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.

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: 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.

Meet 2017’s RareVoice Nominees: Federal or State Advocacy by a Teenager

In this series, we will be introducing you to rare disease advocates who made an impact at the state and federal level. These passionate teens are all nominated for RareVoice Awards in the “Federal or State Advocacy by a Teenager” category.

Adam Foye has lived his whole life with a rare muscle disease known as Titin-related centronuclear myopathy. Despite managing numerous health challenges, he faces barriers head-on and advocates for other rare disease patients. As a person with a rare, chronic health condition he knows how important access to good medical care is.  When the Affordable Care Act came under threat of repeal, he stood up for all people with ongoing medical challenges.

 

After losing her father to the x-linked genetic disease adrenoleukodystrophy (ALD) at the age of five, Taylor Kane started raising awareness and money for ALD research by working with Run for ALD. Now, at age 19, Taylor is the lead advocate for the organization, having helped raise more than $200,000 for ALD research. As a carrier of the ALD gene herself, Taylor founded a support group called Y.A.C. (Young ALD Carriers) to unite, educate and empower teenage and young adult female carriers, and to assist them in affecting positive change through advocacy, social media and the legislative process.

For the past ten years, Ian Mignone’s mother has suffered from Addison’s Disease. While volunteering at the University of Florida Health’s emergency room in Jacksonville, he learned that Emergency Medical Service (EMS) personnel could not treat an adrenal crisis simply because they did not have a protocol in place. Since then, he successfully advocated for and been instrumental in passing emergency protocols in eight Florida counties, and is currently working with twenty-one additional counties to continue to press forward throughout Florida.

 

Emily Muller established herself as a prominent activist in the rare disease community when she launched Emily’s Fight, an awareness campaign, at the age of thirteen. She has spoken at such events as TEDx, the Global Genes RARE Patient Advocacy Summit, SHINE and Starlight Children’s Foundation Midwest Gala, and contributed written pieces to The Mighty and To Write Love On Her Arms. In addition, Emily is a passionate legislative advocate and gained recognition from the House Energy and Commerce Committee after three years of lobbying and internet activism in support of the 21st Century Cures Act.

 

At 18, Shira Strongin is an accomplished, award-winning activist and writer who has an undiagnosed rare disease that she does not let slow her down. She is the founder of Sick Chicks, an international community that works to empower and unite women with varying disabilities and illnesses. She has also been published in Forbes and has a passion for speaking on topics such as disability rights, women’s rights and engaging the young adult community in advocacy.

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

Participate Remotely in In-District Lobby Days This Summer

Please join the more than 230 advocates who are meeting with their federal legislators during August recess by contacting your Members of Congress. It is easy to participate remotely, and you can do it on your own timeframe!

First, check out the action alerts on our website. It only takes a few minutes to enter your address and send a message to your legislators. Current alerts include:

  • The Alliance for a Stronger FDA encourages advocates to request robust appropriations for the Food and Drug Administration (FDA) to ensure the Agency has the resources it needs to recruit and retain qualified staff and efficiently oversee the review of medical products. Click here to take action.
  • The EveryLife Foundation for Rare Diseases asks advocates to urge their legislators to co-sponsor the OPEN ACT (Orphan Product Extensions Now, Accelerating Cures and Treatments; HR 1223/S 1509) which has the potential to double the number of rare disease treatments approved by FDA. Click here to take action.
  • RDLA is working to grow the Rare Disease Congressional Caucus, a forum for Members of Congress to voice constituent concerns, collaborate on ideas, facilitate conversations between the medical and patient community and build support for legislation that will improve the lives of people with rare diseases. Click here to invite your legislators to join the bipartisan Caucus.

Then, if there is not an action alert for your legislative priority or if you want to do more than send an email, call the United States Capitol switchboard at (202) 224-3121 to be connected to the office of your Representative or Senators. Ask to speak with the Legislative Assistant who handles healthcare or a leave message with your “ask” as well as your contact information. The resources on the In-District Lobby Day webpage can help you prepare for your calls.

Participating in In-District Lobby Days in-person or remotely will help you build relationships with key staff members in your legislators’ offices, and ensure that they are aware of the impact of rare disease on local communities.

FDA User Fee Package Passes in the Senate, Awaiting President’s Signature

The Senate has passed the Food and Drug Administration Reauthorization Act (FDARA), a legislative package that supplements FDA funding by requiring manufactures to pay user fees when submitting medical products for review in exchange for a timely approval process.

Rare Disease Advocates Invited to Join Rally for Medical Research on September 14th

Held every September, the Rally for Medical Research brings together more than 300 hundred patient and physician advocacy organizations, universities, research centers and biopharmaceutical industry associations to support sustained federal investment in the National Institutes of Health (NIH).

As noted on the website, “The purpose of the Rally is to call on our nation’s policymakers to make funding for National Institutes of Health (NIH) a national priority and raise awareness about the importance of continued investment in medical research that leads to MORE PROGRESS, MORE HOPE and MORE LIVES SAVED.”

The 5th Rally for Medical Research will be held on September 14th in Washington, DC. Advocates are welcome to attend a training session followed by a reception on September 13th.  For more information and to register, visit the Rally for Medical Research website. Sabah Bhatnagar, Program Director of Rare Disease Legislative Advocates, will be there so please look for her if you attend!

If you are unable to participate in-person, you can join the national Day of Action remotely. Show your support for funding for NIH on social media using the hashtag #RallyMedRes and follow the Rally on Twitter and Facebook.  A Twitter cheat sheet, fact sheets on the importance of NIH funding and additional resources will be available on the Rally website.

Politico Article Explains What’s Next for Healthcare Reform

After the narrow defeat of Affordable Care Act (ACA) replacement plans in the Senate, Senators have left for August recess without voting on other proposals. At the nail-biting conclusion of a week-long debate, the Senate voted 51-49 against the “skinny repeal” bill that would repeal mandates and many of the taxes associated with the ACA. Several Senators have realized that the only path forward requires bipartisan cooperation, despite a highly-polarized environment.

A bipartisan solution will most likely include state flexibility and market stabilization that Republicans desire and funding for cost-sharing subsidies that Democrats support. A recent Politico article states, “Republicans will seek a more flexible 1332 waiver — an existing Obamacare provision that allows states to make changes to the law’s requirements, according to GOP sources. Democrats say funding the approximately $7 billion annual payment to insurance companies to help cover low-income people is the minimum requirement for a deal. The Trump administration has said it is ready to pull the monthly payments at any time in response to the failed repeal effort, a move that could immediately drive companies off the exchanges and further undermine the Obamacare markets.”

Senate Health Education Labor and Pensions (HELP) Committee leadership will set the tone for future replacement proposals. Sen. Alexander (R-TN), the Chairman of the Committee, along with Sen. Murray (D-WA), the Ranking Minority, must bridge the chasm between viewpoints on opposite ends of the spectrum on the HELP Committee.

ACA replacement plans could have a critical impact on the rare disease community. Click here to read the full article from Politico.