Jett Foundation’s study “Your Voice: Impact of DMD; a Qualitative Assessment of the Impact of DMD on Families” was recently published in Orphanet Journal of Rare Diseases. The project began with the enrollment of 60 patients and caregivers into a qualitative and quantitative study that identified concepts of interest important to the patient community. The project has identified disease burden and treatment needs from the perspective of patients and caregivers of those impacted by DMD who represented ambulatory, transitional, and non-ambulatory stages of disability progression.
Sponsored by Jett Foundation, the study was conducted by Engage Health, Inc., with the regulatory guidance of James Valentine, and the scientific guidance of Dimitrios Savva, Linda Lowes, and Natalie Miller, and with the publication assistance of Carolyn Schwartz at the Deltaquest Foundation.
Jett Foundation conducted the study with the collaboration and support of our Duchenne Biotechnology Council (DBC) partners following concerns about the lack of appropriate endpoints in Duchenne drug development that reflect the interests of patients and their families. The project was designed to improve the understanding of the patient perspective and the treatment goals of patients and families across the spectrum of disease, with the goal of supporting the decision-making of regulators, insurers, and policymakers and identifying patient and caregiver concepts of interest across the broad-spectrum disease.
The research identified whether various aspects of life and daily functioning became more or less important to individuals as they transitioned from the ambulatory to non-ambulatory phase of their Duchenne journey. Participants reported their personal perspectives in areas such as sports/recreation, health, relationships, self-esteem and confidence, independence, and emotional function.
“While certain treatment goals such as daily functioning are consistent across the disability trajectory, the study found an evolution of priorities as the person with Duchenne loses mobility and ambulation,” said Eric Snyder, Executive Director of Jett Foundation. “As an organization whose mission is empowering patients and families with Duchenne, it is important to talk about patient-centered drug development. Data such as this is imperative in order to inform researchers about what is meaningful to patients and families at each functional stage of the Duchenne disease process. We want to thank all of the families involved in this groundbreaking research and look forward to sharing more at upcoming scientific and family conferences.”
To learn more about the “Your Voice” study and its results, read the full article at Orphanet Journal of Rare Diseases.