preferable to longer forms.

For example, the PPACT trial was designed coordinate and integrate services for helping patients adopt self-management skills for managing chronic pain, limit use of opioid medications, and identify exacerbating factors amenable to treatment that are feasible and sustainable within the primary care setting. The primary outcome of pain and pain-related disability was measured through a patient questionnaire. Investigators worked closely with each of the participating healthcare systems to identify a suitable questionnaire that was brief enough, had acceptable psychometrics, focused on functioning, and was easily interpretable (DeBar et al. 2018). Given these needs, clinical leaders and primary care physicians (PCPs) at participating healthcare systems agreed that the 3-item PEG (Krebs et al. 2009) was more acceptable to clinicians than the 12-item Brief Pain Inventory (Cleeland and Ryan, 1994) from which the PEG was derived. Clinicians also viewed favorably the PEG’s emphasis on important functional domains (enjoyment of life, general activity, and an additional item requested by clinical stakeholders on sleep) rather than pain intensity (the focus of the frequently used numerical rating scale) when setting goals with patients.

“PCPs want to ask what’s really needed, and we should consider how we can marry that with clinical research.” —Lynn Debar, PI of PPACT

Patient feedback indicated concern that if they self-reported that their pain had improved, they might get taken off opioids, so the emphasis of the PEG on functioning helped clinicians shift the focus of their conversation with their  patients to what would help in improving the patients’ day-to-day functioning rather than getting mired in a sometimes contentious interchange around pain levels and opioids.

The 3-item PEG asks the participant to score the following questions on a 0-10 scale:

1. What number best describes your pain on average in the past week?

2. What number best describes how, during the past week, pain has interfered with your enjoyment of life?

3. What number best describes how, during the past week, pain has interfered with your general activity?

PPCAT investigators also included a fourth item on sleep:

4. What number best describes how, during the past week, pain has interfered with your sleep?