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

We know that traveling to Washington, DC isn’t on option for all advocates. In order to better serve the rare disease community, we’ve provided a number of ways for advocates to participate in Rare Disease Week on Capitol Hill remotely. Below are five ways you can make your voice heard during next week’s events.

  • Monday: Watch the livestream of the Legislative Conference on Monday, February 26th 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 your Members of Congress on Lobby Day on Tuesday, February 27th. 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 contact information for your elected officials here.
  • Wednesday: On Wednesday, February 28th, join our Rare Disease Congressional Caucus briefing via livestream from 12:30 – 1:45 PM EST.  Titled “The Rare Disease Lifecycle: Diagnosis to Treatment”, this briefing will provide insights on a number of steps in a rare disease patient’s journey.
  • Thursday: Watch the livestream of Rare Disease Day at the National Institutes of Health (NIH) on Thursday, March 1st. 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 FacebookTwitter and Instagram. On Twitter, we’re @RareAdvocates; On Instagram, we’re @Rare_Advocates. For posts related to Rare Disease Day or your own advocacy efforts, be sure to use our event hashtag, #RareDC2018, 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 Grant Kerber at gkerber@everylifefoundation.org.

Legislative Conference livestream presented by

 

Thank you to our 2018 sponsors.

Youth Advocacy during Rare Disease Week on Capitol Hill

 As our Foundation continues to build its youth advocacy programming, we are pleased to offer a dedicated youth advocacy track for our 2018 Legislative Conference.  This programming track echoes our belief that it is important for young adults to have their own voice when advocating for issues affecting the rare disease community, and aims to grow that voice to build confidence, develop political leadership skills and raise awareness.

The age range for our youth advocacy program is 13-25 yrs old.  We strongly encourage event participants to be within this age range, but all are welcome.

Young adult events during RDWCH 2018


Monday, February 26th, 2018

Legislative Conference
Ronald Reagan Building and International Trade Center, Atrium Ballroom
1300 Pennsylvania Ave NW, Washington, DC 20004
 Young Adults Track (2 sessions)

• Track C: Advocacy for Young Adults: Your Voice Matters

First, learn a little bit about how our government works. Then, meet and hear from some rare disease young adults who have been making a mark on Capitol Hill by telling their stories.

• Track C: Advocacy for Young Adults: Practice Meetings with Congressional Staff

Young adults can practice telling their stories to Congressional staffers. Attendees can get some tips and tricks for successful meetings on the Hill from those who know best.


Tuesday, February 27th, 2018

Young Adults Meet-Up (a debrief of your experiences on the Hill that day)
Songbyrd Music House and Record Café
2475 18 St NW, Washington, DC 20009


Wednesday, February 28th, 2018

Rare Artist Reception
Russell Senate Office Building Room 325
2 Constitution Ave NE, Washington, DC 20002

Award recipients from the 2017 Rare Artist contest will speak at the Rare Artist Reception, taking place on Wednesday, February 28th in Room 325 of the Russell Senate Office Building.  Awardees from the Rare Artist contest’s Teen category will be present to speak about their work and the impact of rare disease on their lives.  This is a chance to network with other young adults and view the artwork of the rare disease community.  For more information on the reception and to register, click here.


For those who have already registered and are looking for other ways to prepare for Rare Disease Week on Capitol Hill, here are some tips:

  • Tune into our webinar on Thursday, Feb. 15th. To register click here.
  • Watch this video on “Tips on how to dress for Lobby Day”. You will hear directly from two of our speakers for the young adults track, Shira Strongin and Taylor Kane.
  • If you need a letter from the EveryLife Foundation to excuse absence from school, please email our youth advocacy program director, Lindsey Cundiff at lcundiff@everylifefoundation.org.
  • If you are interested in posting on social media throughout Rare Disease Week on Capitol Hill, keep these handles in mind.

Any other questions/concerns about the Youth Advocacy during Rare Disease Week on Capitol Hill, please email Lindsey Cundiff at lcundiff@everylifefoundation.org.

Social Media Info flyer design: Shira Strongin.

CHIP Funding Extended for Six Years

Earlier this month, Congress passed a Continuing Resolution (CR) extending Children’s Health Insurance Program (CHIP) funding for six years. The program provides affordable, comprehensive health insurance for children in families with too much income to qualify for Medicaid, but struggle to afford private insurance. Some states had to temporarily close enrollment or post notices about coverage loss after CHIP expired on September 30, 2017.

Joan Alker analyzed the issue in a Georgetown University Health Policy Institute piece.

President Trump signed the CR into law earlier today. Energy and Commerce Committee Chairman Greg Walden (R-OR) and Health Subcommittee Chairman Michael C. Brugess (R-TX) stated that, “today is a win for the millions of kids and their families that rely on CHIP and its vital services. After a completely unnecessary and avoidable shutdown, families across the country can breathe a sigh of relief knowing they will continue having access to affordable, quality health insurance.”

Scenes from a Successful RareVoice Awards Event

Thank you to all who attended and supported our sixth annual RareVoice Awards. You helped us celebrate rare disease advocates who made an impact at state and federal levels. Enjoy photos from the event on our Facebook page and plan to join us February 25 through March 1, 2018 for Rare Disease Week on Capitol Hill.

Congratulations to our 2017 award recipients:

Senator Amy Klobuchar
Congressional Leadership RareVoice Award

Representative Leonard Lance
Congressional Leadership RareVoice Award

Senator Patty Murray
Congressional Leadership RareVoice Award

Remy Brim, Senate HELP Committee, Ranking Member Patty Murray
Congressional Staff RareVoice Award

Polly Webster, Office of Representative Diana DeGette
Congressional Staff RareVoice Award

Paul Fogelberg, Patient Advocate and Director, Pulmonary Fibrosis Advocates
Federal Patient Advocacy RareVoice Award

Emily Muller, Founder, Emily’s Fight
Federal Teen Advocacy RareVoice Award

Shira Strongin, Founder, Sick Chicks
Federal Teen Advocacy RareVoice Award

Becky Ruth, Member, Missouri State House of Representatives
State Legislator RareVoice Award

Darlene Shelton, Founder and President, Danny’s Dose
State Patient Advocacy RareVoice Award

Taylor Kane, Lead Advocate, Run for ALD
State Teen Advocacy RareVoice Award

 

Thank you to our sponsors for making this event possible!

21st Century Cures Update: One Year Later

On November 30th, the House Energy and Commerce Committee held a hearing to discuss, “Implementing the 21st Century Cures Act: An Update from FDA and NIH.” A year after 21st Century Cures Act was signed into law, Members of Congress asked Francis Collins, Director, National Institutes of Health (NIH) and Scott Gottlieb, Commissioner, Food and Drug Administration (FDA) to provide updates.

Representative Burgess (R-TX) opened the hearing by commending the bipartisan effort required to pass 21st Century Cures. He then went on to highlight key provisions like the Precision Medicine Initiative, Cancer Moonshot, the FDA Innovation Account, and modern clinical trial designs.

Scott Gottlieb emphasized that the FDA is currently examining how to leverage the agency’s accelerated approval pathway. Accelerated approval would include the authority to require confirmatory evidence to support continued marketing efforts and an expedited withdrawal mechanism if a treatment fails to show benefit. To fully leverage these opportunities, cancer drugs already approved for one indication could be approved for a supplemental application if evidence demonstrates. When Representative Matsui (D-CA) asked for updates on rare disease provisions in Cures, Dr. Gottlieb re-emphasized that the accelerated approval mechanism should be applied to rare disease trials when there is clear clinical benefit at an early stage. The FDA is already making robust use of provisions that allow, especially in the oncology setting, the agency to create supplemental indications without having to do additional clinical trials. This is especially relevant because, as Rep. Matsui pointed out, many rare disease patients take drugs off-label.

Click here to read the press release from the Energy and Commerce Committee.

The Senate Health, Education, Labor and Pensions (HELP) Committee will hold a hearing on the same topic on Thursday, December 7th.

FasterCures 21st Century Cures Tracker 

Congress Mulls Healthcare Stabilization Efforts

After President Trump announced his intention to scrap health insurance marketplace subsidies via executive order earlier this month, Senators Patty Murray (D-WA) and Lamar Alexander (R-TN) introduced a bipartisan bill entitled, “the Health Care Stabilization Act of 2017” in an attempt to stabilize the marketplace. Insurer subsidies reimburse health insurance companies that reduce cost-sharing for lower-income Affordable Care Act (ACA) marketplace enrollees. Many critics of the executive order state that without insurer subsidies, millions of individuals would not be able to afford coverage. The bipartisan bill, which has 24 co-sponsors (12 Republican and 12 Democrat), would give states more funding flexibility to establish insurance stability funds and would provide expedited approval of Section 1332 state innovation waivers.

The Congressional Budget Office estimates that the bill could reduce the deficit by $3.8 million over the next 10 years. The CBO report also states that appropriating funds for cost-sharing reductions (CSRs), which the bill requires, would not affect direct spending or revenues relative to the current baseline projections.

In response to the Murray/Alexander legislation, Senate Finance Committee Chairman Orrin Hatch (R-UT) and House Ways and Means Committee Chairman Kevin Brady (R-TX) have announced that they will release legislation that extends the CSR program for two years, but repeals ACA mandates. The Ways and Means Committee has issued a press release.

Meet 2017’s RareVoice Nominees: State Advocacy by a Patient Advocate

This week, we continue to introduce you to advocates nominated for RDLA’s 6th annual RareVoice Awards. The patient activists nominated in the “State Advocacy by a Patient Advocate” category worked to advance state policies that are beneficial for the rare disease community.

Raymond Brooks has worked with West Virginia Senate and House members and Governor Jim Justice to advocate for rare disease patients. He helped get Senate Bill(SB) 339 passed to establish the Commission on Chronic Pain Management. Once passed by the Senate, he worked with Governor Justice to sign SB339 into law. Raymond also worked with Governor Justice to write a proclamation making July 2017 in observation of Arachnoiditis Awareness Month. This is the first time in history that West Virginia proclaimed any period of observance for this rare and painful chronic disease.

 

 

 

Angela Ramirez Holmes is the President of the California Action Link for Rare Diseases (CAL Rare), a non-profit dedicated to providing a common voice for rare disease patients in California. She started her advocacy in rare diseases when her then eight year-old son Andrew was diagnosed with a rare brain condition, arteriovenous malformation (AVM). In the short time since, she and her family have raised over $10,000 for The Aneurysm and AVM Foundation and collected hundreds of toys for UCSF Benioff Children’s Hospital Oakland. In 2017, Angela launched CAL Rare to bring together stakeholders to raise awareness among the general public and decision-makers regarding rare diseases.

 

 

 

Barby Ingle advocates for all challenged with a chronic condition involving chronic pain and rare disease. She does this for patients like her, as well as their families, caregivers, healthcare professionals and the public. Through her activities for International Pain Foundation, Consumer Pain Advocacy Task Force (CPATF), State Pain Policy Advocacy Network (SPPAN), RDLA and the 16 other coalitions she partners with, she is fighting daily for patients’ right to proper and timely access to care, open access to treatment, and bio-psycho-social advances in care, in-person and online. Barby advocates for chronic awareness to help the invisible become visible so that patients can learn how to participate fully in society.

 

 

Anna Payne is a 30-year-old person with cystic fibrosis (CF) who is working on accomplishing her dreams every day. Her passion is for helping to raise awareness and support for those with rare diseases, and her favorite activity is advocacy work. In the past year, she has done her best to voice her concerns and raise awareness for the CF community. She sat down with her local Congressman and Senator, and traveled to the state capitol to speak with various legislators. She has been featured in her newspaper, and an article on philly.com. She does all of this while working full time at a local credit union. She believes that the only way to make a difference is to get involved, to stand up, and raise her voice.

 

 

 

After her first grandson was unexpectedly diagnosed with severe hemophilia B, Darlene Shelton discovered protocols prohibiting paramedics/emergency room (ER) doctors from administering medications carried by rare disease patients. Realizing the danger, she stepped into action creating the Danny’s Dose Alliance and embarked on a journey to spur change. Starting in Missouri working with the EMS Association, Darlene realized the problem pertains to all patients with special medical needs for treatment or medications. The problem appeared to be conflicting protocols, fear of liability and education. The organization’s three main goals became changing protocols, assisting with paramedic/ER education and providing families with education. Danny’s Dose successfully passed Emergency Medical Service (EMS) Protocol legislation in Missouri within two years and is currently assisting with education.

 

Join the celebration to recognize advocates like these on November 15th in Washington, DC! Free registration for the RareVoice Awards is available here.

Meet 2017’s RareVoice Nominees: Federal Advocacy by a Patient Advocate

This week we are introducing you to the advocates nominated for RareVoice Awards in the “Federal Advocacy: Patient Advocate,” category. These individuals not only fought for legislation that helps rare disease patients, but also raised awareness about policy that impacts them within the community.

Lana Clay is a 26-year survivor of leukemia and a doctoral student in Applied Life Sciences who also holds Masters degrees in Public Health Professions Education, Community Health Development and Business Science. She has committed the last 10 years of her life to advocacy and serves as an advocate for the Leukemia and Lymphoma Society. As the creator of the non-profit Childhood Leukemia Survivors (CLS), Lana advocates for various rare diseases. Her goals are to help accelerate therapies, limit barriers and create pathways to affordable care for all cancer patients and survivors.

 

 

Heather Ferguson is the Founder and Executive Director of the Lymphedema Advocacy Group. Her son Dylan was born in 2006 with primary lymphedema. After a prolonged struggle to get a proper diagnosis for Dylan, she was frustrated to learn that the compression garments and supplies he would need to manage this chronic condition were not covered by insurance. In 2009, Heather began working with her state representative and succeeded in passing the North Carolina Lymphedema Diagnosis and Treatment Act. Heather then turned her efforts to improving coverage nationwide with the introduction of the Lymphedema Treatment Act (HR930/S497).

 

 

 

Paul Fogelberg is a pulmonary fibrosis (PF) patient. His 2004 diagnosis predicted his death within five years. Despite those odds, Paul went to work as a volunteer PF advocate. Supported by his wife and three daughters, Paul took his advocacy work to a new level in 2010 by founding an advocacy non-profit, the Pulmonary Fibrosis Advocates (PFA). While federal funding for PF research was ‘flat’ from 2000-2009, the PFA’s advocacy resulted in an increase in PF research funding of $106.1 million since 2010. In addition, the number of PF research grants has doubled from 68 in 2010 to 136 in 2017.

 

 

 

Diagnosed with Klippel-Feil Syndrome (KFS) eight years ago, Sharon Rose Nissley struggled to find information, knowledge, support, care and treatment options for this painful rare musculoskeletal condition that affects the development of the cervical spine and organs. When Sharon’s career as Director of Interior Design was sidelined, she was motivated to find solutions. She developed a network of patients and families internationally which provides resources and support to improve the lives of those affected by KFS and empowers patients to advocate for their needs. Sharon worked with Genetic Alliance to update KFS information for the NIH National Library of Medicine. She actively built support for the 21st Century Cures Act by using her graphic arts abilities on social media, a powerful platform for patients. Sharon now lobbies Congress to pass the Orphan Product Extensions Now, Accelerating Cures and Treatment (OPEN ACT) to bring forth treatments for those with rare disease.

 

 

Join the celebration to recognize advocates like these on November 15th in Washington, DC! Free registration for the RareVoice Awards is available here.

Travel Stipend Applications Open for Rare Disease Week on Capitol Hill 2018

We are excited to host patients, caregivers, physicians and other advocates from across the country at Rare Disease Week on Capitol Hill, which will take place from February 25th through March 1st, 2018. There is much work to do to educate members of the U.S. House of Representatives and Senate on the impact of rare disease on patients and families in their districts.

The EveryLife Foundation for Rare Diseases will provide travel stipends through its Rare Giving program to enable advocates from across the country to participate. In order to receive a stipend, advocates must attend the Legislative Conference and Lobby Day. Stipends are $400 for Maryland and Virginia, $800 for the rest of the continental U.S., and $1,000 for Alaska, Hawaii and Puerto Rico. They are limited to one per family.

Applications are available online through December 10th, and all applicants will be notified by December 18th whether they will receive a stipend or are on the waitlist. This year, close to $75,000 in stipends was awarded to 95 advocates from 48 states!

The schedule of events, details on the hotel room block and additional information is available on the Rare Disease Week on Capitol Hill webpage. Registration will open on January 3, 2018.

Funding for CHIP Has Expired

Congress has missed the September 30 deadline to renew funding for the Children’s Health Insurance Program (CHIP). CHIP is essential for families who do not qualify for Medicaid, but struggle to afford private insurance. A majority of states will deplete their CHIP dollars by March and some by December of this year.

Click here to read more from the Washington Post.