Earlier this week, Chief Advocacy Officer Ryan Fischer addressed the FDA’s Center for Biologics Evaluation and Research (CBER) Office of Tissues and Advanced Therapies (OTAT) at a Public Patient-Focused Drug Development (PFDD) Meeting on Patient Perspectives on Gene Therapy Products. 

We would like to thank FDA CBER OTAT for convening this important forum and inviting PPMD to speak about the importance of incorporating patient experience data into gene therapy study designs and regulatory decision-making. We would also like to thank patient advocates, Jenn Mcnary, Tushar Tangsali, and PPMD Adult Advisory Committee (PAAC) member Colin Werth for providing amazing testimonies. 

PPMD’s Testimony:
Ryan Fischer, Chief Advocacy Officer

During the forum, PPMD’s Ryan Fischer spoke to the unique tools and approaches the FDA can utilize to apply patient experience data in gene therapy studies. 

To kick off his comments, Ryan highlighted PPMD’s community-led therapy development guidance and the progress that has been made since its inception in 2014, including five FDA approvals. The goal of this project was to provide a roadmap for companies that are developing therapies for the Duchenne community. An update of this guidance was formally submitted in September of this year. 

The focus of Ryan’s presentation was on the value of patient preference information. PPMD has been directing preference studies since 2014 with the goal of learning and quantifying how patients and caregivers think and feel about emerging therapies and treatments. Ryan highlighted that the results from these preference studies have consistently shown that patients and caregivers are willing to take on serious risks in exchange for a therapy that could slow progression. The information and data gathered from these studies help PPMD and other stakeholders better advocate and make decisions on behalf of the Duchenne community. 

Ryan showcased different methodologies PPMD has used for preference studies and detailed how important these studies are for PFDD, especially for making decisions around emerging gene therapy treatments. He urged FDA to incorporate patient experience data into decision-making and importantly, into the labeling of future gene therapy products. 

You can read Ryan’s full statement here.

Patient Testimony:
Colin Werth, PAAC Member

PPMD Adult Advisory Committee (PAAC) member Colin Werth provided a powerful testimony speaking to the importance of considering patient perspectives in trial design and decision-making. Click here to read Colin’s full statement.