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.

Stay Informed! Get the Lowdown on 2018 Elections

Heading in to election season, it’s important that the rare disease community’s voice is heard at the state and federal levels. To ensure our continued success, RDLA has put together a collection of resources to help advocates register to vote, know where their representatives stand on rare disease-related issues, and know about ballot initiatives that could affect the rare disease community.

  • Vote411.org – This non-partisan site finds voter requirements for your state and helps you get registered.
  • RDLA’s Legislative Scorecards – These scorecards list the voting record of your Members of Congress, offering a cumulative grade based on their support of recent federal legislation. If your representative doesn’t have a great track record on rare disease issues, In-District Lobby Days takes place from July 31st – September 4th, giving you a chance to speak with your legislator’s local office and encourage them to better support issues affecting rare disease patients.
  • Rare Disease Congressional Caucus – A great way to tell if your representatives support the rare disease community is to see if they’re a member of the Rare Disease Congressional Caucus. The caucus convenes quarterly to inform Congressional offices about issues affecting rare disease patients.
  • RARE on the Road – A partnership between the EveryLife Foundation and Global Genes, this tour of leadership events offers education on legislation currently being considered by Congress and an opportunity to meet other rare disease advocates in your local area. For those looking to move the needle on rare disease legislation or get an idea of how their representatives will vote on upcoming issues, RARE on the Road is a must-attend. Events will be hosted in Houston, Salt Lake City and Nashville this summer. For more information and to register, visit the RARE on the Road website.
  • USA.gov – Hosts information on elections for state and local officials.  Don’t know where your state representatives stand on rare disease issues? Check to see if your state has a rare disease caucus, or call your state representative’s office and ask for their stance on legislation affecting rare disease patients. RDLA provides tools to help advocates find the contact information for most of their elected officials.
  • NORD State-by-State Report Cards – The National Organization for Rare Disorders (NORD) has comprised this useful guide offering state-by-state insights on medical food coverage, newborn screening, Medicaid elegibility and other issues that affect rare disease patients and their families.
  • Ballot Inititatives – Based on your location, there may be local ballot initiatives that could affect the rare disease patient in your life. Ballotpedia provides neutral analysis of ballot initiatives at the state and local level so voters step in to the booth with the most information possible.

Notice a valuable voter resource that’s missing?  Email Grant Kerber at gkerber@everylifefoundation.org and we’ll add it to this page. Let’s make a difference in 2018!

Rare Disease Week on Capitol Hill 2018 Brings Hundreds of Advocates to Capitol to Educate Congress

Thank you to the 750+ rare disease patients, caregivers, researchers and other advocates who joined us during Rare Disease Week on Capitol Hill 2018, which took place February 25th through March 1st.

The week kicked off on Sunday evening at our 8th annual Rare Disease Documentary Screening and Cocktail Reception. The capacity crowd was treated to a screening of The Ataxian, which follows the journey of Freidreich’s Ataxia patients Kyle Bryant and Sean Baumstark as they bike across the United States. Following the screening, Kyle and Sean were on-hand to take questions from the crowd, with topics ranging from their lives with Freideich’s Ataxia to the filmmaking process.

On Monday, more than 450 advocates joined us in-person for the Legislative Conference, with more than 125 participating remotely through the event livestream. Experts from Capitol Hill and patient advocacy organizations discussed what to expect from Congress following changes to the Orphan Drug Tax Credit, how to build effective relationships with Members of Congress and their staff, and key pieces of legislation for 2018. Video and presentations will be available on the Legislative Conference resource page.

Tuesday morning, advocates began Lobby Day at breakfast with remarks by Joel White, Founder and President of Horizon Government Affairs. Mr. White was followed by Dr. William Gahl, Head of the Undiagnosed Diseases Program at the National Institutes of Health (NIH) and Debra Lewis, who serves as Acting Director of the Food and Drug Administration’s Office of Orphan Product Development. Advocates left the breakfast energized by the speakers and ready to educate Members of Congress and their staff about issues affecting the rare disease community.

And educate they did! In all, 371 advocates representing 49 states participated in a total of 294 Lobby Day meetings, discussing the Rare Disease Congressional Caucus, precision medicine legislation, incentives for rare disease drug development and other key legislative topics. Thanks to these advocacy efforts, the Rare Disease Congressional Caucus picked up 4 new members in the Senate and 9 in the House.

Wednesday’s events began with a Rare Disease Congressional Caucus briefing, titled “The Rare Disease Lifecycle: Diagnosis to Treatment.” Caucus Co-Chair Representative Leonard Lance (R-NJ) welcomed advocates and Congressional staff before our expert panel covered several topics affecting the rare disease community. Videos from the briefing are available here.

On Wednesday evening, we hosted the annual Rare Artist reception, which featured winning entries from the 2017 Rare Artist contest.  We were honored to have seven awardees in attendance speak about the importance of art as therapy and the struggles they experience as rare disease patients. Also on display were photographs from Rick Guidotti’s Positive Exposure campaign, which put the beauty of rare disease patients on display for all to see.

We closed the week at the NIH, where EveryLife staff was on hand to meet attendees and provide information about our programs assisting the rare disease community.

After another successful year, we want to thank everyone who participated in Rare Disease Week on Capitol Hill in-person and remotely.  Your support and appreciation means the world to us.  We hope to see even more of our vibrant community in 2019!

Follow Rare Disease Legislative Advocates on Twitter and Facebook for news on upcoming events.

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.

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.

New Report on Economic Impact of NIH Research Helps Make Case for Increased Funding

The National Institutes of Health (NIH) is the largest biomedical research agency in the world committed to improving health by conducting and funding research. Significant cuts to the NIH budget in the President’s Fiscal Year (FY) 2018 budget and proposal for the remainder of FY2017 are of significant concern to patients and researchers, and threaten the pace of scientific discovery.

NIH has several initiatives focused on increasing understanding of rare diseases and the speed at which new treatments are developed. These include the Rare Diseases Clinical Research Network, Office of Rare Diseases Research and Therapeutics for Rare and Neglected Diseases program as well as the Undiagnosed Diseases Network, which seeks to provide answers to patients with rare genetic diseases and the Clinical Center, which is the largest hospital dedicated to clinical research in the U.S.

Data in a new report illustrates that NIH research creates jobs across the country and helps make the case that funding should be increased, not decreased. According to United for Medical Research (UMR), research funded by NIH supported close to 380k jobs and $64.799 billion in economic activity in FY2016.

“NIH-funded research is an engine for economic and medical progress, making the drastic cuts to the NIH proposed in the President’s initial budget doubly dangerous. These cuts will stymie the jobs creation and economic activity happening today as a result of NIH-funded research and they will slow down or completely stall critical research on a range of diseases, including some of our most costly health problems…” stated UMR President Lizbet Boroughs.

You can find the impact of NIH funding in your state here. The one-pager on the need for increased funding for NIH and the Food and Drug Administration (FDA) from the Lobby Day during Rare Disease Week on Capitol Hill is available here, and a video of the presentation on this topic by Sara Chang of Research!America during the Legislative Conference can be found here in the section entitled “Rare Disease Legislation in the Queue”.

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.

Rare Disease Week on Capitol Hill Engaged 600+ Advocates

Thank you to the 600+ rare disease patients, caregivers, researchers and other advocates who joined us for at least one event during Rare Disease Week on Capitol Hill in Washington, DC from Monday, February 27th, through Thursday, March 2nd.

The week began at the National Institutes of Health (NIH), where the EveryLife Foundation’s Chief Advocacy and Science Policy Officer presented an update on our work to improve the state newborn screening system and create incentives to encourage biopharmaceutical companies to repurpose approved medicines for rare diseases.

That evening, we hosted a cocktail reception, screening of the documentary Up for Air, and panel discussion. Senator Ed Markey (D-MA) and Representative Jim McGovern (D-MA) made brief remarks.

Aproximately 350 advocates joined us for the Legislative Conference on Tuesday, which was livestreamed for the first time. Experts from Capitol Hill and patient advocacy organizations discussed what to expect from the new Congress and Trump Administration, how to build effective relationships with Members of Congress and staff, and key legislation. Video and presentations will be available on the Legislative Conference resource page.

Advocates began Lobby Day at breakfast with remarks by Dr. Janet Woodcock, Director of the Food and Drug Administration’s Center for Drug Evaluation and Research, as well as Representative Gus Bilirakis (R-FL) and former Representative Brian Baird (D-WA), who urged advocates to be brief, polite and persistent in their meetings with Members of Congress and staff.

328 rare disease advocates participated in 270 Lobby Day meetings, discussing the Rare Disease Congressional Caucus, healthcare reform, incentives for rare disease drug development and other key legislative topics.

On Wednesday evening, we hosted the annual Rare Artist Reception which featured winning entries from the 2016 contest and remarks from several of the artists.

The final event of the week was a Rare Disease Congressional Caucus briefing entitled, ” Advancing Rare Disease Treatments in the Era of Cures and Health Care Reform.” Caucus Co-Chair Senator Amy Klobuchar (D-MN) welcomed advocates and Congressional staff. Video will be posted on the Caucus webpage within a few weeks.

Follow Rare Disease Legislative Advocates on Twitter and Facebook for news on upcoming events.

Four Ways to Participate Remotely in Rare Disease Week on Capitol Hill

If you are not able to join us and advocates from across the country at Rare Disease Week on Capitol Hill on February 27th through March 2nd, you can still make your voice heard on Capitol Hill and participate in some of the programming.

Here are four ways you can participate remotely:

  1. Watch the livestream of Rare Disease Day at the National Institutes of Health (NIH) on Monday, February 27th. The agenda and link to register are available on the NIH website. Speakers include representatives from Global Genes, EveryLife Foundation for Rare Diseases and FasterCures as well as leaders from NIH and the Food and Drug Administration (FDA). If you are on social media, use the hashtag #RDDNIH to be part of the conversation.
  2. Watch the livestream of the Legislative Conference on Tuesday, February 28th, to learn what to expect from the new Congress and Trump Administration, how to build an effective relationship with Members of Congress and staff, and about key legislation for the rare disease community. The draft agenda, link to register for the free livestream and legislative issue briefs are available here. If you are on social media, use the hashtag #RareDC2017 to quote speakers, comment and ask questions.
  3. Call your Members of Congress on Lobby Day on Wednesday, March 1st. The one-page background papers for each of the key legislative issues discussed at the Legislative Conference are available here. 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.
  4. Email your Members of Congress on Lobby Day on Wednesday, March 1st. Chose the current RDLA action alerts you support. We make it easy for you to send an email to your legislators. You can ask them to join the bipartisan Rare Disease Congressional Caucus, which hosts quarterly briefings on Capitol Hill to raise awareness and educate Members and staff.

Please join us to help educate the new Congress and shape healthcare policy to better meet the needs of the rare disease community!

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Video from Rare Disease Congressional Caucus Briefing on PDUFA Now Available

The final Rare Disease Congressional Caucus briefing of 2016 focused on the Prescription Drug User Fee Act (PDUFA), the agreement between the biopharmaceutical industry and Food and Drug Administration (FDA) regarding user fees paid by industry to supplement federal funding for human drug review.

Congress will need to reauthorize PDUFA before the current agreement expires at the end of September 2017, which will be an opportunity to include provisions to encourage the development and streamline the review of rare disease treatments.

Watch the videos to learn more from representatives from debra of America, National Health Council, Biotechnology Innovation Organization, Genentech and office of U.S. Representative Leonard Lance (NJ).

http://rareadvocates.org/driving-innovation-for-lifesaving-therapies-through-pdufa-reauthorization-in-2017/