Type C FDA Meeting on Gene Editing/ Therapy

In October 2022 FARA coordinated a meeting with over a dozen companies and the FDA Office of Tissues and Advanced Therapies (OTAT), one of three product offices within Center for Biologics Evaluation and Research (CBER). The objectives of the meeting included growing the understanding of FA with this regulatory agency as well as discussion of pre-competitive issues and questions directly related to the development of gene therapies and gene editing products. Prior to the meeting, FARA solicited questions from the industry partners and wrote a briefing book with background information and specific questions to be discussed at the meeting. FARA drafted minutes from the meeting that were reviewed by the FDA staff that attended the meeting. These materials are now available here.

Image of FA Ambassadors Jack, Alex, an Shandra. Text reads: "During the meeting with FDA, FARA ambassadors, Jack, Alex, and Shandra shared their personal experiences living with FA and discussed patient preferences related to treatments, benefit-risk considerations, and clinical trial experience."

FDA Patient Listening Sessions

Periodically, FARA will respond to regulatory agency requests to provide input on policies under development. For example, FARA has participated in Patient Listening Sessions, which provide an opportunity for patients and advocacy organizations to share their experiences and perspectives by talking directly with FDA staff. Written contributions provided by FARA to regulatory agencies can be found below.

Patient Focused Drug Development Meeting

In June 2017, FARA held an Externally Led Patient Focused Drug Development Meeting. The goal of the meeting was to give FA patients and their families, friends, and caregivers a forum to discuss their perspectives on living with the disease: what symptoms patients experience, how it affects their lives, what concerns them about the disease, their opinions on current and future treatment options, and experiences with research studies and clinical trials.

A recording of the meeting in its entirety can be accessed by clicking the image to the left.

Voice of the Patient Report
Woman speaker at event