Engaging Patients and Caregivers Managing Rare Diseases to Improve the Methods of Clinical Guideline Development [Methods Study], United States, 2016-2020 (ICPSR 39626)
Version Date: Dec 15, 2025 View help for published
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Dmitry Khodyakov, RAND Corporation
https://doi.org/10.3886/ICPSR39626.v1
Version V1
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Clinical practice guidelines help doctors decide on treatments to recommend for their patients. Guidelines are based on research that looks at the benefits and harms of different treatments. Patient and caregiver input can improve the usefulness of guidelines. But guideline developers often rely on the input of only a few patients and caregivers.
In this study, the research team created a process for getting feedback on guidelines from larger groups of patients and caregivers. This process is called the RAND/PPMD Patient-Centeredness Method, or RPM. The team tested RPM with guidelines for Duchenne muscular dystrophy, or DMD. DMD is a severe form of muscle loss that mostly affects young boys.
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Study Purpose View help for Study Purpose
To develop and test a scalable process for systematically eliciting patient and caregiver input on clinical practice guidelines.
Study Design View help for Study Design
This mixed-methods study developed an online modified-Delphi approach for eliciting feedback on clinical practice guidelines from large numbers of patients and caregivers, called the RAND/PPMD Patient-Centeredness Method (RPM). Researchers tested RPM by asking patients with Duchenne muscular dystrophy (DMD) and their caregivers to rate the patient-centeredness, which the researchers operationalized as importance and acceptability, of recommendations for the management of DMD included in the 2018 DMD care considerations.
Researchers recruited 24 patients and 71 caregivers from the Duchenne Registry to provide input. Participants were randomly assigned to one of two panels. In round one, participants in each panel used nine-point Likert scales to rate the importance and acceptability of 19 recommendations. Participants also explained their ratings and the factors that influenced their responses.
In round two, participants reviewed bar charts showing how their round one responses compared with the responses of other panel members and whether the panel reached consensus on each recommendation's importance and acceptability. Participants then discussed their feedback in a moderated online forum.
In round three, participants could revise their original ratings based on round two feedback and discussions.
Researchers assessed the extent to which agreement among participants on importance and acceptability ratings changed between rounds one and three, the replicability between the two panels' final ratings, and the patient-centeredness of DMD care considerations, which the researchers defined as two panels agreeing that a recommendation is both important and acceptable.
Patients, caregivers, clinicians, and guideline developers helped design and provide feedback on the study.
Universe View help for Universe
Patients with Duchenne muscular dystrophy and their caregivers across the US
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Ratings of importance and acceptability of 19 recommendations from the Duchenne muscular dystrophy care considerations made by 95 members of the Duchenne Registry
Notes
The public-use data files in this collection are available for access by the general public. Access does not require affiliation with an ICPSR member institution.
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This study is maintained and distributed by the Patient-Centered Outcomes Data Repository (PCODR). PCODR is the official data repository of the Patient-Centered Outcomes Research Initiative (PCORI).