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 is embedded below and 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