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.

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.

Free 21st Century Cures Tracker Available from FasterCures

FasterCures, a non-profit think tank with the goal of speeding and improving the medical research system, recently debuted a new resource to track implementation of the 21st Century Cures Act.

The free 21st Century Cures tracker focuses on the more than 100 sections in Division A, which include the key provisions on biomedical research:

“A few examples of the issues we will be tracking include the impact of changes to the National Institutes of Health’s administrative requirements for grantees, how the Food and Drug Administration implements new changes designed to enhance patient engagement and how the Department of Health and Human Services is using its new authorities to promote health information technology interoperability.

Many external factors will affect the progress of these initiatives, including policies related to hiring freezes and uncertain funding levels.”

FasterCures welcomes your feedback and suggested updates for the tracker, which can be submitted here.

Congress Begins Process of Reauthorizing Prescription Drug User Fee Act

Timely reauthorization of the Prescription Drug User Fee Act (PDUFA) was one of the hot issues discussed during the Legislative Conference during Rare Disease Week on Capitol Hill.

First enacted in 1992, PDUFA enables the Food and Drug Administration (FDA) to collect user fees from biopharmaceutical companies in order to enable the Agency to review the safety and efficacy of new medicines more quickly. According to PhRMA, it took FDA more than two years to review new medicines and more than 70% of medicines were approved outside of the U.S. before PDUFA.

Every five years, FDA and the biopharmaceutical industry negotiate a new user fee agreement, which Congress must enact in legislation. PDUFA was reauthorized in 2012 in the Food and Drug Administration Safety and Innovation Act, which also included provisions creating the Breakthrough Therapy designation as well as the Rare Pediatric Disease Priority Voucher program. PDUFA is due to be reauthorized this year, as the current user fee agreement expires on September 30th.

Released last year, the draft PDUFA VI agreement includes specific performance goals for drug review, proposed plans for enhanced use of biomarkers, expanded patient engagement, and improved specialization of reviewers for rare diseases.

The Senate Health, Education, Labor and Pensions Committee will convene a hearing on PDUFA reauthorization on March 21st at 10am ET with testimony from senior FDA leadership, and will be available by livestream. The House Energy and Commerce Subcommittee on Health will hold a hearing on PDUFA on March 22nd at 10:15am.

As discussed on the March webinar, it is important for PDUFA to be reauthorized by the end of July or FDA will need to send furlough notices to staff who review new medicines.

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.

How to Prepare for Success at Rare Disease Week on Capitol Hill

We are very excited to host more than 600 rare disease patients, caregivers and other advocates from across the country next week in Washington, DC.

As we discussed in a webinar earlier this month on Rare Disease Week on Capitol Hill, there are a few things you can do to prepare to ensure a productive experience and minimize last-minute stress:

  1. Visit the websites of your Representative and both Senators to learn more about them and find possible points of connection. Not sure who they are? You can find out here. While you’re there, sign-up for their newsletters so you can learn about district office hours and town halls.
  2. View the issue briefs for the legislative asks to be discussed at the Legislative Conference which are available here. You’ll have the opportunity to ask questions of the experts at the Legislative Conference.
  3. Practice your personal story. If you are in a large group of advocates in Senate meetings, you may only have 2-3 minutes so be concise. Think about how it might tie-in to your ask (such as more funding for research). If there is no treatment for your rare disease or if you use medicines off-label, you might want to mention that.
  4. Download the list of events during Rare Disease Week on Capitol Hill with locations and Metro stops. We also recommend that you print your hotel confirmation and flight/train/bus details.
  5. Print a map of the DC Metro and/or download a ride-sharing app such as Uber or Lyft.
  6. Check the weather and pack appropriately. It looks like you may need an umbrella but can leave your snow boots at home!

Safe travels, and we look forward to seeing you next week!

Kids v Cancer Creates Pediatric Cancer Legislative Yearbook for 2016

Kids v Cancer compiled a yearbook summarizing the achievements of pediatric cancer advocacy organizations in 2016 and outlining steps that pediatric cancer organizations plan to take in 2017. The Pediatric Cancer Legislative Yearbook 2016 is available here.

 

 

 

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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