PPMD has a long history of working alongside our community to document treatment preferences and share those insights with regulatory authorities such as the FDA. Through town halls, listening sessions, and conversations at community events, we consistently hear that stability is a critically important (transformative) outcome in both care and treatment. There is no consensus on what “stability” means to families or the relative desirability of stability to the Duchenne community, factors that are critically important as regulators review therapies.
We are pleased to collaborate with a team led by Dr. John F. P. Bridges at The Ohio State University to launch a new patient preference survey. This study aims to better understand how individuals with Duchenne and their caregivers value stability as an outcome of therapeutic intervention, and to capture what stability truly means in the context of treatment and care. The survey is now recruiting participants.
Eric Camino, PhD, PPMD’s Vice President of Research and Clinical Innovation shares, “These patient preference studies are invaluable tools for quantifying and sharing the values and needs of our community to regulatory decision makers. It’s crucial that what this community defines as benefit is taken into consideration when decisions regarding therapy approvals are being made.”
This project builds on PPMD’s extensive body of patient preference research. Recent preference work has reflected community views on newborn screening and risk tolerance related to gene therapies. Dr. Bridges and his team have played a leading role in advancing patient preference work in Duchenne for more than a decade.
Dr. Bridges added, “Our partnership with PPMD continues to advance and apply cutting-edge techniques to promote patient-focused drug development. We have worked closely with the Duchenne community to develop this survey to quantify the importance of stability and to understand what it means to families.”
If you are an adult with Duchenne or a caregiver to an individual with Duchenne and are interested in participating, please follow the link below:
2025 Treatment Preference Study
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