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Bicameral Bipartisan Rare Disease Congressional Welcome! Please silence your cell phones. Broad Thank you! Let’s Hit the Road: Welcome and Overview Angie Rowe Christina Hartman Executive Director Senior Director of Policy and Advocacy Global Genes EveryLife Foundation for Rare Diseases Connect with Us! Global Genes EveryLife Foundation for Rare Diseases Rare Disease Legislative Advocates @GlobalGenes @EveryLifeOrg @RareAdvocates @globalgenes @Rare_Advocates Global Genes Angie Rowe Executive Director Global Genes Global Genes Signature Events Signature Programs EveryLife Foundation for Rare Diseases Christina Hartman Senior Director of Policy and Advocacy EveryLife Foundation for Rare Diseases The Power of Patient Advocacy Mission and Core Principles Accelerating biotech innovation for rare diseases therapies through science- driven public policy What We Believe: No disease is too rare to deserve treatment Rare disease therapies should be safe and effective We could do more with the science we already have What We Do: Advocate for evidence-based public policy and regulatory reform How We Get it Done: Scientific and policy expertise Grassroots action Our Programs are Designed to: 1) Serve and Support Rare Disease Patients 2) Promote Awareness about Rare Diseases 3) Build A Grassroots Advocacy Community 4) Advance Regulatory Science and Policy 5) Drive Public Policy and Legislative Change We Succeed by Giving Rare Disease Patients a Voice • We do not speak on behalf of patients • Our programs seek to Educate patients about the challenges of drug development and the legislative and regulatory process Train advocates on how to tell their stories to affect policy change Create opportunities to allow patients to be heard by policy makers and to build relationships with elected officials Provide financial recourses to ensure patients can travel to policy events • Patients have been & will always be the key to advancing innovation Science & Public Policy Brings together FDA, NIH, industry and patients to address urgent regulatory challenges through case examples and expert led discussion Collaboration with Stakeholders is Key! We give Patient Organizations & Industry Partners an equal seat at the table to work together on shared goals. Seeks to improve newborn screening policies at the State and Federal levels to ensure earliest access to treatment Advocating for more specialized review divisions and a FDA Center of Excellence for Rare Diseases to increase the expertise of FDA reviewers and improve access to accelerated approval pathway Improve the Regulatory Process 11th Annual Rare Disease Scientific Workshop Science of Small Trials in the Age of Biological Plausibility We convene leaders from FDA, NIH, patient advocacy organizations, and the biopharmaceutical industry to build the science to improve the clinical development process for rare diseases. Please join the discussion on September 5th in Washington, DC. Collaboration is Key! We bring patient organizations, industry leaders, and other rare disease stakeholder organizations together to provide valuable insight on prioritizing future initiatives. It’s free for patient organizations to join one of our three working groups: 1) Public Policy 2) Regulatory 3) Newborn Screening Our next in person meeting is Wednesday, December 5th in Washington, DC. Newborn Screening Goals 1. Pass the federal Newborn Screening Saves Lives Act Reauthorization before it is expires at the end of 2019 Remove the anti-scientific privacy language preventing the advancement of screening for new diseases Seek innovative policy solutions to improve the NBS public health program to prepare for 2024 Reauthorization 2. Increase the number of diseases that are on the federal Recommended Uniform Screening Panel (RUSP) 3. Align the states with the federal RUSP NBS Action Center • The Foundation can add 1 state/year – but there are 40+ states to go • We must empower advocates to advance newborn screening legislation in their states Newborn Screening Bootcamp: April 6, 2019 In partnership with Genetic Alliance Train patient and industry partners in how to get their diseases on the Recommended Uniform Screening Panel (RUSP) Before the APHL Newborn Screening Symposium Saturday, April 6th in Chicago, IL Advocating for more specialized divisions of the FDA FDA Center of Excellence for Rare Diseases Enhance the Patient Voice Rare Disease Legislative Advocates • Educates patient advocates about how public policy impacts availability and access to treatments. • Trains advocates on how to tell their stories to affect policy change • Creates opportunities to allow patients to be heard by policy makers and to build relationships with elected officials • Builds awareness on Capitol Hill and ensures Congress hears directly from patients and others in the rare community. If the 30 million rare disease patients united with one voice they would be politically unstoppable! RDLA Advocacy Programs Brings 800 patients to Washington DC to learn about how legislation impacts access to treatments & to meet with Congress Bicameral & Bipartisan Caucus that ensures the Rare Disease Community has a permanent voice on Capitol Hill through regular briefings to educate Congress Helps build relationships with Members of Congress and allows advocates who cannot come to DC to meet with their Members during August Recess Monthly meetings/webinars & E-blasts to provide updates to the community about rare disease legislation Provides $100,000+ in funding to the community in grants and travel scholarships to ensure Congress and FDA hear directly from patients and caregivers. Honors Advocates who give patients a voice in state & federal government & helps strengthen relationships with Congressional Staff Takes place annually during Congressional summer recess Meetings for 2019 take place July 29th – Sept 4th Registration opens May 8th Deep dive webinar training July 25th Meet your legislatures in person Build your relationship with MOCs Be prepared Community Support Rare Giving provides $100,000+ in funding to the community in grants and travel scholarships to ensure Congress and FDA hear directly from patients and caregivers. Apply Online at RareGiving.org Travel Stipends to attend meetings with FDA, NIH and Members of Congress Sponsorships for patient or physician education programs or events that: Promote collaboration between rare disease groups Provide education about legislation and advocacy Advance non-disease specific policy to benefit rare disease patients Advance FDA patient focused drug development Deadline to apply for 2020 events is October 31st, 2019! Building the Grassroots Community Discounted Office space and conference rooms for rare disease organizations to have a DC office. Supports the advocacy capacity of the entire rare disease community and promotes collaboration. Promotes awareness of rare diseases and highlights the talent of the rare community. Anyone in the Rare Artist Community can participate in our annual Contest! Be sure to vote for your favorite pieces – find out how at RareArtist.org! First of its kind collaboration harnessing the core competencies of Global Genes and EveryLife with the goal to bring increasing value, insights and knowledge to patients and advocates challenged by rare disease through regional conferences. A group of 16-30 year olds from the rare disease community, who focus on building advocacy skills in the rare space. The group is relatively new and looking for more young adult champions. The primary goal is to ensure young adults have a growing impact on public policy and legislation. 1012 14th Street NW • Suite 500 • Washington, DC Rare Artist promotes awareness of rare diseases and highlights the talent of the rare community. The 11th Annual Contest for 2019 is opening July 1st. Young Adult Representatives of RDLA (YARR) are a group of 16-30 year olds from the rare disease community, who focus on building advocacy skills in the rare space. The group is relatively new and looking for more young adult champions. The primary goal is to ensure young adults have a growing impact on public policy and legislation. Goals of YARR: . Representation in each state . To educate peers about rare disease, the importance of playing an active role in advocacy and how to be heard on Capitol Hill . To ensure young adults have a growing impact on public policy and the legislative process . To instill confidence in the next generation of rare disease advocates . To provide class credit, community service hours and a resume building platform for young adults in the rare disease community Apply here - https://www.surveymonkey.com/r/XKJLF2N Rare Disease Issues are Not Partisan They Are Personal We are not Elephants or Donkeys We are Zebras We are the 1 in 10 Americans Affected by Rare Diseases Young Adults The Future of Rare Disease Advocacy Dan Pezzetta YARR member and Global Genes Contributor Advocate for Aortic Stenosis Young Adult Representative of RDLA YARR A program powered by the EveryLife Foundation of Rare Diseases The Next Generation of Rare Disease Advocacy #hearusyarr Our Goals ➢Educate other young adults about rare disease, the importance of playing a role in advocacy, Educate and how to get your voice heard on Capitol Hill. ➢Ensure young adults have a growing impact on public policy for the rare disease community. Provide Ensure ➢Instill confidence in the next generation of Rare GOALS Disease Legislative Advocates. ➢Representation in each state. Instill Represent ➢Provide class credit, community service hours, Confidence and a
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