RareVoice Award Nominations Open through July 31st

Do you know anyone who has gone above and beyond to become a rare disease policy leader and political advocate in their state or in our nation? Rare Disease Legislative Advocates (RDLA) has opened nominations for the Rare Voice Awards to be given on November 15th in Washington, DC. This year marks the sixth annual RareVoice Awards so please nominate your favorite advocate and plan to attend the celebration. Nomination categories include:

Congressional Staff

Do you know a staffer who been instrumental in helping create or implement legislation that benefits the rare disease community?

Government Agency Staff

Do you know a government agency staffer who has gone out of their way to help rare disease patients have a voice?

Patient Advocate – Federal Level Advocacy

Do you know an advocate or organization who has worked with the House of Representatives and/or the Senate to spearhead legislation at the federal level?

Patient Advocate – State Level Advocacy

Do you know an advocate or organization who has crusaded on the state level to support and advance rare disease legislation?

RareVoice Award nominations are open to the public. We encourage the community to nominate individuals and organizations who advocate for legislation and policy that benefits the rare disease community.  Winners are selected by an independent committee composed of representatives from the rare disease community and previous RareVoice Award recipients.

To nominate a rare disease champion, please click HERE. Nominations close on July 31st.

Winners will be announced LIVE at the 6th annual RareVoice awards ceremony on November 15 in Washington, DC at Arena Stage in the the Mead Center for American Theater. These champions will receive the coveted Abbey award.

The award is named after Abbey Meyers, the founder of the National Organization for Rare Disorders (NORD), and represents the “rare voice” speaking on behalf of the patients, especially children, who might not otherwise be heard. The Abbey statue was commissioned for the RareVoice Awards from the renowned sculptor Nobe. Each statue is handmade in bronze.

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.

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!

Sign up to receive news from Rare Disease Legislative Advocates to ensure that you don’t miss any updates on Rare Disease Week on Capitol Hill! You can also follow us on Twitter and Facebook.

Registration Now Open for Rare Disease Week on Capitol Hill

Join fellow rare disease patients, caregivers and other advocates from across the country at Rare Disease Week on Capitol Hill from February 27th through March 2nd, 2017. This is an opportunity for YOU to help educate the new Congress and shape healthcare policy to better meet the needs of the rare disease community.

All of the events are free for rare disease advocates, but advance registration is required for each.

Monday, February 27th, 8:30am-4pm: Rare Disease Day at the National Institutes of Health (NIH)
You can attend in-person or watch via livecast, and registration is available on the NIH website.

Monday, February 27th, 5:30-9:30pm: Cocktail Reception and Rare Disease Documentary Screening presented by Vertex featuring Up for Air at the Naval Heritage Center
Registration is available HERE.

Tuesday, February 28th, 9am-5pm: Legislative Conference presented by Horizon Pharma at FHI 360. Registration closed on February 12th.

Wednesday, March 1st, 7:30-8:30am: Lobby Day Breakfast at Hyatt Regency on Capitol Hill. Registration is now closed.

Wednesday, March 1st, 9am-5pm: Lobby Day Meetings with Members of Congress on Capitol Hill. Registration closed on February 12th.

Wednesday, March 1st, 5-7pm: Rare Artist Reception at the Kennedy Caucus Room of the Russell Senate Office Building
Registration is available HERE.

Thursday, March 2nd, 12-1:30pm: Rare Disease Congressional Caucus Briefing in Room 902 of the Hart Senate Office Building
Registration is available HERE.

For more information on Rare Disease Week on Capitol Hill including the introductory webinar held last month and a link to register for the next webinar on February 8th, please visit http://rareadvocates.org/rdw/.

Sign up to receive news from Rare Disease Legislative Advocates to ensure that you don’t miss any updates on Rare Disease Week on Capitol Hill! You can also follow us on Twitter and Facebook.

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/

Webinar Provides Overview of Rare Disease Week on Capitol Hill 2017

 

Rare Disease Legislative Advocates recently hosted a webinar which provided an overview of Rare Disease Week on Capitol Hill, to be held February 27th through March 2nd, 2017 in Washington, DC.  The week of events brings together rare disease community members from across the country to be educated on federal legislative issues, meet other advocates, and share their unique stories with legislators. There will be new Members of the House and Senate next year, and it is critical for them to meet members of their communities affected by rare disease.

All of the events are free for patient advocates, and registration will open on January 4th.

Not Able to Attend? Submit Your Unique Perspective to be Hand-Delivered to Congress

We want every Member of Congress to hear from constituents affected by rare disease, and you can make your voice heard even if you can’t join us in person. Please share your unique perspective and let your legislators know what issues matter most to you by filling out our online form by February 12th so that we can hand-deliver it on Lobby Day.

Check the Rare Disease Week on Capitol Hill webpage for more information and updates.

Travel Stipends for Rare Disease Week on Capitol Hill Increased!

The EveryLife Foundation for Rare Diseases will provide travel stipends to enable advocates from across the country to participate in Rare Disease Week on Capitol Hill, to be held February 27th through March 2nd, 2017.

Thanks to a generous donation to our Rare Giving program by Horizon Pharma, we are able to increase the amount offered to recipients! Advocates in Maryland and Virginia are eligible to receive $400 stipends, while advocates in other states in the continental U.S. are eligible to receive $800. Advocates in Alaska, Hawaii and Puerto Rico are eligible to receive $1,000 stipends.

In order to receive a stipend, advocates must attend the Legislative Conference and Lobby Day. Applications are available online through December 18th. All applicants will be notified whether they will receive a stipend or are on the waitlist on January 3rd.

We hosted a webinar earlier this month to provide an overview of the events during Rare Disease Week on Capitol Hill, highlight travel options to Washington, DC and answer questions.  You can find the webinar recording and schedule of events online.

We hope you will plan to join us to educate the new Congress and help shape healthcare policy to better meet the needs of the rare disease community. You can sign up to receive news from Rare Disease Legislative Advocates to ensure that you don’t miss any updates on Rare Disease Week on Capitol Hill. You can also follow us on Twitter and Facebook.

Thank you to

Video from Rare Disease Congressional Caucus Briefing on Strengthening Medical Innovation Now Available

On September 14, 2016, the Rare Disease Congressional Caucus gathered a contingent of thought-leaders in the rare disease space to discuss Strengthening Medical Innovation in America for Rare Disease Patients.