In 2017, FARA released the Friedreich’s Ataxia “Voice of the Patient” report (link below); a unique summary of direct testimony and survey responses from individuals living with Friedreich’s Ataxia (FA). This report is based on the proceedings surrounding the Externally Led Patient Focused Drug Development Meeting held on June 2, 2017 in Hyattsville, MD. 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.
This meeting, and the report, represent important accomplishments and milestones for our community.
- This was the largest FA meeting ever held with more than 400 people participating in person or online; including >20 FDA representatives, 25 drug company representatives and >350 FA patient and family participants.
- It was the first time FA patients and other members of the FA community had an opportunity to communicate directly with representatives of the Food and Drug Administration.
- Important themes and insights emerged that can inform drug development and regulatory decision making, such as the conclusion that “while the majority of patients noted that balance, dexterity and fatigue affected their lives the most now, most patients’ greatest concern for their future is the development/ progression of cardiomyopathy in the future, because it is life shortening”.
This document faithfully summarizes the testimony, comments and survey responses shared by individuals living with FA and their family members and caregivers.
We are very grateful to FDA for giving us the opportunity to hold this meeting, and for taking the time to participate and listen to our community. We hope that regulators and companies developing therapies for Friedreich’s ataxia will read this report and better understand the great unmet medical need of FA, the impact it has on patients and what patients seek in terms of treatment. This, in turn, will help companies develop therapies that affect the aspects of disease most important to patients, and regulators to approve those therapies with the greatest impact on patients.
We are grateful to all the individuals in the FA Community who participated in this meeting through providing testimony (both oral and written), attending the meeting, watching online and/or responding to the survey questions. We believe that your engagement in this process provided huge insight into the disease ,which will help the drug development community develop effective therapies.
The report can be read at http://curefa.org/pdf/news/FA-Voice-of-the-Patient.pdf
A cover page for the report that describes disclosures and authorship is available here: http://curefa.org/pdf/research/CoverPage-VOP-Report.pdf
A recording of the meeting in its entirety can be found at https://www.youtube.com/watch?v=Va1D4SqrSfw
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 (see link) with background information and specific questions to be discussed at the meeting. FARA drafted minutes from the meeting (see link) that were reviewed by the FDA staff that attended the meeting. These materials are now available here.
Meeting Briefing Package (PDF)
Meeting Minutes (PDF)
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 FARA has provided to regulatory agencies can be found below.
OTAT Listening session FARA comments Nov 2022 (PDF)