Update on RAND/PPMD Project on Engaging Patients & Caregivers

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

Researchers at RAND Health and Parent Project Muscular Dystrophy (PPMD) have been funded by the Patient-Centered Outcomes Research Institute (PCORI) to conduct the study "Engaging Patients and Caregivers Managing Rare Diseases to Improve the Methods of Clinical Guideline Development."

In this study, we will explore whether the clinical care guidelines for Duchenne muscular dystrophy are consistent with patients' needs and preferences. In the past, patients and caregivers have not been consistently involved in the guideline development process. This study addresses this critical gap by creating a new online method for engaging patients and caregivers in guideline development. We recently published an article that details our plans for developing this new method. You can read it here.

Our method will allow many patients and families to provide their input on how well a given approach to care specified in a guideline addresses patients' most common needs, concerns, values, or preferences. Our hope is to develop a scalable, low burden, and easy-to-use method that would allow the patient voice to be included in future guideline development projects regardless of the clinical condition. We believe that patient and caregiver involvement will make clinical care guidelines more meaningful and useful to the patient community.

Our study consists of several different steps, two of which have been completed. At this time, we would like to thank all of you who have already helped us develop the new method and encourage other members of the Duchenne community to respond to our invitation (to be sent February 2018) to participate in the third and fourth steps of our project where we will be testing the implementation of the method and discussing the results of the study at the annual PPMD Connect Conference. Below is a brief description of different phases of our project.

 

DatesStudy StepsDescriptionNumber of ParticipantsEstimated Time CommitmentCompensation
Completed in July 2017 Patient/caregiver input on study design Online survey and discussion  40 Approximately 1-2 hours over the course of 1 week $50
Completed in December 2017 Pilot test of the proposed method A three-round online panel: survey-discussion-survey 10 Approximately 3-4 hours over 5 weeks Up to $150
March-April 2018 Implementation of the new method A three-round online panel: survey-discussion-survey 95 Approximately 3-4 hours over 4-5 weeks Up to $175
June 2018 Follow up discussion Face-to-face discussion of results at PPMD Connect Conference 12 Approximately 1-1.5 hours $50 and free lunch

 

We will start inviting many of you in February 2018 to participate in the third step of our project. If you have any questions about this study or would like to participate in a particular study step or steps, please feel free to contact Ann Martin, MS, CGC (This email address is being protected from spambots. You need JavaScript enabled to view it.), Kathi Kinnett, MSN, CNP (This email address is being protected from spambots. You need JavaScript enabled to view it.), or Dmitry Khodyakov, PhD, MA, BA (This email address is being protected from spambots. You need JavaScript enabled to view it.).

Updated 2/8/2018