When the FDA issues a Complete Response Letter (CRL), it can be an emotional moment for individuals and families who are eager for progress in Ataxia research and treatment. A CRL means the FDA has completed its review and determined that the application cannot be approved in its current form. Simply put, the FDA does not believe the data provided on a therapy met the threshold for approval and has asked for additional data. If you would like to understand why a CRL was issued, the FDA has a publicly available search tool where you can access a copy of the letter.
Many people in our community may want to voice their experiences, share the urgency they feel, or ask decision-makers to consider the real-world impact of delays.
While the National Ataxia Foundation does not advocate for or against approval of specific therapies, we can help empower patients and families who wish to share their perspectives in a constructive and effective way. All feedback on a decision is helpful – positive, negative, and neutral.
This roadmap outlines 4 ways individuals can communicate with policymakers, federal agencies, and the public so your voice can help inform the systems shaping Ataxia drug development.
1. Write to FDA Leadership and Decision-Makers
Hearing directly from people affected by Ataxia is vital to ensuring that the FDA understands the lived experience behind scientific data. If you wish to share your story or opinion with the FDA:
- Explain your lived experience with Ataxia or how Ataxia affects your family.
- Describe what treatment delays mean in daily life, such as: mobility changes, disease progression, safety concerns, caregiving burden, and quality-of-life impacts.
- Share your perspective on why timely evaluation of therapies matters.
- Be specific, respectful, and focused on the patient experience, this is what most influences regulators.
FDA Contact Information:
- Guide to Submitting Comments to the FDA – Share your knowledge and expertise with FDA to make your voice heard regarding pending regulatory policies.
- Contact the Public Engagement Staff at FDA – They help patients become more informed and involved in FDA regulatory decisions related to medical products.
- FDA Social Media Handles:
- Facebook – @U.S. Food and Drug Administration
- Instagram – @fda
- X – @US_FDA
- Dr. Marty Makary, FDA Commissioner Social Media Handle:
- X – @DrMakaryFDA
2. Engage With Your Senators and Representatives
Members of Congress provide oversight of federal agencies, including the FDA. They play an important role in ensuring policies reflect the needs of constituents, especially in rare disease communities.
If you choose to contact your elected officials, consider:
- Sharing your personal story and how Ataxia affects you or your loved one.
- Explaining the importance of rapid but responsible drug development pathways.
- Highlighting the urgency families feel when treatment options are limited.
- Asking them to support strong oversight and ensure the patient voice remains central in FDA decision-making.
Find Your Local Representatives
You may contact your legislators by email, social media, mail, phone, or scheduling a meeting at their district office. Contact information for each individual can be found using the tools below. Social media handles can usually be found by visiting the individual’s website or searching for their verified page on social media.
3. Share Your Story with Media
Personal stories have tremendous power. They help the public and policymakers understand the real-world impact of Ataxia in a way data cannot.
If you choose to share your story publicly:
- Focus on how Ataxia affects daily life and long-term hopes.
- Explain why treatment development matters to you personally.
- Be clear about what you are asking for: attention, urgency, or support for regulatory flexibility.
- Consider writing an opinion piece or speaking to local news reporters.
Be mindful that media interviews reach broad audiences, so think about what you are comfortable sharing publicly.
4. Use Social Media to Spread Awareness
Social platforms can help amplify your voice, connect with others who share your concerns, and keep policymakers aware of community needs.
If you decide to post:
- Share your personal experience with Ataxia and its impact.
- Explain how delays in treatment development affect families.
- Tag FDA accounts or elected officials to keep issues on their radar.
- Encourage respectful, constructive dialogue. Positivity and clarity are often more persuasive than anger or frustration.
Social media posts can also direct others to take action by contacting their own representatives.
Key Laws Supporting Patient Advocacy in Drug Development
Below are two major U.S. federal laws that patients and families can reference when speaking with the FDA or elected officials. These laws reinforce the importance of the patient voice, regulatory flexibility, and faster access to therapies for serious and rare diseases.
- Accelerating Access to Critical Therapies for ALS Act (ACT for ALS)
ACT for ALS directs the FDA to consider the urgent needs of people living with serious neurodegenerative diseases and to use every appropriate tool—like flexible trial design and accelerated pathways—to evaluate treatments efficiently. Patients may reference this law when asking the FDA or Congress to prioritize urgency, patient experience, and regulatory flexibility in Ataxia drug reviews.
Learn more. - 21st Century Cures Act
The 21st Century Cures Act requires the FDA to integrate patient input and real-world experience into regulatory decisions and encourages flexible, innovative approaches to evaluating treatments. This law empowers patients to remind policymakers that the FDA must consider lived experience, quality of life impacts, and unmet medical needs in its reviews.
Learn more.
Tips for Effective Advocacy
No matter which path you choose, these suggestions can help strengthen your message:
- Lead with your lived experience. Policymakers want to understand why this matters to you.
- Share the human impact, not scientific conclusions. Patients don’t need to be experts in trial design to speak powerfully.
- Be respectful and specific. Constructive messages are more likely to be heard and considered.
- Stay factual. Focus on personal experience, urgency, and the need for progress.
Additional Resources
- Using Storytelling to Raise Awareness for Your Disease
Not sure where to start or how to tell your story? This resource from Global Genes has a guide to storytelling for rare disease. - Download Example Letter to a Member of Congress
- Understanding the FDA Process
- FDA Complete Response Letter Search Tool
When the FDA issues a CRL, a copy of the letter is available publicly. To find the letter, search the sponsor company’s name. The tool is case sensitive. Capitalization may affect search results. If you do not find a CRL you are expecting to find, try again but change to capital or lowercase for the first letter of each word in the company name. - NAF’s Advocacy Initiatives and Tools
If you have questions about advocacy or want guidance on how to share your story effectively, our Advocacy team is here to help. You can reach us anytime at support@ataxia.org.
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