Rare Disease Legislative Advocates Honor Leaders Who Inspire Transformational Public Policy Solutions

2019 RareVoice Awardees (l to r): Nikia Vaughan, State Advocacy; Debbie Jessup, Congressional Staff; Shayne Woods, Congressional Staff; Isabel Bueso, Federal Advocacy; Dr. Emil Kakkis, Lifetime Achievement; Aidan Abbott, Teen Advocacy

(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.

2019 RareVoice Awards Finalists Announced

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.

Congratulations Advocates on Over 300 Rare Across America Meetings!

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!

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/.

Travel Stipend Application Open for Rare Disease Week on Capitol Hill 2020

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.

RareVoice Awards Nominations Closing Soon

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 svonfelden@everylifefoundation.org.

RDLA Announces Rebrand of In-District Lobby Days to Rare Across America

Rebrand reflects program’s evolution and commitment to the patient voice

The Rare Disease Legislative Advocates (RDLA), a program of the EveryLife Foundation for Rare Diseases, today announced the rebranding of its In-District Lobby Days program, which will now be called Rare Across America. The rebrand reflects the program’s evolution and reinforces the RDLA’s commitment to providing resources and opportunities for rare disease advocates, thereby impacting public policy. In addition, the refresh better positions the program as an extension of Rare Disease Week on Capitol Hill, an annual legislative advocacy event attracting more than 800 advocates to the nation’s capital.

“Changing public policy is a year-round commitment. We must keep the momentum going,” says Julia Jenkins, the EveryLife Foundation for Rare Diseases Executive Director. “Rare Across America is a great way to connect patients and their care givers to Members of Congress in their home districts, where they can build powerful relationships.”

In addition to a new name and logo, the program has adopted a new tagline, “Every voice, in every district, matters,” underscoring the power of patient engagement.

Under the Rare Across America program, 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 July 29th to September 8th, 2019. 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.

Advocates interested in participating should register between May 8th and July 3rd at www.RareAcrossAmerica.org.

The first training webinar for registered advocates, entitled “What to Expect at Your Meetings,” will be hosted on July 25th.

About Rare Disease Legislative Advocates
The Rare Disease Legislative Advocates (RDLA) is a program of the EveryLife Foundation for Rare Diseases designed to support the advocacy of all rare disease patients and organizations. The 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.

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Biggest Rare Disease Week on Capitol Hill Ever!

Thank you to the more than 800 rare disease advocates who traveled from 49 states plus the District of Columbia to join us during Rare Disease Week on Capitol Hill 2019, which took place February 24th – 28th! Four-hundred and fifty of these advocates participated in 298 meetings with Members of Congress to bring our message: […]

Five Ways YOU Can Participate in Rare Disease Week on Capitol Hill Remotely

We are excited so many advocates will be joining us in DC for Rare Disease Week on Capitol Hill. For those who are not able to travel to Washington, DC, there are a number of ways to participate from your own home! Please feel free to share with your family and friends too! Below are five ways you can make your voice heard during the week’s events.

Monday: Watch the livestream of the Legislative Conference on Monday, February 25th to learn about key legislation affecting the rare disease community. The draft agenda is available here, and you can register for the free livestream here.

Tuesday: Call or email your Members of Congress on Lobby Day on Tuesday, February 26th. The one-page background papers for each of the key legislative issues discussed at the Legislative Conference will be available here prior to Rare Disease Week. You can review them and decide which you might want to ask your Members of Congress to support. You can find more information on calling and emailing your Senators and Representative here.

Wednesday: On Wednesday, February 27th, join our Rare Disease Congressional Caucus briefing from 2:00 pm – 3:00 pm EST. Titled “Rare Disease 101”, this briefing will provide insights on rare diseases and its’ impact on rare disease patients and their families. Register for the free livestream here.

Thursday: Watch the livestream of Rare Disease Day at the National Institutes of Health (NIH) on Thursday, February 28th. The agenda and link to register are available on the NIH website. Speakers include leaders from NIH and the Food and Drug Administration (FDA), as well as representatives from a number of patient advocacy groups.

Social media: Stay engaged with our acitivites by connecting with us on Facebook, Twitter and Instagram. On Twitter, we are @RareAdvocates; on Instagram @Rare_Advocates. For posts related to Rare Disease Day or your own advocacy efforts, be sure to use our event hashtag, #RareDC2019, and to tag RDLA in your posts.

We hope that you will take part in these remote participation opportunities! If you have questions regarding any of these events, please email Shannon von Felden at svonfelden@everylifefoundation.org.

Top 5 Reasons Why YOU Should Join Us for Rare Disease Week on Capitol Hill 2019!

1.) Learn about policy issues that impact you at the Legislative Conference.

2.) Share your unique story with members of Congress during Lobby Day, which is kicked off at our Lobby Day Breakfast.

Read more

Get Ready for Rare Disease Week on Capitol Hill 2019

Rare Disease Legislative Advocates (RDLA) will bring together over 500 patient advocates 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 24-28, 2019, advocates will have an opportunity to meet with Members of Congress and learn best practices for successful advocacy.

Rare Disease Week on Capitol Hill 2019 kicks off on February 24th with a documentary screening and cocktail reception.

Patient advocates will attend the all-day Legislative Conference on February 25th to learn about federal legislation and policies that affect the rare disease community. Policy experts from Capitol Hill and non-profit organizations will share their expertise with advocates. In addition, advocacy professionals will teach attendees how to be effective advocates on the Hill and build strong relationships with their Members of Congress.

On February 26th, a Lobby 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.

The next day, the Rare Disease Congressional Caucus will host rare disease advocates and Congressional staffers for a lunch briefing on February 27th. Later in the evening, the Rare Artist Reception will feature the 2018 Rare Artist contest winning artwork and highlight the importance of art as therapy for rare disease patients.

We will close out the week at 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.

RDLA offers travel stipends to participants to offset the cost of attending Rare Disease Week on Capitol Hill. Stipend awardees in Maryland and Virginia will receive $400, and awardees in the rest of the continental United States will receive $800. Stipend awardees in Alaska, Hawaii, and Puerto Rico will receive $1000.

Two stipends will be awarded per state and limited to one per family. Stipends are prioritized to have a diverse representation of rare diseases and for those who have not attended Rare Disease Week or received a stipend in the past. The 2019 travel stipend application is open until December 14th, 2018 and can be found at rareadvocates.org/rdw. Applicants will hear by December 21st whether they will receive a travel stipend or have been placed on the waiting list.

Registration for Rare Disease Week on Capitol Hill begins on January 3, 2019. A hotel room block has been organized at the Washington Court Hotel in Washington, DC between February 24th and 28th for $219 per night. Attendees can contact the Washington Court Hotel directly and make a reservation with the “EveryLife Foundation” room block to reserve with this rate.

Information on Rare Disease Week on Capitol Hill 2019, the travel stipend application, 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.