Background: Patients are increasingly viewed as key stakeholders who can contribute in meaningful ways to clinical research and are emphasized in research funded by the Patient-Centered Outcomes Research Institute (PCORI). We are not aware of other peer-reviewed publications that report methods and outcomes of patient engagement to refine study design for a PCORI-sponsored clinical effectiveness trial.
Objective: The aim of this report was to describe the process and outcomes of involving patients in the design of a clinical trial to promote adherence to supplemental oxygen therapy among patients with chronic obstructive pulmonary disease.
Methods: In-person focus groups and individual discussions via telephone and email were used to elicit feedback to refine the intervention and clarify outcomes of highest importance to patients.
Results: A total of 25 patients and five caregivers provided feedback. Their feedback has informed decisions regarding the length of intervention sessions (20 min and in some cases longer was acceptable), the importance of including caregivers, and discussion topics (e.g., social discomfort about using oxygen in public, identifying personally relevant reasons to use oxygen, pulmonary rehabilitation). Multiple outcomes were rated as highly important to patients (physical function, fatigue, sleep, anxiety, depression, and ability to participate in social roles and activities), and the outcome that was ranked as most important varied by individual. Therefore, multiple patient-reported outcomes will be used as endpoints for the clinical trial.
Conclusions: Patient involvement led to refinements of the intervention and clinical trial endpoints to better address the expressed needs and concerns of patients and caregivers.