The SDS Alliance focuses on making therapies and cures a reality for SDS patients worldwide. A major stakeholder in this journey is the FDA (the US Food and Drug Administration). Engaging with the FDA early and meaningfully is a high priority on our roadmap.
On November 15th, Dr. Eszter Hars (SDS Alliance president and CEO) joined the FDA CBER OTAT Patient-Focused Drug Development Listening Meeting — Patient Perspectives on Gene Therapy Products meeting. We took the opportunity to highlight our community’s needs and perspectives, as several gene therapy projects are advancing - slowly but surely - towards the clinic.
"This meeting was our first step in working with the FDA to facilitate the evaluation of SDS therapies in the future to make them a reality." says Dr. Hars.
The meeting was hosted by the FDA’s Center for Biologics Evaluation and Research (CBER) Office of Tissues and Advanced Therapies (OTAT) to better understand patient perspectives on gene therapy products, including cell-mediated gene therapies. This event was held to meet an FDA commitment that is part of the seventh authorization of the Prescription Drug User Fee Act (PDUFA VII).
“Patients and their advocates possess the unique, first-hand perspective of what it is like to live with or care for an individual with a disease, as well as the impact of available treatments on daily life. Patients and caregivers can provide valuable input into the discussion of investigational therapies by describing their experience with a disease or condition, and by defining meaningful change in terms of their specific disease and the risks they are willing to accept. With the potential for more gene therapies to become available to patients in the future, it is important to understand patient and caregiver perspectives on these products.” ~FDA CBER OTAT
The FDA is also collecting feedback and comments via written and electronic comments. These comments must be submitted by December 15, 2022.
About the FDA
The United States Food and Drug Administration (FDA or USFDA) is a federal agency of the Department of Health and Human Services. The FDA is responsible for protecting and promoting public health through the control and supervision of food safety, tobacco products, dietary supplements, prescription and over-the-counter pharmaceutical drugs (medications), vaccines, biopharmaceuticals, blood transfusions, medical devices, electromagnetic radiation emitting devices (ERED), cosmetics, animal foods & feed and veterinary products. (See more at FDA.gov and on wikipedia here: https://en.wikipedia.org/wiki/Food_and_Drug_Administration)
OTAT (a part of the FDA) oversees development for a wide variety of biological products, including gene and cell therapies, tissues and tissue engineering products, xenogeneic products, and more. OTAT strives to lead all regulatory decisions with data, impartiality, and compassion and always welcomes the participation of patients and their advocates in formal meetings related to the development of investigational products. Learn more about OTAT and view our available resources.