If the first FARA advocacy newsletter sparked your interest in advocacy, this month's will help it burn even brighter! Rare disease day provided a great opportunity to raise awareness of FA - let’s keep that momentum going throughout 2021! You will find information below on engaging advocacy events you can attend and important legislation that you can support. And, to ensure you have the tools you need to be most effective, FARA has launched a new advocacy page filled with information on current initiatives, as well as, guidance and resources to help you take action on the items that matter the most to you. Check it out!

Status Update of the FA Community Petition: Food and Drug Administration and Reata Pharmaceuticals: Allow Individuals with Friedreich's Ataxia Access to Omaveloxolone

FARA received a letter from the FDA Acting Director of the Center of Drug Evaluation and Research (CDER), Dr. Patrizia Cavazzoni, to acknowledge receipt of the FA Community's Petition. The letter indicates that the petition has been shared with appropriate FDA staff, that they appreciate the concerns and unmet medical need of the FA community, and notes CDER's commitment to flexibility in the evaluation of new product development, especially for rare and serious disease. In addition, Dr. Cavazzoni, mentions that, as required by law, FDA cannot provide details, feedback or comments on investigational new drug applications to anyone but the sponsor (e.g. drug company, Reata in this case). While the letter from the FDA did not provide additional information about the status of omaveloxolone, we are grateful that the agency acknowledged receipt of the petition and their commitment to consider the patient voice.  As reported earlier this month, Reata Pharmaceuticals also acknowledged the FA Community petition to advance regulatory approval of omaveloxolone in FA. In a subsequent press release, Reata shared additional data from the extension study that supports a disease modifying effect of omav and reported that they are reaching out to FDA to request a Type C meeting to continue dialogue on regulatory path forward. To read the full press release, click here. FARA will continue to work to ensure that the voices of the community are considered in all drug development programs. As additional information becomes available, FARA will keep the community informed.

COVID-19 Response

President Biden signed the American Rescue Plan into law on Thursday, March 11th! This law contains many important provisions to help our country through the COVID-19 crisis. Here are 5 changes the American Rescue Plan makes that matter to the rare disease community: Increases funding and support to help Americans afford private health insurance through healthcare.gov or their state marketplace, and provides protection from paying back excess subsidies due to income changes.  Provides 100% premium support for COBRA insurance through September 30 for individuals who have lost coverage due to involuntary layoffs or reduced hours.   Provides additional federal support for vital home-and community-based services that enable many rare diseases patients to live in their homes rather than moving to a facility for care. Expands state coverage to help uninsured Americans access COVID-19 vaccines and treatment and curb the spread of the virus. Provides new incentives for states to expand their Medicaid programs and provide health insurance to millions of low-income Americans.

Rare Disease Day

Rare Disease Day was a huge success thanks to the incredible actions of so many! Social media platforms were filled with posts, pictures, tweets and hashtags raising awareness and much needed attention on the 25-30 million Americans living with a rare disease, most without any approved treatments. Hundreds tuned in remotely for separate Rare Disease Day programs hosted by both the NIH and the FDA. Rare Across America, sponsored by the Everylife Foundation, resulted in 373 Congressional meetings with more than 650 advocates attending. And, the colors of Rare Disease Day lit up the night at many well known landmarks thanks to NORD.

A special moment occurred when Jacob Thompson shared his FA journey at Rare Disease Day at NIH 2021. Jacob Thompson won first place in the NCATS Rare Diseases Are Not Rare! 2020 Challenge with his moving piece "Keep on Fighting."   Deserving an encore performance in case you missed it last month, click the button below to hear Jacob's award winning piece.

Advocacy Training Webinars

RARE on the Road is a rare disease leadership series hosted by Global Genes and the EveryLife Foundation, providing critical education and insight into the rare disease community. Due to the COVID-19 pandemic, all 2021 events in this series will be virtual. Click here to learn more and to register.

The Living Rare, Living Stronger NORD Patient and Family Forum featuring the Rare Impact Awards is officially virtual for 2021! Join us for networking, education, inspiration, hope and fun this June. Be sure to take advantage of the special rate for patients, caregivers and NORD member representatives. Information on registration and scholarships can be found here.

Rare Disease Week on Capitol Hill 2021: Save the date for Rare Disease Week on Capitol Hill! The event will be July 19th – 22nd with hope we can all be together again in person this summer. Be on the lookout for more information coming soon! In the meantime, you can learn more about Rare Disease Week on Capitol Hill here.

Capitol Hill Updates

RISE Act, H.R.869/S.289: A $1.9 trillion relief package was signed into law by the President on March 11, 2021. The package includes some funds for research recovery at the National Science Foundation (NSF) and the National Institute of Standards and Technology (NIST), but not for any other research agency. The bipartisan, bicameral RISE Act (H.R.869/S.289) would authorize $25 billion to fill NIH and other research agency funding gaps. FARA signed onto Research!America’s letter to President Biden asking him to champion the RISE Act and include the associated funding in the next spending vehicle he advances.

Speeding Therapy Access Today Act of 2021 (STAT Act), H.R.1730/S.670 was introduced on March 10, 2021. The STAT Act, is a bipartisan bill that was created with the input of the rare disease community aimed at improving the development of and access to therapies for the rare disease community. The centerpiece of the STAT Act is the creation of a Rare Disease Center of Excellence at the US Food and Drug Administration. Championed by the Everylife Foundation, they just released a brand new website dedicated to the STAT Act to provide more detail and a call to action at www.everylifefoundation.org/stat-act/.

BENEFIT Act, S.373: Senators Roger Wicker (MS) and Amy Klobuchar (MN) introduced the Better Empowerment Now to Enhance Framework and Improve Treatments (BENEFIT) Act which would amend the Food, Drug and Cosmetic Act to ensure that the patient experience, patient-focused drug development (PFDD), and related data be considered as part of the risk-benefit assessment. The BENEFIT Act builds on provisions in 21st Century Cures that required the FDA to report on how patient experience data was used.

Senate Introduces Newborn Screening Reauthorization Act: The Newborn Screening Saves Lives Reauthorization Act, H.R. 482/S. 350 was introduced in the Senate by Senators Wicker (MS) and Hassan (NH) on February 19th, 2021. It had been previously introduced in the House on January 25, 2021. This Act would continue critical federal programs that aid states to improve and expand their newborn screening programs, support parent and provider education, and ensure laboratory quality and surveillance for newborn screening. Authorizations for these programs expired in 2019. Click here to contact your Members about this bill.

Announcements

FARA and NAF sent a letter of welcome to all Members of the 117th Congress highlighting that we look forward to working with them on legislative measures which will accelerate much needed treatments to our Ataxia communities.

EveryLife Foundation for Rare Diseases has published The National Economic Burden of Rare Diseases Study. This study is the first of its kind, providing the most comprehensive assessment of the total economic burden of rare diseases (RDs) in a single year. The National Burden of Rare Diseases Study estimated the economic cost of 379 rare diseases reached nearly $1 trillion in the U.S. in 2019. The results help to ensure that the experience of the rare disease community is reflected accurately in policy discussions. This powerful tool can also increase public awareness of the public health crisis of rare disease. For more information and the report findings, visit www.burdenstudy.org. View the Rare Disease Caucus briefing, “Economic Burden of Rare Diseases in America: A Public Health Crisis”, recording here.

The National Organization for Rare Diseases (NORD) has published a report, “Orphan Drugs in the United States: Rare Disease Innovation and Cost Trends Through 2019,” which examines the state of drug development for rare diseases. As US policymakers consider options to address rising health care costs while still meeting the needs of patients, this report found that rare diseases account for just 11% of medical invoice spending in the United States, and 79% of all orphan products treat only rare diseases. The report also highlights recent progress in drug development that has resulted in more people with rare diseases having access to critical treatments.

FDA: Orphan drug designation requests up in 2020: The FDA received 753 new requests for orphan drug designation last year, up 41% from 2019, and the agency completed review of all requests within 90 days. The agency approved 32 novel drugs and biologics with orphan drug status last year. There was also a significant jump in rare pediatric disease designation requests, which rose 330% from 2019 to 2020, for a total of 284 requests in 2020. “Orphan drug designation is an important incentive in rare disease product development and this growth will continue to support development of treatments for rare diseases. Given the ongoing increase in volume and complexity of designations, we continue to enhance and modernize our processes,” Acting FDA Commissioner Janet Woodcock, MD, and Director of the Office of Orphan Products Development Janet Maynard, MD, wrote in a recent FDA Voices article.

One Person’s Impact

In closing, FARA would like to congratulate FA Ambassador Shandra Trantham on being selected as one of fifteen young adults out of 45 applicants to participate in the Everylife Foundation YARR Leadership Academy. The YARR Leadership Academy is a series of six on-line courses on the roles and opportunities for patient representation in policy making, drug development and the regulatory process. Each course is three hours so Shanda will get 18 hours of advocacy training, culminating in a Capstone Project! In addition, she will receive a $1000 travel stipend to put her new skills to work during Rare Disease Week on Capitol Hill in July. Shanda has been an instrumental advocate for the FA community in countless ways, yet Shandra’s desire to enroll in YARR, to build on her talents, speaks volumes to the type of leader she strives to be. Well done, Shandra!

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