Engaging Patients and Caregivers Managing Rare Diseases to Improve the Methods of Clinical Guideline Development

A Research Protocol

Published in: JMIR Research Protocols, Volume 6, Number 4 (April 2017): e57. doi: 10.2196/resprot.6902

by Dmitry Khodyakov, Sean Grant, Ian D. Coulter, Arlene Fink

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Clinical guidelines provide systematically developed recommendations for deciding on appropriate health care options for specific conditions and clinical circumstances. Up until recently, patients and caregivers have rarely been included in the process of developing care guidelines.


This project will develop and test a new online method for including patients and their caregivers in this process using Duchenne muscular dystrophy (DMD) care guidelines as an example. The new method will mirror and complement the RAND/UCLA Appropriateness Method (RAM) — the gold standard approach for conducting clinical expert panels that uses a modified Delphi format. RAM is often used in clinical guideline development to determine care appropriateness and necessity in situations where existing clinical evidence is uncertain, weak, or unavailable.


To develop the new method for engaging patients and their caregivers in guideline development, we will first conduct interviews with experts on RAM, guideline development, patient engagement, and patient-centeredness and engage with Duchenne patients and caregivers to identify how RAM should be modified for the purposes of patient engagement and what rating criteria should patients and caregivers use to provide their input during the process of guideline development. Once the new method is piloted, we will test it by conducting two concurrently run patient/caregiver panels that will rate patient-centeredness of a subset of DMD care management recommendations already deemed clinically appropriate and necessary. The ExpertLens™ system-a previously evaluated online modified Delphi system that combines two rounds of rating with a round of feedback and moderated online discussions — will be used to conduct these panels. In addition to developing and testing the new engagement method, we will work with the members of our project's Advisory Board to generate a list of best practices for enhancing the level of patient and caregiver involvement in the guideline development process. We will solicit input on these best practice from Duchenne patients, caregivers, and clinicians by conducting a series of round-table discussions and making a presentation at an annual conference on Duchenne.


The study protocol was reviewed by RAND's Human Subjects Protection Committee, which determined it to be exempt from review. Interviews with RAM experts have been completed. The projected study completion date is May 2020.


We expect that the new method will make it easier to engage large numbers of patients and caregivers in the process of guideline development in a rigorous and culturally appropriate manner that is consistent with the way clinicians participate in guideline development. Moreover, this project will develop best practices that could help involve patients and caregivers in the clinical guideline development process in other clinical areas, thereby facilitating the work of guideline developers.

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