Earlier this year, hundreds of advocates representing every state gathered in Washington, DC for the Rare Disease Week Legislative Conference. Their energy and enthusiasm raised the roof, as they shared experiences and prepared to visit their legislators on Capitol Hill.
What a difference a pandemic makes. We replaced our daily routines with the mostly quieter reality of sheltering at home. When it comes to living with a rare disorder, however, there is no pause button. The needs and concerns of rare disease families and communities continue, and often grow. Access to healthcare providers and treatment facilities is more challenging for patients and caregivers alike.
As the world pauses for COVID-19, it is more important than ever to raise awareness, push for new legislation, and insist on continued investment in rare disease research. In short, we need to Fast Forward for RARE.
But don’t take our word for it. Listen to the advocates – strong and courageous people who live with rare disorders, care for family members, lead and serve patient communities, and honor the lives of angels. People with diverse experiences, including Tanya, Heather, Netanel, Lynn, Chris, Eddie, Felicia, Gabriel, and many more.
This year for the first time, SmithSolve partnered with Rare Disease Legislative Associates to introduce Fast Forward for RARE, a new video suite created to prepare rare disease advocates to tell their story with impact and inspire action. We shared lessons gained from leading the “practice your pitch” workshop at each of the last four legislative conferences. Advocates had the opportunity to practice their pitch on camera, receive personalized feedback, and apply what they learned on Capitol Hill and beyond.
Now, you can listen to their stories by visiting FastForwardforRARE.org. Hearing directly from rare disease advocates is a powerful experience that reminds us that living with a rare disease isn’t rare at all. While every person and every disorder is unique, collectively more than 350 million people in the rare disease community worldwide face common challenges.
Interested in joining the conversation? Register for Rare Across America, then enroll in the Fast Forward for RARE webinar taking place on Tues., July 21, 2020 at 3:00 p.m. ET. You’ll receive tips and coaching to help you share your story with impact and inspire action. Together we can advance important legislation, improve understanding of rare diseases, and support development of new treatments.
Fast Forward for RARE is sponsored by SmithSolve and Rare Disease Legislative Advocates. To learn more, visit www.FastForwardforRARE.com.
This summer, rare disease advocates across the country have the opportunity to meet with their federal legislators close to home in the state and district offices. You can make an impact on federal policy, share your rare disease story, and meet other rare disease advocates! Registration for Rare Across America is now open till July 3rd.
Rare Disease Legislative Advocates (RDLA) staff organizes meetings for rare disease advocates with their Members of Congress and/or the Member’s staff. The meetings take place in the Member’s district office during the month of August, while Congress is in recess from August 3rd to September 7th, 2020. The RDLA team also helps to prepare advocates for their meetings, providing legislative resource materials and hosting pre-meeting training webinars.
No prior advocacy experience is necessary.
Registration for this event and all RDLA events are free for rare disease advocates.
For more information, visit www.RareAcrossAmerica.org.
SAVE THE DATE: The Rare Across America training webinar will take place on July 9th at 2 pm EST and a special Social Media training webinar will take place on July 14th at 1:00 pm EST.
More than 900 patients and families from across the country attended Rare Disease Week on Capitol Hill 2020 from February 25th – 28th, an annual event hosted by the Rare Disease Legislative Advocates (RDLA). The Week featured events designed to educate and activate advocates and to foster relationships within the community. Advocates representing 227 patient organizations participated in 393 meetings with Members of Congress in one day.
“This year was by far our biggest Rare Disease Week on Capitol Hill. The event has grown each year since it started in 2012,” said EveryLife Foundation for Rare Diseases Executive Director Julia Jenkins. “We are witnessing a surge of engagement from the rare disease community. These advocates are energized and are demanding more treatment and diagnostic opportunities. They know it’s possible, and they can’t afford to wait.”
Thanks to their efforts, at least nine Members of Congress joined the Rare Disease Congressional Caucus, including: Rep. Emanuel Cleaver (MO-5), Rep. Antonio R. Delgado (NY-19), Senator Charles Grassley (IA), Rep. Dusty Johnson (SD-AL), Senator Doug Jones (D-AL), Rep. Bob Latta (OH-5), Rep. Ted Lieu (CA-33), Rep. Ralph Norman (SC-5), and Rep. Martha Roby (AL-2). These new Members bring the number of Rare Disease Congressional Caucus members to 170. In addition, various pieces of legislation impacting the community received increased support.
One in 10 Americans suffers from one of 7,000 known rare diseases. A disease is defined as rare when it affects fewer than 200,000. Ninety-three percent of the 7,000 known rare diseases have no U.S. Food and Drug Administration-approved therapies. Fifty percent of rare disease patients are children.
The RDLA is a program of the EveryLife Foundation for Rare Diseases designed to support the advocacy of all rare disease patients and organizations.
Rare Disease Legislative Advocates (RDLA), a program of the EveryLife Foundation for Rare Diseases, will bring together over 800 patients, caregivers, and others in Washington, DC for a week of events dedicated to empowering patients, families, friends, and healthcare professionals to become legislative advocates. During the week of February 25-28, 2020, rare disease advocates will have an opportunity to meet with Members of Congress and to learn about policy updates and best practices for successful advocacy.
Rare Disease Week on Capitol Hill 2020 kicks off on February 25th with a Rare Disease Congressional Caucus lunch briefing followed by a documentary screening and cocktail reception in the evening.
Rare disease advocates will attend the all-day Legislative Conference on February 26th to learn about federal legislation and policies that affect the rare disease community. Policy experts from Capitol Hill, non-profit organizations, and industry will share their expertise with advocates. In addition, advocacy professionals will provide opportunities for participants to refine techniques for effective advocacy on the Hill and building strong relationships with their Members of Congress.
On February 27th, a Hill Day breakfast will be held with keynote speakers. After breakfast, 500 rare disease advocates will go to Capitol Hill to meet with their Senators, Representatives, and Congressional staffers to discuss key legislation, policies and the Rare Disease Congressional Caucus. Later in the evening, the Rare Artist Reception will feature the 2019 Rare Artist contest winning artwork and highlight the importance of art as therapy for rare disease patients.
We will close out the week at the National Institutes of Health (NIH) in Bethesda, Maryland. On February 28th, the NIH will host Rare Disease Day at NIH. This event aims to raise awareness about rare diseases, the people they affect, and NIH research collaborations to advance new treatments. The EveryLife Foundation will be there to meet patients and provide information on their programs as well.
In addition, the Food and Drug Administration (FDA) has announced a Rare Disease Day public meeting at the FDA White Oak Campus in Silver Spring, MD on Monday, February 24th.
Registration for Rare Disease Week on Capitol Hill begins on January 3, 2020. Information on Rare Disease Week on Capitol Hill 2020, the week’s events and locations, travel and hotel accommodations, and registration for the event can be found at rareadvocates.org/rdw.
We are excited for this powerful week of events and to continue to advocate with the rare disease community.
(Washington, D.C., December 5, 2019) Rare Disease Legislative Advocates (RDLA), a program of the EveryLife Foundation for Rare Diseases (ELF), presented RareVoice Awards to individuals whose public policy advocacy leadership is saving lives and providing hope to millions of children and adults who live with rare diseases. RDLA is committed to growing the patient advocacy community and working collaboratively, thereby amplifying the patient voice to be heard by local, state, and federal policy makers.
“It is our mission to ensure the 30 million people living with a rare disease in the U.S. has access to early diagnosis and treatment,” said Julia Jenkins, Executive Director of ELF. “The individuals we honor tonight are courageous, inspirational change-agents who have demonstrated passion and dedication in the drive for federal and state legislation that helps make that vision a reality.”
At the event, held at Arena Stage in Washington, D.C., United States Senator Roger Wicker (R-MS) and Representative Lucille Roybal-Allard of California (D-40), were honored with the RareVoice Abbey Award for Congressional Leadership for their tireless efforts in support of the rare disease community.
Dr. Emil Kakkis, MD, PhD, President & CEO of Ultragenyx, geneticist, ELF Founder and current board member, was presented with the Lifetime Achievement Award for dedicating his life to the development of novel life-saving drug treatments for rare diseases, most notably, the creation of Aldurazyme to treat patients with mucopolysaccharidosis (MPS 1), a rare genetic disorder that affects many body systems and that leads to organ damage.
A highlight of the event was the presentation of the Teen Advocacy RareVoice Award to Aidan Abbott. Abbott, a 14-year old from Slinger, Wisconsin, was born with ectodermal dysplasias (ED), a rare congenital disease that affects hair, skin and sweat glands. Some patients with ED are born without many or all of their teeth and suffer collapsed jaws and facial deformation.
After struggling to obtain health insurance coverage for his missing teeth, Aidan worked with Senator Tammy Baldwin (D-WI) to write and name the Ensuring Lasting Smiles Act, legislation that would ensure health plans cover medically necessary services related to a patient’s anomaly or birth defect, including any serious dental and oral-related procedures that are necessary to maintain health and overall function.
Via her video salute to Aidan at the event, Senator Baldwin said, “Aidan never stops fighting for families like his. Your story continues to inspire me, and I can’t wait to see what we accomplish together.”
When presented with his award, Aidan told attendees, “Never stop fighting for what you know is right. I won’t stop fighting until the Ensuring Lasting Smiles Act becomes the law of the land because it will make a huge difference for me and others who have ectodermal dysplasias and congenital anomalies.”
Additional awards were presented to the following outstanding advocates:
• Isabel Bueso, a native of Guatemala, was presented with the Federal Advocacy Award. Isabel came to the U.S. to participate in a clinical trial for mucopolysaccharidosis type VI (MPS VI). For six years she has become an advocate for rare disease issues, including newborn screening, NIH funding and orphan drug designation. Isabel is awaiting notification of her medical deferred action status, since the program was reinstated this fall.
“Isabel Bueso is a remarkable soul,” said California Congressman Mark DeSaulnier (D-11). “Whether advocating for students with disabilities or fighting to save herself and her family from deportation, Isabel has demonstrated time and again that she is brave and strong. In the face of adversity, I have come to admire Isabel for her courage to use her voice to help others and her vibrant and positive attitude. I cannot think of a more deserving person for this honor.”
• Nikia Vaughan, of Baltimore, Maryland, the mother of daughter Cimone, seven, who was diagnosed with sickle cell disease in 2012, was presented the State Advocacy Award for her effort to pass State Bill 161 in 2018. The landmark legislation put into place new mandatory healthcare services for children with sickle cell in Maryland schools.
Maryland Representative Dutch Ruppersberger (MD-2), Nikia’s Congressman, congratulated her by saying, “Maryland’s Second District is home to many amazing community leaders and Nikia Vaughan is no exception. Nikia has been a passionate advocate for sickle cell awareness on behalf of her daughter, Cimone, and even helped pass legislation establishing new guidelines for providing services to students with sickle cell in our public schools. It is an honor to congratulate Nikia on her nomination for a RareVoice award and thank her for her tireless work on behalf of the entire rare disease community.”
• Debbie Jessup, Health Policy Advisor to Representative Roybal-Allard (CA-40), and Shayne Woods, Senior Policy Advisor to Representative Gus Bilirakis of Florida (R-12) were presented with awards in the Congressional Staff category.
Mike Porath, founder and CEO of The Mighty, the leading digital health community, served as masters of ceremonies for the event, while Tilea West, the 2019 Cherry Blossom Queen and rare disease advocate, presented the awards.
The RareVoice Awards is an annual celebration, now in its eighth year, to honor advocates who give rare disease patients a voice on Capitol Hill and in state government. Patient advocates, industry executives, and Congressional and government agency staff gather to honor these outstanding advocates in the rare disease community.
The Rare Disease Legislative Advocates (RDLA) today announced the 2019 RareVoice Awards patient advocate finalists. View the finalists.
The RareVoice Awards is an annual celebration, now in its eighth year, to honor advocates who give rare disease patients a voice on Capitol Hill and in state government. Patient advocates, industry executives, and Congressional and government agency staff gather to honor these outstanding advocates in the rare disease community. Award recipients are chosen by committee from nominations received from the rare disease community. Awardees receive an “Abbey” statuette commissioned specially for the RareVoice Awards, and named for Abbey Meyers, founder of the National Organization for Rare Disorders (NORD).
The honoree in each category as well as the recipients of the Lifetime Achievement and Congressional Leadership Awards will be announced at the 2019 RareVoice Awards on December 4th in Washington, D.C. The event is free and open to the public. Register today.
Thank you to the nearly 600 rare disease advocates who participated in meetings with their federal legislators during the August recess this summer! There were 303 meetings with Members of Congress or their staff in 49 states plus the District of Columbia. This is our highest turnout yet for our in-district meetings program, Rare Across America!
Rare disease advocates from around the country spoke to their legislative officials and their staff on important rare disease issues. Due to the advocates meetings and hard work, we saw the following results!
A Rare Disease Burden Study included in the Senate Appropriations Committee Bill: The Senate Appropriations Committee included language to direct the GAO to study what is known about the total impact rare diseases have on the U.S. economy, including direct medical costs, non-medical costs, loss of income, and the societal consequence of undiagnosed and untreated rare disease. Once passed into the law, the GAO will provide a report within two years.
Six New Cosponsors of the Newborn Screening Saves Lives Reauthorization Act, H.R. 2507/S. 2158: Reauthorize critical existing federal programs that provide assistance to states to improve and expand their newborn screening programs, support parent and provider education, ensure laboratory quality and effective surveillance. More info here.
New cosponsors: Senators Tammy Baldwin (WI), Robert Casey (PA), Amy Klobuchar (MI), Martha McSally (AZ), Krysten Sinema (AZ), and Debbie Stabenow (MI)
12 New Members of the Rare Disease Congressional Caucus. The caucus now has 132 members in the U.S. House of Representatives and 22 in the Senate. Click here to invite your legislators to join the bipartisan Caucus.
New Members: Representatives Sean Casten (IL), Steve Chabot (OH), James Comer (KY), Jason Crow (CO), Kevin Hern (OK), Chris Pappas (NH), Max Rose (NY), Darren Soto (FL) and Senators Edward Markey (MA), Gary Peters (MI), Debbie Stabenow (MI), and Tina Smith (MN)
25 New Cosponsors of the Lymphedema Treatment Act, H.R. 1948/S. 518: Proposes to provide Medicare coverage of lymphedema compression treatment items.
New cosponsors: Senators Lindsey Graham (SC), Maggie Hassan (NH), Rick Scott (FL) and Representatives Anthony Brown (MD), Ken Calvert (CA), Salud Carbajal (CA), Judy Chu (CA), Henry Cuellar (TX), Elijah Cummings (MD), John Curtis (UT), Sharice Davids (KS), Dwight Evans (PA), Jesus Garcia (IL), Josh Gottheimer (NJ), John Larson (CT), Donald McEachin (VA), Donald Payne (NJ), Michael Simpson (ID), Elissa Slotkins (MI), Bryan Steil (WI), Dina Titus (NV), David Trone (MD), Lauren Underwood (IL), Maxine Waters (CA), Randy Weber (TX)
53 New Cosponsors of the Ensuring Lasting Smiles Act, H.R. 1379/S. 560: Proposes to require private health insurance to provide coverage of medically necessary treatments of congenital anomolies including dental services.
New cosponsors: Senators Benjamin Cardin (MD), Steve Daines (MT), Kamala Harris (CA), Doug Jones (AL), Martha McSally (AZ), Pat Roberts (KS) and Representatives Jahana Hayes (CT), Darin LaHood (IL), Michael Guest (MS), Suzanna Bonamici (OR), Trent Kelly (MS), Paul Cook (CA), William Keating (MA), Ro Khanna (CA), Stephen Lynch (MA), Nita Lowey (NY), Doug Lamborn (CO), John Larson (CT), Grace Napolitano (CA), Donald Norcross (NJ), Adriano Espaillat (NY), John Sarbanes (MD), Rashida Tlaib (MI), Scott Peters (CA), Mikie Sherrill (NJ), Suzan DelBene (WA), Adam Schiff (CA), Bradley Schneider (IL), Dwight Evans (PA), Lori Trahan (MA), Tom Cole (OK), David Price (NC), Frank Lucas (OK), Katherine Clark (MA), Mark Meadows (NC), Daniel Lipinski (IL), Madeleine Dean (PA), Antonio Delgado (NY), Sharice Davids (KS), Michael Waltz (FL), Andy Harris (MD), Cathy McMorris Rodgers (WA), Zoe Lofgren (CA), Dina Titus (NV), Cedric Richmond (LA), Theodore Deutch (FL), Jim Banks (IN), Doug Collins (GA), Cheri Bustos (IL), Ross Spano (FL), Mike Bost (IL), and James Comer (KY), Jerry McNerney (CA)
8 New Cosponsors of the Medical Nutrition Equity Act, H.R. 2501: Proposes to require private health insurance, Medicare, and Medicaid to provide coverage of medically necessary food for digestive and inherited metabolic disorders.
New cosponsors: Representatives Earl Blumenauer (OR), Rick Larsen (WA), John Moolenaar (MI), Joe Neguse (CO), Dean Phillips (MN), John Rutherford (FL), Kim Schrier (WA), Chris Stewart (UT)
Thank you again to all the advocates who participated in Rare Across America meetings this summer and continue to build relationships with Members of Congress. If you have not already, please take the time to follow up on your meetings by emailing the staffer you met with and thank them for the meeting and repeat your ask.
Please save the date for Rare Disease Week on Capitol Hill on February 25th to 28th, 2020 in Washington, DC. Rare Disease Week attracts hundreds of patient advocates from around the country to Washington, DC for a week of events dedicated to empowering patients, families, and friends to become legislative advocates. Advocates will have an opportunity to meet with Members of Congress and learn best practices for successful advocacy. No advocacy experience required. The travel stipend application for Rare Disease Week is open. Deadline to apply for a travel stipend is December 2, 2019. You can apply here. Registration for Rare Disease Week on Capitol Hill opens on January 3, 2020 at https://rareadvocates.org/rdw/.
We are excited to welcome rare disease advocates from across the country to Rare Disease Week on Capitol Hill 2020 in Washington, DC from February 25-28, 2020! Rare Disease Week on Capitol Hill brings rare disease community members together to learn about federal legislative issues, meet other advocates, and share their rare stories with legislators.
The travel stipend application for Rare Disease Week on Capitol Hill 2020 is now open! Please find the application here.
In order to enable more advocates to attend, the EveryLife Foundation will offer a limited number of travel stipends. Stipend awardees in Maryland and Virginia will receive $400; awardees in Alaska, Hawaii, North Dakota, and Puerto Rico will receive $1000; and awardees from the other 45 states will receive $800.
Stipend recipients are required to attend the Legislative Conference on February 26th and the Hill Day on February 27th but may attend the other events as well. Only one person from a family can be awarded a travel stipend.
The Application deadline to apply for a Rare Disease Week on Capitol Hill 2020 Travel Stipend is December 2nd, 2019. All advocates that apply will be notified by December 20th, 2019.
*Any young adult under the age of 18 who receives a travel stipend must travel with an adult chaperone.
For more information on the events during Rare Disease Week on Capitol Hill 2020, please visit www.rareadvocates.org/rdw.
The RareVoice Awards nominations for 2019 are open! We encourage the community to nominate individuals and organizations who have gone above and beyond to become rare disease policy leaders, having passed or supported legislation for the rare disease community at the state and federal level. There is even a category for teenagers/young adults who have made a big impact.
The RareVoice Awards nominations will close on Friday, August 30th. Please submit your nominees for the RareVoice Awards at www.rareadvocates.org/rarevoice-awards.
The RareVoice Awards celebration will take place on December 4, 2019 to honor advocates who give rare disease patients a voice on Capitol Hill. Patient advocates, industry executives, and Congressional and government agency staff gather to honor these outstanding advocates for the rare disease community. 2019 will mark the 8th year of the RareVoice Awards, which will be held at the Arena Stage in Washington, DC.
Award recipients are chosen by committee from nominations received from the rare disease community. Awardees receive an “Abbey” statuette commissioned specially for the RareVoice Awards, and named for Abbey Meyers, founder of the National Organization for Rare Disorders (NORD). This is a free and widely attended event.
If you have any questions about the RareVoice Awards, please contact Shannon at firstname.lastname@example.org.