Clinical practice guidelines (CPGs) provide systematically developed recommendations for deciding on appropriate health care options for specific conditions and clinical circumstances. Interest in engaging patients in CPG development has increased in recent years. Because CPG developers tend to include only a small number of patients or their caregivers, scalable engagement methods are needed.
The project's goals were to develop and test a new online method for including a large number of patients and their caregivers in the CPG development process using Duchenne muscular dystrophy (DMD) guidelines as an example. We had 3 specific aims: (1) to develop an online modified-Delphi (OMD) approach to systematically engage patients with rare diseases and their caregivers in CPG development; (2) to test the OMD approach; and (3) to develop best practices for using OMD to engage patients and their caregivers in the CPG development process.
To achieve aim 1, we interviewed 9 experts on CPG development and patient engagement, conducted a literature review on online patient involvement in CPG development, consulted with 6 patients with DMD and 25 caregivers, and pilot tested a prototype of the OMD approach with 3 patients with DMD and 8 caregivers. To test the OMD approach, which we called the RAND/Parent Project Muscular Dystrophy Patient-Centeredness Method (RPM) (aim 2), we ran 2 concurrent panels in which 24 patients with DMD and 71 caregivers rated the patient centeredness of 19 recommendations already deemed clinically appropriate and necessary. A recommendation was considered patient centered if both panels deemed it important and acceptable. We measured consensus using the RAND/UCLA Appropriateness Method's approach, evaluated differences in consensus between rounds 1 and 3, assessed replicability of findings between panels, and measured participant satisfaction with the RPM. We also conducted a thematic analysis of participants' comments about patient centeredness and semistructured telephone interviews about their opinions of the RPM. To achieve aim 3, we relied on the data collected for the previous 2 aims and the results of a focus group with 9 caregivers and 2 clinicians.
The RPM we developed in aim 1 consists of 4 rounds. Participants provide input on the reasons for, barriers to, and facilitators of seeking care (round 0); rate the importance and acceptability of CPG recommendations (round 1); review and discuss the initial results (round 2); and revise their original ratings (round 3). In testing the RPM (aim 2), we determined that its iterative nature helped participants move toward consensus: More participants agreed with each other on more questions in round 3 than round 1. Although we did not detect any disagreement among participants in either round, the number of recommendations deemed important/acceptable or uncertain changed. In terms of replicability, both panels reached identical decisions on 34 of 38 rating questions (either importance or acceptability) in round 3, yielding a κ of 0.69 (a substantial level of agreement). Twelve of 19 recommendations met our patient-centeredness criteria. Common reasons for not being deemed patient centered were lack of evidence specific to DMD and concerns about insurance coverage, access to treatment, and patient safety. Participants expressed satisfaction with the RPM, noting that the method helped them better understand DMD and prepared them to discuss difficult issues with providers. Although panelists appreciated participating in an educational, interactive, and convenient discussion forum that allowed them to share their opinions and learn from each other, they noted that participation in a 3-round process requires some effort. In general, the RPM was viewed as empowering by patients and caregivers who felt it would be useful for CPG developers. Based on the study findings, we identified 11 best practices for patient and caregiver engagement in CPG development using OMD approaches (aim 3).
We developed a new online approach that makes it feasible and convenient to engage large numbers of patients and their caregivers in a rigorous and culturally appropriate manner, consistent with the way clinicians participate in CPG development. The RPM produces results that are at least as replicable as those of clinical panels. Patients and caregivers were generally highly satisfied with the OMD process, noting the RPM's ability to facilitate learning and giving them an opportunity to revise their responses, which is important for achieving consensus. The best practices we identified could help involve patients and their caregivers in the CPG development process in other clinical areas, thereby facilitating the work of guideline developers.
Limitations included testing the approach with a sample of already finalized recommendations, not double-coding all interview data, and developing our approach as well as best practices only in the DMD context.
