Pragmatic clinical trials (PCTs) are research studies that are conducted in the context of routine clinical care and therefore have the potential to represent and prioritize the perspectives of an array of key individuals, including patients.

Terms We Will Use

Several terms used throughout this chapter may have different meanings in different contexts, such as explanatory trials. For the purpose of this chapter, the following terms are defined as such:

• Patient partners: A patient engaged with a research team to play a role in trial design, trial conduct, and/or dissemination of information from the trial. Patient refers to individuals seeking healthcare or community resources related to their health or well-being.
• Partner (sometimes referred to as “stakeholder”): “An individual or group who is responsible for or affected by health- and healthcare-related decisions that can be informed by research evidence” (Concannon et al 2012). Note: The term stakeholder has been widely used in the context of public health and clinical research. However, many are encouraging the use of partner to reduce jargon and promote inclusion.
• Engagement: A bidirectional relationship between the partner and researcher that results in informed decision-making about the selection, conduct, and use of research (Concannon et al 2012). The word ‘engagement’ is not being used in the regulatory context, but is being used to describe involvement/interaction with partners.
• Patient engagement: Although this is often referred to as “participant” engagement, we use the term patient for clarity because, in the PCT setting, some trials include clinicians as the recipients of interventions.
• Lived experience: The valuable, unique perspective that partners bring to clinical research from firsthand involvement in situations or events in their everyday lives.
• Patient-centered care: “Patient-centered care focuses on the patient and the individual's particular health care needs. The goal of patient-centered health care is to empower patients to become active participants in their care” (Reynolds 2009).
• Other partners: Additional individuals with valuable perspectives for PCTs who are not patient partners (PCORI 2014). Perspectives of other partners, such as clinicians and health system leaders, are also indispensable for PCTs. We describe the full breadth of partners for PCTs in the chapter "Building Partnerships and Teams to Ensure a Successful Trial."

What Is Patient Engagement?

Because patients can provide valuable insights and perspectives about clinical care for specific conditions, they are key partners for PCTs.

Once regarded as “subjects” who had research performed on them, patients are now contributing across the spectrum of clinical development, including in the design and planning of research protocols, selection of outcomes and endpoints, development of recruitment and retention strategies, and dissemination of research results. (Uhlenbrauck et al 2018)

Comprehensive patient engagement plays an instrumental role in PCTs because these trials are designed to inform an aspirational model for how routine clinical practice should operate. The Patient-Centered Outcomes Research Institute (PCORI) recommends patients be considered valuable partners whose expertise will influence research to be more patient-centered, relevant, and potentially sustainable (PCORI 2018). The term patient engagement refers to an array of activities with various levels of involvement from patient partners (Table).

Table. Level of Patient Engagement in Research

Adapted from Duffet (2017).

While it may be ideal to engage patients at every stage of a trial, patient engagement occurs on a spectrum, and researchers should prioritize time and resources for engagement activities that provide the most value.

Perspectives of other partners, such as clinicians and health system leaders, are also indispensable for PCTs. We describe the full breadth of partners for PCTs in the chapter "Building Partnerships and Teams to Ensure a Successful Trial." Resources for clinician and health system engagement can also be found on the Health Care Systems Interactions Core page. Resources, such as this ePCT Quick Start Guide for Researchers and Health System Leader Partnerships, are designed to promote successful partnerships.

Patient engagement can be ongoing or targeted.

• Ongoing engagement refers to engaging with patient partners at multiple points throughout a trial’s life cycle until results are communicated back to patients and, ideally, knowledge learned is sustained in clinical practice.
• Targeted engagement generally refers to engagement at one point in time that elicits information.

This chapter explores key principles of patient engagement, engagement strategies, potential barriers, and case studies in the PCT context.