When effectively engaged, patient partners can give valuable insight on the relevance, acceptability, and feasibility of a PCT (Ellis et al 2017). They can also describe how an intervention and related trial processes may be perceived or experienced by patients in a real-world setting. Insight from these lived experiences, when used to help shape the intervention itself, can play a role in designing a trial that is impactful and generalizable outside the research setting.

Patients have the unique perspective and ability to provide information about lived experiences that can inform research (FDA 2020), including:

• Symptoms they experience and how these affect day-to-day functioning and quality of life, including which symptoms are most bothersome
• Burden of living with or managing a health condition
• Experience with treatments, including side effects and burdens related to treatment
• Views on potential treatment outcomes and importance of different possible outcomes
• Expectations of benefits
• Tolerance for harms or risks, including what is an acceptable trade-off between potential benefits and risks
• Views on unmet medical needs and available treatment options
• Attitudes toward uncertainty

PCORI has assembled a searchable collection of peer-reviewed articles on the evidence for patient engagement. Overall, patient partnerships have been found to be valuable throughout the research process, from planning research to conducting studies and disseminating results (PCORI 2023). Active involvement of patients in research can lead to research of greater quality and relevance owing to the unique perspective that users can bring to a research project (Brett 2014).

In addition to the qualitative value of patient engagement in research, a study from CTTI’s Patient Groups and Clinical Trials Project concluded that engagement activities may have considerable financial value related to their role in avoiding protocol amendments and improving enrollment, adherence, and retention (Levitan et al 2017).

Furthermore, patient partners have reported benefits from participating in engagement activities, such as experiencing acceptance, gaining knowledge, and contributing to helping others (Poole et al 2023).

PCORI notes that patient partners should be recognized appropriately for their valuable contributions to research (PCORI 2023).

PCT Experiences

The principal investigators of all NIH Pragmatic Trials Collaboratory Trials who reported conducting patient engagement activities felt that they provided value. Dr. Isabel Roth, who led partner engagement activities for the OPTIMUM trial, described PCTs as “a natural stage for engagement.”

Activities highlighted by the research teams included:

• Conducting semistructured interviews around trial design
• Developing a patient advisory panel to vet materials
• Hosting post-intervention interviews
• Having patient partners review consent forms
• Holding focus groups around recruitment

NIH Collaboratory researchers described that patient partners provided helpful direction on their trial interventions, suggestions to improve the patient experience, and guidance on trial messaging for participants. Patient partners can also add insight about the potential implementation of research findings and sustainment of interventions. The AIM-Back trial, described below, concluded that partner engagement can increase acceptability of new care pathways to patients and providers and enhance uptake of effective interventions by health systems (Ballengee et al 2023). In this way, patient engagement in PCTs can be a tool for health systems to help meet the goals of patient-centered care.

Case Study: PREPARE

The Person Empowered Asthma Relief (PREPARE) trial was a PCORI-sponsored PCT coordinated by the Duke Clinical Research Institute (co-PI, Frank Rockhold, PhD). It was designed to determine how to improve outcomes for Black and Latinx patients experiencing mild to moderate asthma. Patient partners were engaged throughout the trial and were instrumental in helping the research team understand the study populations and potential barriers to their participation. Patient partners reviewed all patient-facing study materials and provided feedback, in which they emphasized the importance of simple messaging, identifying clinics that treat the target populations, using the specific Spanish-language vernacular, giving adequate and immediate payment for survey completion, and sending appreciation notes from investigators.

Case Study: AIM-Back

In the Improving Veteran Access to Integrated Management of Low Back Pain (AIM-Back) trial, which sought to improve access to recommended and patient-centered treatments for low back pain among veterans, input from patient partners led to several changes in the care pathway to improve the patient experience and realistic usability. These changes included transitioning from telephone-based delivery to a flexible telehealth model, increasing specificity about pain modulation activities, and reducing the number of physical therapy visits. AIM-Back researchers highlighted that patient partners provided immense value in centering the trial around the patient experience. They concluded that diverse input should be considered before implementing new interventions in PCTs (Ballengee et al 2023).