Advocacy

• Establish a Health and Human Services (HHS) Public-Private Partnership for Rare Neurodegenerative Diseases between the NIH, the FDA, and eligible stakeholders with a connection to the patient population(s) to advance the understanding of rare neurodegenerative diseases and foster the effective development and evaluation of treatments;
• Commission the publication of an FDA Action Plan on rare neurodegenerative diseases;
• Implement an FDA grant program to fund research and therapy development for life-threatening or severely debilitating rare neurodegenerative diseases.

Increased federal funding for research

• Research relies on our federal partners at the National Institutes of Health (NIH) and the Food & Drug Agency (FDA). The NIH is the steward of medical and behavioral research for the National. The FDA is the regulatory authority over new drug applications. The NIH & FDA are complementary in their roles and functions — NIH supports and conducts biomedical and behavioral research and FDA ensures the safety and effectiveness of medical and other products.
• Elected officials will be working on appropriations for the NIH and FDA in April. These agencies are vital to the ataxia drug development programs and they need the resources to appropriately manage the demands of COVID in addition to all the other important research programs they review.

Photo credit: National Organization for Rare Disorders

DOES YOUR STATE HAVE A RARE DISEASE ADVISORY COUNCIL?

In 2015, the first rare disease advisory council was created in North Carolina as a result of the rare disease community coming together and demanding a stronger voice in government. With over 7000 rare diseases, it would be impossible for state legislators to understand the challenges rare disease patients face every day around diagnosis, treatment, and access. Additionally, state policies are often determined without consulting the individual disease community that will be directly affected. A state rare disease advisory council provides an avenue for all stakeholders including patients, caregivers, doctors, insurers, drug manufacturers, and researchers to offer insight and recommendations to state elected officials and leaders as those decisions are being considered.

North Carolina sparked similar legislation to be enacted throughout the country. Today, with the support of the National Organization of Rare Diseases (NORD) Project RDAC and other patient organizations like FARA, sixteen states have rare disease advisory councils with others currently considering creating one. Kudos to the states who have acted!

A comparison state program would be a state legislative caucus. A caucus is a group of legislators coming together around a common interest, such as rare disease. To learn more, check out these resources from the Everylife Foundation.

Creating a Rare Disease State Legislative Caucus

Comparing State Caucuses and Advisory Councils

Letter to FDA & Reata Pharmaceuticals regard Access to Omaveloxolone
A Call to Action was held from January 5, 2021 – January 20, 2021 where the entire FA community (individuals with FA, family, friends, caregivers, supporters, other rare disease advocates) to sign-on a letter to the Food and Drug Administration (FDA) and Reata Pharmaceuticals. This letter requests Reata to submit a New Drug Application (NDA) on an urgent basis and the FDA to exercise the flexibility granted by law and contained in the FDA guidance in considering approval of an NDA for Omaveloxolone in FA based on the existing evidence from clinical trials.
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Community Home Health and Telehealth Policies
In the early weeks and months of the COVID-19 public health emergency, Congress and the Administration took important steps to ensure patients have access to essential care while the nation grappled with controlling the spread of the virus. Specific time-limited regulatory flexibilities have removed significant barriers to care and improved access for the 30 million Americans living with a rare disease or condition including Ataxia. However, these flexibilities are at risk of going away when the public health emergency ends. We urged policymakers to recognize how these flexibilities have benefited members of the rare disease community and consider which policies should be kept in place after the public health emergency ends.

Creating Hope Reauthorization Act
This bill will permanently authorize the Pediatric Priority Review Voucher (PRV) program which will further the opportunity to spur innovation in rare and neglected diseases that disproportionately impact children.
(Photo credit: Duke University - The Fuqua School of Business)

Preparing for Meetings

Meeting with your representative can be one of the most effective ways to share your message. A little preparation before will not only strengthen your position but make you feel more comfortable. Here are some helpful hints to get ready.

• Be prepared. Learn about current issues. Check out FARA's 2021 Initiatives for resource information. Read relevant material and/or listen to any webinars offered. Gather materials that support your position and bring them to the meeting. It is up to you to educate the Member on the particular issue or piece of legislation you are addressing. There is no way that they would know or understand what the implications of their legislative actions would be on FA specifically, or rare disease more generally, unless you tell them!
• Develop your “elevator speech.” How would you explain ataxia to someone who has never heard of it? Include a brief explanation of your diagnostic odyssey if pertinent. Think about details of how ataxia impacts your life daily. Tie your experiences to the issue(s) currently being considered.
• Learn about your elected official. Check out his/her website, read his background, see what areas he/she is most interested in. You may find a personal connection (town, school, activity, sports team) that you both share and this could open a conversation. Advocacy is about building relationships and sometimes that happens over the most unusual items in common.
• Be prompt, be concise, be direct. Elected officials, and their staff, have busy schedules but meeting constituents is an important part of the job. Be considerate of their time but confident in why you are there. Tell them your “ask” or what you want them to do.
  • Don’t be nervous. They are folks just like you, and in fact, they work on your behalf!
  • You will likely meet with a staffer. Do not be disappointed – they are the ones that learn the issues and advise the official. They will also most likely be the person who follows up with you. Good connections with staff will translate to good communications with your representative.
• Make a connection to constituency. Members of Congress want to represent the best interests of their district or state. Who else would be impacted by the action? Would revenue or jobs be impacted?
• Thank them, leave material behind, follow up. Thank them for their time and, if appropriate, offer assistance. Make sure to leave behind relevant material including the FA Fact Sheet. Send a follow up email that includes points covered during the meeting, next steps, and any additional information requested. Follow up periodically on the status of your issue. Each communication helps to develop a relationship with your representative's office.