Real-World Evidence: Patient-Reported Outcomes (PROs)
Section 5
Choosing PRO Measures
When choosing a PRO measure for an ePCT, investigators should ask the following questions:
- Will measures from core outcome sets or other widely available measures be acceptable 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?
- Will the measure be acceptable and not burdensome to my patient population?
When deciding what measure to choose, investigators may want to consider the NIH-sponsored measures described in the Additional Resources Section or the Core Outcome Sets described in Section 3.
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?
SECTIONS
sections
- Introduction
- How Are PRO Measures Used?
- Best Practices for Collecting PRO measures in Pragmatic Clinical Trials
- NIH HEAL, FDA, and Other Core Outcome Sets
- Choosing PRO Measures
- Cultural Adaptation and Linguistic Translation
- Case Study From Guiding Good Choices for Health
- Incorporating PRO Data Into the EHR
- Patient-Focused Drug Development
- Additional Resources
Resources
Grand Rounds
Development of Harmonized Outcome Measures for Use in Research and Clinical Practice (Richard Gliklich, MD, Michelle Leavy, MPH, Elise Berliner, PhD)
REFERENCES
Cleeland CS, Ryan KM. 1994. Pain assessment: global use of the Brief Pain Inventory. Ann Acad Med Singap. 23:129-38. PMID: 8080219
DeBar L, Benes L, Bonifay A, et al. 2018a. Interdisciplinary team-based care for patients with chronic pain on long-term opioid treatment in primary care (PPACT) - Protocol for a pragmatic cluster randomized trial. Contemp Clin Trials. 67:91-99. doi:10.1016/j.cct.2018.02.015. PMID: 29522897.
Krebs EE, Lorenz KA, Bair MJ, et al. 2009. Development and initial validation of the PEG, a three-item scale assessing pain intensity and interference. J Gen Intern Med. 24(6):733-738. doi:10.1007/s11606-009-0981-1. PMID: 19418100.
Owen-Smith A, Mayhew M, Leo MC, et al. 2018. Automating collection of pain-related patient-reported outcomes to enhance clinical care and research. J Gen Intern Med. 33(S1):31-37. doi:10.1007/s11606-018-4326-9. PMID: 29633139.
current section : Choosing PRO Measures
- Introduction
- How Are PRO Measures Used?
- Best Practices for Collecting PRO measures in Pragmatic Clinical Trials
- NIH HEAL, FDA, and Other Core Outcome Sets
- Choosing PRO Measures
- Cultural Adaptation and Linguistic Translation
- Case Study From Guiding Good Choices for Health
- Incorporating PRO Data Into the EHR
- Patient-Focused Drug Development
- Additional Resources