When pragmatic trials test interventions designed to change how someone feels or functions in their day-to-day lives, outcomes can be obtained through direct patient report, as patients are typically the best source of information about how they are feeling and managing outside of the clinic. For example, the PPACT trial tested whether multidisciplinary services, such as physical therapy and psychological interventions, would lead to improvements in pain, as reported by participants.

As defined by the U.S. Food and Drug Administration (FDA), a patient-reported outcome (PRO) is “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" (FDA Guidance for Industry 2009). Work funded by the Patient-Centered Outcomes Research Institute (PCORI) defines PROs as “the patient’s report of the impact of health, disease, and treatment from the patient perspective, generally collected via questionnaires (Snyder and Wu 2017).” This definition makes a distinction between patient-generated data collected from devices (such as pedometers or home blood pressure monitors) and patients’ direct report of outcomes, which typically include information about symptoms, functioning, satisfaction with care or symptoms, adherence to prescribed medications or other therapy, and perceived value of treatment.

In this chapter, we describe how PROs are used in different settings and how to choose and integrate a PRO measure into an embedded pragmatic clinical trial protocol. In addition, we briefly describe resources for measures, including core outcome sets for PROs.