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.

RDLA Welcomes Sabah Bhatnagar as Program Director

Sabah Bhatnagar recently joined the EveryLife Foundation team as Program Director of Rare Disease Legislative Advocates (RDLA). She was previously the Government and Industry Affairs Coordinator at the Association of Clinical Research Organizations (ACRO), where she advocated for policy and regulation that support clinical research and the development of innovative, safe, and effective treatments and promoted a more efficient clinical trial process. As a Health Policy Communications Associate at the Alliance for Health Reform, a non-partisan organization focused on educating policy-makers about health policy, she helped execute over 70 events on a variety of health policy topics.

Sabah is a rare disease patient and holds a B.S. in biology from the University of Mary Washington. She is based in Washington, DC and looks forward to engaging the rare disease community.

Trump Administration Releases Fiscal Year 2018 Budget Proposal

The President’s budget has dominated headlines since it was released yesterday, prompting concerns from many in the healthcare sector and questions among advocates about what it means. You can find the budget and supplemental materials here.

Federal Budget Process

This two minute video from AAAS explains the long process of creating and passing the federal budget. While the President makes a recommendation, it is up to Congress to pass funding bills. It is important for rare disease advocates to contact their legislators to request funding for the federal programs they support.

 Funding for National Institutes of Health (NIH)

The proposed budget would reduce funding for NIH by $5.8 billion. Congress recently passed a budget with an increase in NIH funding for the remainder of FY2017 despite the President’s call for a reduction, and there is bipartisan support for biomedical research funding. Many health advocacy groups voiced opposition to the proposed cut, as did the Chairman of the House Appropriations subcommittee that oversees health.

The drastic cuts in the Administration’s 2018 budget for the National Institutes of Health (NIH) are a significant blow to medical research and the patients who depend on it.” –United for Medical Research

Steep funding cuts for the federal health agencies are counterproductive at a time when innovative research is moving us closer to identifying solutions for rare diseases, new prevention strategies to protect Americans from deadly and costly conditions, advances in gene therapy, new technologies for understanding the brain, and treatments that harness the ability of our immune system to fight cancer.” – Research!America 

Funding for Food and Drug Administration

The Administration’s proposed budget would cut funding for FDA by $871 million and increase the amount of user fees. The user fee agreements between FDA and industry were finalized last year and the reauthorization process is underway in both the House and the Senate. The user fee agreements must be reauthorized by July 31st or the FDA must begin to notify the staff who review new medicines ad devices of potential layoffs.

We are concerned that should the FDA, as a consequence of this proposed budget shift, become significantly underfunded, the agency will have to choose among important public health priorities. Beyond the needed investment in FDA’s core functions, sufficient funding is necessary to spur innovation and provide critical oversight initiatives for drugs, biologics, and food.” – Muscular Dystrophy Association

Funding for Other Health Programs of Interest to Rare Disease Community

The budget also includes a cut of nearly 20% for the Children’s Health Insurance Program (CHIP) as well as cuts to Medicaid. A speaker from the American Academy of Pediatrics explained the importance of both programs at the Legislative Conference during Rare Disease Week on Capitol Hill earlier this year. You can access the video and slides here.

What You Can Do to Impact the Budget

To start, call or write your legislators to ask them to support funding for the federal programs that are most important to you. You can find their contact information here.

You can also participate in In-District Lobby Days during the summer Congressional recess from August 7th through September 6th.  When you register, you can specify when you are available and how far you can travel.  We’ll schedule meetings for you in the local offices of your Representative and Senators based on that information, and hold a webinar on July 28th to help you prepare.

EveryLife Foundation and Global Genes Unite to Present RARE on the Road in Atlanta, Kansas City and Portland

The EveryLife Foundation and Global Genes are joining together to host a series of RARE on the Road events this summer to educate rare disease patients, caregivers and other advocates in an interactive and engaging environment.

Building on the successful regional Legislative Conferences hosted by Rare Disease Legislative Advocates, the RARE on the Road events will highlight topics from capacity building to a patient’s role in drug development, including a hands-on workshop and networking lunch. These FREE regional events will help advocates learn, grow and become independent activists for their rare disease community. The general agenda for all three events is available here.

Registration is available by clicking the links below. Each event will run from 8:30am through 4:30pm.

Saturday, May 13th, 2017: Loudermilk Conference Center, Atlanta, GA

Monday, June 5th, 2017: Kauffman Foundation Conference Center, Kansas City, MO

Saturday, July 15th, 2017: Shriners Hospital for Children, Portland, OR

A limited number of $100 travel scholarships are available for rare disease patients and caregivers who otherwise would not be able to attend. Applications are available online and are due one month before each event, except for applications for a scholarship to RARE on the Road in Atlanta which are due by April 20th.

You can help spread the word by sharing the flyer (available here) with your community.

Utilizing Facebook and Twitter to Engage Policymakers is Key Part of Advocacy

Facebook and Twitter provide opportunities to engage state and federal policymakers as well as other rare disease advocates who can take action on their own and/or increase the reach of your message.

You can find a list of Twitter handles for U.S. Senators here, and another for members of the U.S. House of Representatives here. The National Conference of State Legislatures has a list of state legislative social media websites that is available here.

How can YOU use social media? Below are several examples. For all of them, make sure to tag your legislators on Facebook and use their official Twitter handles to make sure they see your posts.

  • Encourage your legislators to support or oppose specific legislation, and thank them when they do.

  • Are your Representative and Senators members of the Rare Disease Congressional Caucus? Tweet to thank them if they are or ask them to join if they are not, and post a respectful request on their Facebook pages.

  • Thank legislators (or their staff) for meeting with you, especially if they agree to something you requested such as supporting legislation. Take a photo to include in your post, if possible.

  • Use your Twitter account to ask your followers to take action. Retweet action alerts from other organizations that you support in order to expand the number of people who see it and participate.

  • You can also increase the number of people who participate in action alerts by sharing them on Facebook. Rare disease advocacy organizations rely on help from individual advocates who not only take action, but call on their friends and family to do so as well.

Emily Eckland, Digital and Social Media Communications Manager for Eli Lilly & Company, discussed Best Practices for Staying in Touch and Social Media to Build Momentum at the Legislative Conference during Rare Disease Week on Capitol Hill. The video of her presentation is available on the Legislative Conference resource page.

Rare Disease Congressional Caucus Briefing Videos Now Available

The Rare Disease Congressional Caucus hosted a briefing during Rare Disease Week on Capitol Hill which focused on Advancing Rare Disease Treatments in the Era of Cures and Health Care Reform.

After welcoming remarks from Caucus Co-Chair Senator Amy Klobuchar (D-MN), the panelists addressed implementation of the 21st Century Cures Act, reauthorization of the Prescription Drug User Fee Act, efforts to repeal and replace the Affordable Care Act, new models for rare disease drug development such as repurposing, and the role of incentives to encourage the development of orphan therapies.

Videos from the briefing are available here. A full list of Caucus members and videos from previous briefings are available on the Caucus webpage.