RAND and PPMD Project on Engaging Patients and 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 will try to address 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 will have several different steps, and we will be asking the Duchenne community to contribute to all of them. You will be able participate in more than one step. However, participation in one step does not mean that you will be required to participate in other steps. Given the online nature of the project, the first three study steps will not require you to leave your home, and you will be able to participate when it is convenient to you. Below is a brief description of each step, a rough timeline, the number of people we plan to engage, time commitment required, and compensation offered.

 

DatesStudy StepsDescriptionNumber of ParticipantsEstimated Time CommitmentCompensation
June-July 2017 Patient/caregiver input on study design Online survey and discussion  40 Approximately 1-2 hours over the course of 1 week $50
September-October 2017 Pilot test of the proposed method A three-round online panel: survey-discussion-survey 10 Approximately 3-4 hours over 3 weeks Up to $150
March-April 2018 Implementation of the new method A three-round online panel: survey-discussion-survey 80-95 Approximately 3-4 hours over 3 weeks Up to $150
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 reaching out to many of you soon. 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 Lucas, 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.).

Posted 5/10/2017