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 resume building platform for young adults in the rare community. 4 Advocacy Committee available with membership

https://everylifefoundation.org/young-adult-representatives/ Let’s Go on a Road Trip: My Rare Disease Journey

Erin Ward Co-Founder and President MTM-CNM Family Connection, Inc. Erin Ward Co-Founder & President My inspiration…My RARE Story… Ways I’ve shared our RARE story….

• With providers-caring for Will • Educating school to best educate Will • Opportunities at the hospital • Research Teams • Patient Partner Roles • Redefining my career • Through creation of non-profit • Family Conferences • Partnering with pharma industry • Legislative Advocacy WHY tell your story & how storytelling is impactful…

• To raise awareness • To educate • To encourage understanding • To promote empathy • To build alliances • To trigger action • To motivate • To support others on a RARE journey • To inspire those working in the RARE community & beyond • To create positive change • To take legislative action • To accelerate treatments for RARE diseases WHO to share your RARE story with…

• Every conversation is an opportunity to refine your story and key message • Family and friends • Providers: medical, school, supports • Your RARE community and Global Genes • Researchers and medical teams • Pharma Industry partners • In the broader community • With legislative leaders HOW to tell your story & how your story evolves..

• Start where you are • Be who you are • Share what feels right for you and your family • Understand the purpose or your goals • Know your audience • After every time, self reflect and ask for feedback • Appreciate the impact it has on others • Reflect on the impact it has on you! The Power of Telling Your RARE Story MTM-CNM Family Connection’s Mission: Connecting families to…

Research • Connecting patients & families to research & researchers

Resources • Website, Newsletters, Family to Family

Relationships • Conference, Facebook group, Traveling Turtle MTM-CNM Family Conference: Through the Years

Houston, TX 2009 Minneapolis, MN 2013

Minneapolis, MN 2011 MTM-CNM Family Conference: Through the Years

Chicago, IL 2015 Nashville, TN St. Louis, MO 2017 2019

RARE Connections are priceless Thank you & connect with us: • Website: www.mtm-cnm.org • Email: [email protected] • Facebook: MTM-CNM Family Connection • Twitter: @mtmcnmfamily Tales From the Road: How to tell Your Story and Build Capacity Special thanks to Emma Rooney!

Ashley Yee Director of Patient Education, Patient Engagement Global Genes Rare Disease Stories Matter • Why bother telling your story? • Benefits the storyteller • Cathartic • Promoting a goal • Identifying commonalities / connections • Give us the chance to reflect and process what we’ve gone through • Benefits community • Increase awareness about specific disease or conditions • Breakdown feelings of isolation • Build community networks Methods • Be creative • What is at your disposal? • Think about relevancy to your audience • Be comfortable Art washes away from the soul the dust of everyday life. ~ Pablo Picasso

Sea of Grief, Christina Baltais. 2018 awardee in the adult category of Rare Artist. Bio available HERE The aim of art is to represent not the outward appearance of things, but their inward significance. ~ Aristotle

A Dog’s Eye, Julia Band Orange. 2016 awardee in the children’s category of Rare Artist Bio available HERE

Perseverance - By Julia Boonnak

My son climbs mountains every day. He doesn’t know any different and he doesn’t have a choice. He hasn’t got a big roar but a tiny little voice. He finds life easier when it’s full of fun and play. He shines like all children do and does it his way.

Rare disease families climb mountains every day. We are required to work harder and be better than our best. We learn to be nurses, therapists, mentors, coaches, there is no time to rest. We advocate for research and we want it straightaway. We’ve got each other to share with and you know what? We’re OK.

A rare global community climbs up mountains every day. Roped to courage up in front and fear pulls back the rear. Never give up searching but the end may remain unclear. Slice feet, cut legs, draw tears you sweet but wretched pathway. You’ll never take our hearts our hope and you’ll be beaten someday.

Modalities Now that you have determined your method, how will you share it? • Social Media • Conferences • Online communities: RARE Daily, The Mighty Privacy Share what you are comfortable sharing • What level of control will you have over how it’s presented? • Is it clear the ideas expressed are your own? • What is the life span of the story? • Can this story negatively impact you? • Any implications? • How will you feel after sharing this story? • Are you prepared to receive negative and positive feedback? Know Your Audience • Audience- Who is the person? Who is in the crowd? • Expectation • Time • Objective/Goal/Ask- Why this story? • Conflict/Impact/Resolution Components of a Good Story • Three major parts • Opener • Body • Close Opener • Grab your audience attention- keep your goal in mind • Introduce yourself • Summary of what your about to tell them • Landscape of concern • Talk about prevalence • Challenges of your community • Statistics Body • Tell your story! • Keep your goal in mind • Why choose this story? Remember that it is ok to shorten or move away from portions of your story and not every detail needs to be said or given in chronological order • Relatable challenges • Symptoms that may be similar to find connections • Empathy for your story Closing • Be memorable! Remember your goal • Summarize your story • Remind them of the impact • Have an ask • Remind your audience they can be part of the change Lets Get Started Available Resources

• Global Genes Resource Hub • Speak Easy a Guide to Public Speaking • How to Promote your Rare Disease Story Through Social Media • Using Storytelling to Raise Awareness for your Rare Disease

Gain Momentum and Drive Change: A Historical Primer

Lisa Schill Event Development Consultant EveryLife Foundation for Rare Diseases My Journey into the Rare Disease World

Our mission is to advance research of the RASopathies by bringing together families, clinicians, and scientists.

RASopathiesnet.org How do we get to a cure? 11-14 years to develop a new treatment!!

Up to 2.6 BILLION dollars!!

The Drug Development & Approval Process. (2016). http://www.fdareview.org/03_drug_development.php Mullin, Rick.(2014, November 24). http://www.scientificamerican.com/article/cost-to-develop-new-pharmaceutical-drug-now-exceeds-2-5b/ That is A LOT of bake sales!! Who here is looking for a treatment or cure? Who controls treatments and cures? Drive LEGISLATION to Create Change & CURE THE PROCESS Why Public Policy Matters

How many people here today have an FDA approved therapy??

Small disease patient population + Complex diseases + High cost of developing a treatment = No profit/no investment/no treatment The Need – 30 Million Americans • Rare Disease is defined as less than 200,000 patients in the US • 83% of Rare Diseases have less than 6000 patients in the US • More than 500 new rare disease treatments developed since the passage of the Orphan Drug Act 30 years ago • 93% of the 7,000 rare diseases have no approved therapies • Many of the treatments in development for diseases already have treatments available • The science exists to treat many of these diseases • WE MUST DO BETTER: Public policy is needed to close the innovation gap Public Policy Can Do What Bake Sales Cannot • Create incentives for company’s to develop drugs • Establish a predictable development & regulatory pathways to de-risk the investment in rare disease therapies • Bring down the cost of developing a treatment • Allow for rare disease therapies to use Accelerated Approval Pathways • Allow for alternative statistical models for clinical trail design • Ensure patients have access and can afford therapies What can we learn from the AIDS movement? Improving FDA Policies Make a Difference

• HIV drug development example • AIDS activists stormed FDA in late 80’s • 1992: FDA forced to create “Accelerated Approval” – New regulations allow use of a surrogate or biomarker to determine if the drug is effective – If the surrogate is “reasonably likely to predict clinical benefit” – Reduces the time and cost of development • Biggest surge ever in innovation – 25 drugs in 15 years period after reg change in 1992 – All approved under Subpart H Accelerated Approval The MOST Important Lesson From the AIDS Movement Educated & activated patients are far greater at making change than paid lobbyists!

15 Know Your Power

Political power comes from the ability to mobilize people to take action! The Power of Grassroots Advocacy

Legislation & Public Policy impact science, the drug development process and access to treatments • You have the POWER to effect change • Take an Active Role in the Political Process – Vote, volunteer on a campaign, attend a Town Hall – Attend hill days, request a meeting with your MOC – Build relationships with your elected officials • Influence Legislation & Policy – Build coalitions – Activate your personal contacts – Support others with their policy goals as you will need their help when you seek your goals – Don’t let someone speak for you You Have the Power to Move Congress Maximizing the Patient Power • Why does this matter to me? – Legislation & public policy impact science, the drug development process and access to treatments – GET EDUCATED! Important to understand the “ecosystem” of the drug development process and healthcare system to see how policies will effect your family • Make sure rare disease organizations are representing your interests – You can & should influence an organization’s polices • Organizations are only as strong as their grassroots – Members of Congress are more responsive to constituents than organizations – Take Action: patients must contact Congress • Call, email, attend a Town Hall, request a meeting, etc – Build relationships with your Members of Congress & staff Patient Organization’s Role

• Share information – We need to educate patients/parents/caregivers, not just the heads of patient organizations • Pick one or two issues that most affect your patients to champion • Join or form Coalitions to work on your priority issues • Support other organizations efforts, you will need them to support your efforts • Sign onto letters to support legislation, your organization’s support counts! • Activate your grassroots The Very Quick History of U.S. Drug Regulation Including An Even Briefer Overview of Rare Disease Legislation FDA is Officially Established! As a response to the public outrage at the unhygienic conditions in the Chicago stockyards featured in Upton Sinclair’s book “The Jungle." 1906 Pure Food and Drugs Act Aimed to rein in long-standing, serious abuses in the consumer product marketplace.

Harvey Washington Wiley, chief chemist of the Bureau of Chemistry of the U.S. Department of Agriculture, FDA’s predecessor. ** Slide images credited to FDA’s centennial presentation The Food, Drug, and Cosmetic Act of 1938 ° Required proof of safety ° Authorized inspections ° Outlawed false claims Thalidomide tragedy lead to The Kefauver-Harris Amendments of 1962 • Required proof of effectiveness • Gave FDA control over investigations • Gave FDA authority to regulate advertising of prescription drugs • Established good manufacturing practices • The Orphan Drug Act of 1983 authored by Hatch & Waxman • Defines RARE as affecting fewer than 200,000 patients in the US • Creates incentives for companies to develop treatments What’s in ODA • Orphan Drug Tax Credit (ODTC): – Allows companies to claim a tax credit of up to 50% of their qualified clinical testing expenses (2018 tax bill changes this tax credit to 25%) • The Orphan Products Grant Program since 1983 – Received over 1800 applications, reviewed over 1400 applications – Funded over 500 studies leading to 45 drugs on the market • The FDA grants 7 years of exclusivity to the drug – The FDA cannot approve the “same drug” from another company • (Orphan drugs also excepted from FDA User Fees) • Special Treatment? Companies developing rare disease therapies are still required to provide the same amount of safety and efficacy data despite small patient populations Has it worked? 1984 Hatch/Waxman Act • 1984 Drug Price Competition and Patent Term Restoration Act • Establishes a generic drug approval process to make available more low costs treatments, bringing down prescription drug costs • Find the balance between two potentially competing policy interests: • Pharmaceutical Innovation • Low Cost Generic Drugs • Why is important? • If you don’t get the balance right diseases will remain untreated

28 THE AIDS CRISIS demanded changes in regulations • Standard drug testing and approval process ° Preclinical testing ° Clinical studies – Phase I, II, III ° Post-marketing surveillance • Early access to experimental therapies ° Serious diseases ° No alternative treatments The Prescription Drug User Fee Act of 1992 (PDUFA)

° FDA allowed to collect fees from industry ° FDA required to reduce review time ° Must be reauthorized every 5 years Modern Times Legislation • 1997 Best Pharmaceuticals for Children Act & Pediatric Research Equity Act (BPCA & PREA) • Creates incentive & requires industry to study treatments on children • 2002 Rare Disease Act of 2002 • Establishes the Offices of Rare Diseases at the NIH • Authorized funds for rare disease research (never appropriated) • 2008 Newborn Screening Saves Lives Act • 2012 Food & Drug Administration Safety & Innovation Act (FDASIA) (PDUFA V reauthorization) • 2016 21st Century Cures Act • 2017 RACE for Children Act • 2018 Right to Try Act • 2018 STAR Act • 2018 Sickle Cell Disease and Other Heritable Blood Disorders Research, Surveillance, Prevention, and Treatment Act Food & Drug Administration Safety & Innovation Act • Rise of the Rare Disease Patient Legislative Advocacy • Feb 2012 RDLA hosted it’s first Lobby Day • 5 Provisions were included to benefit Rare Disease Patients – Pediatric Priority Review Voucher for Rare Diseases – Patient Focused Drug Development – ULTRA/FAST Language to improve access to accelerated approval pathway for rare diseases – Breakthrough Therapies – EXPERRT Act • More work was needed – lead Congress to Launch the 21st Century Cures Initiative 21st Century Cures Initiative

It started with a visit from two little princesses…..

Rare Disease Legislative Advocates Rare Disease Week on Capitol Hill CURES New Jersey Roundtable 21st Century Cures Act • Rare Disease Community Priorities: – Improve biomarker qualification – Improve FDA’s ability to recruit & retain staff & keep up on the latest science – Billions in NIH & hundreds of millions in FDA Funding – Expanding Hope Act (Priority Review Vouchers) – Neurological Disease Surveillance – Compassionate Use Reform & Enhancement Act – Patient Focused Drug Development

21st Century Cures in Committee 51-0 Vote

21st Century Cures Floor Vote On to the Senate!

21st Century Cures becomes a LAW

Grants for Advancing RAS/RASopathy Therapies Key Takeaways

• Create relationships with Members and their staff - remember it takes time to develop relationships • Don’t choose sides – rare diseases affect everyone Republican, Democrat, and Liberal • Call, email, and meet with your Members and their Staff • Have your friends and family reach out as well • Thank your Members and their staff for their time and their help • Anyone can change policy – whether you are 5 or 105 • The more we collaborate together, the more our voices will be heard Implementation of the 21st Century Cures Act at the NIH . Streamlined Certificates of Confidentiality to protect research participants . Data sharing guidelines and NIH Date Commons . Inclusion of children, seniors, and lactating women . Next Generation Research Initiative to focus on early and mid career investigators . Improve ClinicalTrials.gov to be more user friendly . Advisory Committee on Rigor and Reproducibility, will develop formal policy . NIH Innovation Fund . Precision Medicine Initiative (All of Us) . BRAIN Initiative . Cancer Moonshot . Regenerative Medicine Innovation Project 56 Implementation of the 21st Century Cures Act at the FDA . FDA steering committee to implement CURES Act provisions . https://www.fda.gov/RegulatoryInformation/LawsEnforcedbyFDA/SignificantAmendment stotheFDCAct/21stCenturyCuresAct/ucm562475.htm . Oncology Center of Excellence . Regenerative medical advanced therapy designation program . Digital health software modifications . Breakthrough devices program . Modernizing review of reusable devices . Patient focused drug development (5 year plan) . Drug development tools (FDA and NIH working together on biomarker qualification efforts) . Continuous manufacturing . Novel clinical trial designs . Combination products . Real world data and real world evidence (use of EHR) 57 . FDA Priority Review voucher to help incentivize rare pediatric diseases How to Partner with the FDA

. The patient voice is a high priority at FDA, so make sure yours is heard! . Disease-specific guidance development . Host or participate in a patient focused drug development workshop (either FDA led or externally-led) . Join the Federal Register https://www.federalregister.gov/ 58 YOUR Voice Matters! 10 Years of Policy & Advocacy Milestones: 2014 – 2018 Sept 2014 Scientific Workshop “Rationalizing Safety Testing to Enable Clinical Studies and Approval in the US for Rare Disease Treatments” to address the offshoring of clinical trials June 2015 FDA Guidance on ERT allows flexibility in Toxicology Requirements bringing down approval time by nearly a year December 7, 2016, the Senate passed the 21st Century Cures Act 94 to 5, encompassing many of the Foundation’s Goals 2016 passed CA NBS Legislation unanimously in 7 months 2017 passed Florida NBS Legislation unanimously in 3 months 2017 Helped Launch the CA State Caucus 2018 First Caucus Briefing secured $2 Million in Funding for whole genome sequencing pilot 2018 FDA Announced More Focused Review Divisions 10 Years of Policy & Advocacy Milestones 2009 – 2013: The First 5 years May 14th 2009 – Launch the CureTheProcess Campaign at the NORD summit in Washington DC 2010 Helped establish the Rare Disease Congressional Caucus Feb. 2012 Held the 1st Lobby Day bringing all Rare Disease Groups together July 2012 FDASIA passed with the 5 rare disease provisions, including the Foundation’s ULTRA/FAST language requiring FDA to issue a guidance on allowing for biomarkers as endpoints for rare diseases Dec 2013 Congress Introduced the OPEN ACT Keep in Touch!

Please sign-up for our email lists at EveryLifeFoundation.org/contact-us and RareAdvocates.org/contact for newsletters, action alerts and event invitations. Like us on Facebook as both the EveryLife Foundation for Rare Diseases and Rare Disease Legislative Advocates. Follow us on Twitter as @EveryLifeOrg and @RareAdvocates. Instagram @rare_advocates Questions? Email [email protected]

The Path to Partnering with Congress: Remarks from Your Representative

Representative Joseph McKenna Massachusetts State Representative 18th Worcester District Group Photo!!

What is on the Legislative Road Ahead? Accelerating Change in the Rare Disease Community

Steve Silvestri Director of Public Policy EveryLife Foundation for Rare Diseases You Have a Constitutional Right to Lobby The First Amendment How to Navigate Congress Your Roadmap to Congress You Don’t have to be an Expert… but Know Enough to be Effective

• Understand the legislative process – How does a bill become a law? • Understand the difference between state & federal legislatures • Understand the difference between the House & Senate – 435 Members in the House of Representatives – 100 Members in the Senate • Know your Committees of jurisdiction & key Members – Committee & Subcommittee Chairs & Ranking Members – House & Senate Leadership • Don’t Be Partisan – Rare Diseases is a bipartisan issue! Role of Political Parties

• Leadership in Congress is based on how many seats your political party controls • Majority Party: Political party that controls the most seats in the House or Senate • Minority Party: Political party that does not control the majority of seats in the House or Senate • The majority party elects the senior leaders of both the House and Senate 116th Congress House Leadership Senate Leadership

• Senate was designed to be more moderate, deliberative and slow • It’s the “cooling off” period for Legislation – Some bills get so cold they die

Senator Mitch McConnell (R-KY) Senator Charles Schumer (D-NY) Congressional Committees that Impact Health & Drug Development

One of the most power committees in Congress • House Energy & Commerce Committee (E&C) – Frank Pallone (New Jersey - 06) - Chairman – Greg Walden (Oregon - 02) Ranking Member • Subcommittee on Health – Anna Eshoo (California - 18) - Chairman – Michael Burgess (Texas - 26) - Ranking Member Senate HELP Committee • Senate Health Education Labor & Pensions Committee

• Subcommittee on Children and Families – Chairman Rand Paul (KY) – Ranking Robert P. Casey, Jr (PA) • Subcommittee on Primary Health and Retirement Security – Chairman Michael Enzi (R-WY) – Ranking Member Bernard Sanders (I-VT) 12 Who to Engage

• Your hometown Member – Issue staff – www.house.gov – www.senate.gov • Relevant Committee Members – Subcommittee staff • Leadership • Government Agencies • State & Local Representatives

– Tip: Staff are an amazing resource, and in-district meetings are often easier to schedule and more beneficial When to Engage • ANYTIME & All The Time!! • Schedule in-district meetings during district work periods. • Pay attention to a bill’s progress through the House/Senate and schedule meetings around key events in this cycle. • Don’t wait until it is too late. Be vocal early about the issue you care about. How to Engage

• In-person meetings – DC – In District • Letters • Articles/Op-Eds • Town Halls • Phone Calls • Social Media • Make it personal Why Relationships Matter Elevating Rare Disease to be a Public Health Crisis WE NEED DATA Rare Disease Burden Study Lewin Group Study Study to be completed in 2019 Analytical approach will estimate direct medical cost and lifetime medical costs of 5 ICD 10 codes: Lysosomal storage diseases E75, E76, E77 Congenital malformation syndromes predominantly associated with short stature Q87.1 Genetic susceptibility to other disease Z15.89 Mitochondrial metabolism disorder unspecified E88.40 Metabolic disorder E88.9 Goal is to measure economic impact of major rare disease on US economy, including direct medical costs, non-medical costs, & indirect costs Specific Tasks

Task 1: Analyze claims data to estimate direct medical costs associated with selected rare diseases Task 2: Use systematic literature review to inform estimates of indirect costs and non-medical costs of rare diseases Findings from both tasks will be combined to derive estimated lifetime costs of selected rare diseases Data Sources

Task 1: Direct Medical Cost of Rare Diseases Using Claims Data Data Sources: Claims data from commercially insured research database (OptumInsight health research database – de-identified Normative Health Information database (dNHI) comprising 100 million lives from 1993 to present (Lewin will only include <65 years) Medicare Standard Analytical Files (SAFs) containing random 5% of Medicare fee-for-service claims (>65 years) Data Sources

Task 2: Estimating Indirect Costs and Non-Medical Costs based on a Systematic Literature Review Data Sources: Will include literature published in the English language (European and US studies) Focus on indirect costs and other non-medical impacts of rare diseases Lewin will work closely with EveryLife to ID relevant areas of focus and research questions Final Report

Report will detail findings from study, including: Results from direct medical cost estimates Results from indirect and non-medical cost estimates Report will describe data, assumptions and methodology along with discussion of study strengths and/or limitations Findings from project will be interpreted within the evidence in existing literature Report will be no more than 25 pages; Executive Summary will also be provided NASEM Study

National Academies of Science, Engineering & Medicine has resources to perform a comprehensive burden study Timeline for this would be several years Would let nation at large understand true economic and societal burden of estimated 30 million Americans suffering from rare diseases Would demonstrate public health & resource need related to rare diseases Study will build on foundation laid by Lewin Report Congressional Request

$1.5 million appropriation in the Fiscal Year 2020 Labor, Health and Human Services, Education Appropriations bill and direction in report language for the National Academies of Sciences, Engineering and Medicine to conduct a study on the burden of rare disease. Appropriations Timeline

March: President released Budget Proposal March/April: Appropriations Committee hearings • Deadline to submit appropriations requests May: Appropriations bills released and marked up by Committee June: Bills considered, amended, voted on by full House of Representatives and Senate September: Final appropriations bills signed into law by the President Stakeholder Engagement Submit request to Members of Congress

Tell your story and why the burden study matters

Request Members of Congress sign on to Dear Colleague Letter led by Rare Disease Caucus

Sign on to stakeholder letter to Congress

Spread the word with policy makers and your community! Key Members of Congress

House Senate House Appropriations Chair: Nita Lowey (D-NY) Senate Chair: Richard Shelby (R-AL) House Ranking Member: Kay Granger (R-TX) Senate RM: Patrick Leahy (D-VT)

L-HHS Subcommittee Chair: Rosa DeLauro (D-CT) Subcommittee Chair: Roy Blunt (R-MO) Subcommittee RM: Tom Cole (R-OK) Subcommittee RM: Patty Murray (D-WA)

Lucille Roybal-Allard (D-CA) Lamar Alexander (R-TN) Barbara Lee (D-TX) Lindsey Graham (R-SC) Mark Pocan (D-WI) Jerry Moran (R-KS) Katherine Clark (D-MA) Shelley Moore Capito (R-WV) Lois Frankel (D-NY) John Kennedy (R-LA) Cheri Bustos (D-IL) Cindy Hyde- Smith (R-MS) Bonnie Watson Coleman (D-NJ) Marco Rubio (R-FL) Andy Harris (R-MD) James Lankford (R-OK) Jaime Herrera Beutler (R-WA) Dick Durbin (D-IL) John Moolenaar (R-MI) Jack Reed (D-RI) Tom Graves (R-LA) Jeanne Shaheen (D-NH) Jeff Merkley (D-OR) Brian Schatz (D-HI) Tammy Baldwin (D-WI) Chris Murphy (D-CT) Joe Manchin (D-WV) Practice Your Pitch

“Stories constitute the single most powerful weapon in a leader’s arsenal” – Dr. Howard Gardner Know Your Audience: The Legislators Find ways to connect!

What political party are they affiliated with? What committees do they participate in? What legislation have they supported? What do YOU have in common with them?

Remember: Our issues are bipartisan! We are the RARE Party! Telling Your Story

Know the right ingredients: Problem Solution Call to Action Practice Makes Perfect

It is natural to try and tell your whole story, but you will have better impact when you tell part of your story with a clear call to action.

Practice with friends Practice in the mirror Practice recording on your phone Use Social Media to Reach Policymakers

• Thank legislators by using their official Twitter handle for a vote, meeting or even a thoughtful response to an inquiry. Include a photo of the meeting! • Engage in discussions about legislative initiatives. Call to Action – Action Alert Rare Disease Burden Study Leave a Message for your MOC’s One Number – 3 Contacts! Dial 1-844-872-0234 and wait for the automated voice machine Press 1 and enter your zip code XXXXX. This will connect you to your first Senator Sample script Senate ASK: Rare Disease Burden Study Press * if you wish to skip an office Sample script House ASK: Rare Disease Burden Study Massachusetts Senate Speed Dial & Twitter Senator Elizabeth Warren Main: (202) 224-4543 @SenWarren

• Senator Ed Markey Main: (202)224-2742 @SenMarkey MA Representatives Speed Dial & Twitter

Rep. Richard Neal @RepRichardNeal 202-225-5601 Rep. James “Jim” McGovern @RepMcGovern 202-225-5759 Rep. Lori Trahan @RepLoriTrahan 202-225-3411 Rep. Joseph Kennedy III @RepJoeKennedy 202-225-5931 Rep. Katherine Clark @RepKClark 202-225-2836 Rep. Seth Moulton @teammoulton 202-225-8020 Rep. Ayanna Pressley @RepPressley 202-225-5111 Rep. Stephen Lynch @RepStephenLynch 202-225-8273 Rep. William Keating @USRepKeating 202-225-3111 Rare Disease State Awareness Opportunities

Ensuring patients have a voice in state policy • Rare Disease Caucus • Rare Disease State Advisory Councils • Rare Disease State House Events and Lobby Days Rare Disease Caucus

• Federal Level: Bicameral Bipartisan Rare Disease Congressional Caucus – Holds quarterly Hill briefings on rare disease policy issues to educate Members and staff about important policy issues – Ensures patients have a voice in shaping policy – Opportunity for Members to work on bipartisan lifesaving legislation – Opportunity for members to engage with the rare disease ecosystem including patients, academia, hospitals, biopharmaceutical industry, investors and government agencies • State Level: – Pennsylvania – California State Rare Disease Advisory Councils

● Five states have created Rare Disease Advisory Councils (aka Commission or Task Force)  Alabama, Connecticut, Illinois, North Carolina & Rhode Island

● These are official advisory bodies intended to increase awareness of rare disease within state government. Some commons features:  Require that a rare disease survivor and caregiver be on the council itself  Broad representation from different medical disciplines and state departments  Specific purpose is to make recommendations to the governor and/or legislature about ways to improve care for rare disease community  Often features a collaboration with local universities

● In 2018, rare disease advocates are seeking to build councils in new states:  Georgia, Kansas, Massachusetts  Michigan bill filed (H. 5983)  Kentucky and Minnesota passed bill but vetoed by Governors

For more information, please contact Tim Boyd at NORD ([email protected]) Massachusetts Rare Disease Advisory Council Massachusetts Newborn Screening Rare Disease Day 2019 • Rare Disease Day is a great opportunity to raise awareness among legislators and give the rare community a platform to be heard • In 2016 rare disease advocates in partnership with NORD hosted events in 36 states – The goal is to leverage these events on specific state policy issues – In 2017 - 2019, many of the events functioned as “Hill days” on specific legislative priorities – We need events at all 50 state houses! – For more info on State House events, visit www.rarediseaseday.us • Personal Goal: 1000 rare disease advocates in DC & 500 at every state house!! Want To Join the Action on Capitol Hill?

• RDLA’s Advocacy Tools • In-District Lobby Days • Rare Disease Week on Capitol Hill 2020 So, You Want to Be an Advocate? Sign up for monthly newsletters, action alerts and invitations to the monthly webinars and other FREE events at RareAdvocates.org. Stay Informed on Key Policy Issues

• Monthly interactive webinars highlight urgent state & federal legislation • RareAdvocates.org serves as a legislative clearinghouse • Monthly e-blast shares latest policy news, action alerts & events • Online Calendar lists policy events and legislative deadlines • Facebook & Twitter accounts feature breaking

news & action alerts 46 From July 29th through September 4th , rare disease advocates from across the country will meet with Members of Congress in their local offices to advocate for legislation benefiting the rare disease community.

Registration for RARE Across America will open May 8th. Like all of our programs, it is free for patients, caregivers and other rare advocates. Goals of RARE Across America

• Strengthen your relationships with Members of Congress and staff • Advance legislation that would benefit the rare disease community

A best-practice of effective advocacy is to make your friends before you need them! We NEED to gain allies in both parties who will champion rare disease causes. How To Participate

• You specify the dates that you are not available within the timeframe (July 29th – September 4th). • You also specify how far you are able to travel for meetings with your Representative and Senators. • We retained Advocacy Associates to schedule the meetings and coordinate with each advocate. • They will do their best to accommodate as many constituents as possible when scheduling each meeting with Congressional offices. • Please DO NOT schedule your own meetings, we will take care of that for you!!! Preparing for RARE Across America • Everyone who registers for RARE Across America will be invited to a training webinar July 25th. • The webinar will provide detailed information including tips on how to make your meetings successful and what to research prior to your meetings. • Questions will be welcome during the webinar! Don’t be shy. • If you can’t join us, you’ll be able to access the archived webinar within a day at http://rareadvocates.org/in- district-lobby-days/ Rare Disease Week on Capitol Hill

2019 was our eighth and most successful year!

800+ patients, caregivers, researchers and other advocates joined us for at least one event during the week! Rare Disease Week on Capitol Hill 2020

When: February 25th through February 28th Who: Rare disease patients, caregivers and other advocates including physicians What: Series of events aimed at empowering patients Where: Washington, D.C. Cost: FREE for advocates to attend Rare Disease Week on Capitol Hill

Why should YOU attend? • Learn about key legislative initiatives with the potential to benefit rare disease patients. • Educate legislators on the unmet needs of rare disease community and build (or strengthen) your relationship with them. • Raise awareness of your specific rare disease and rare diseases in general. • Network with other rare disease advocates, Members of Congress and staff, leaders at FDA and NIH, and representatives of biopharmaceutical companies. Financial Assistance Available through Rare Giving

The Foundation provided nearly $77,000 in travel stipends this year to enable over 100 patients and caregivers to attend who wouldn’t have been otherwise able to do so. • Advocates in Alaska, Hawaii and Puerto Rico received $1,000. • Advocates in Maryland and Virginia received $400. • Advocates in the rest of the continental US received $800. Applications for travel stipends for Rare Disease Week on Capitol Hill 2020 will open in October. Collaboration is Key to Saving Lives – Join Us

WE ARE BUILDING A MOVEMENT. EVERY VOICE MATTERS. Being Seen and Heard: Social Media Strategies to Click and Connect

Colleen Young Community Director Mayo Clinic Connect Being Seen and Heard: Social Media Strategies to Click and Connect

Colleen Young, Community Director, Mayo Clinic Connect @colleen_young

From Connection to Engagement & Action In today’s presentation, we’ll explore how you can:

• Reach and engage your ideal audience • Build a social media strategy that works • Drive traffic to your website or blog • Learn, inform and influence

@colleen_young What about you?

What would you most like to learn about social media today? Social Networking … is the use of technology combined with social interaction to • Connect • Learn • Collaborate • Create • Inform • Influence @colleen_young Strategy: Ws & an H

• Why do you want to use social media? • Who do you want to connect with and why? • Where are they? • What do you want to learn, share, influence? • What do you want them to do and feel? • How do they want to engage?

@colleen_young Goal: Why do you want to use social media?

• What do you want to accomplish? • Educate? • Influence? • Advocate? • Organize? • How will you know you’ve succeeded?

@colleen_young Audience: Who do you want to reach?

• Who do you want to influence? • Elected officials • Researchers • Physicians • Parents • Others with the disease • Social media influencers P.S. Everybody is not a good answer.

@colleen_young Get to know them

• Converse with “harder to reach” people and organizations; network with people you otherwise don’t have access to

• Learn the language of the people you wish to connect with

• Find people where they are and when they are there (scheduling posts)

@colleen_young Where are they? Choosing the right platform

• Is your audience already using it?

• Does it have the communication capabilities you need?

• Do you have the time to manage it?

@colleen_young Social Media Use 2018

• 73% of U.S. adults use YouTube

• 68% are Facebook users

• Not the whole story

Pew Research Center, March 2018, “Social Media Use in 2018” http://www.pewinternet.org/2018/03/01/social-media-use-in-2018/ Demographic differences Facebook: Pages vs. Groups Facebook Live and Periscope

• Let you broadcast live, interactive video to the world

• No FCC license required: smart phone or web cam

@colleen_young Twitter: Powerful Tool for New Connections

• Most posts are public

• Followers vs. Friends

• Hashtags help you find users with common interests

@colleen_young Know your hashtags, communities & influencers LinkedIn: Professional Networking YouTube: Search-Optimized Video

@colleen_young Visual Social Media: Instagram & Pinterest

@colleen_young Importance of Images and Video Don’t just pitch the media. Be the media! Blogging

• Free blog setup and hosting

• Upgrade options

• Powerful SEO benefits

• Link from social accounts and embed assets (e.g. videos, presentations)

@colleen_young Measure for success

• Unlimited measurement opportunities in digital

• Focus on those that relate to strategy and goals

• Examples: • Clicks, comments, engagements • Traffic or unique visitors to home base • Email newsletter sign-ups/Activists recruited • Content shares

@colleen_young Summary

• Identify why, what, who, where and how. • Plan for success. • Never go it alone. Collaborate. • Be creative. • Measure, evaluate and course correct. • Learn and plan again.

@colleen_young Resources for Your Social Media Exploration @colleen_young Evolving Behaviors & Roles

MENTOR Regularly posts, learns, supports, teaches • Welcomes and checks on members encourages participation • Connects people, develops relationships and creates topical discussions • Is rewarded

HELPER Participates, learns and supports • Posts questions • Shares experiences • Replies to other members, offers support INFO SEEKER Participates and learns • Posts question(s) • Seeks answers and support

OBSERVER Follows and learns • Reads • Follows • Likes Mayo Clinic CONNECTors

Moderators

Volunteer Mentors

@colleen_young Improving patient experience with members The Mayo Clinic Social Media Network Free Membership: SocialMedia.MayoClinic.org

Social Media Certification

35 @colleen_young

Questions?

Mayo Clinic Social Media Network socialmedia.mayoclinic.org

Maintain Your Ride: Self-care Check-up

Deborah Jacobs, LMHC, ATR-BC Licensed Counselor and Expressive Art Therapist Accelerate Your Mission: Your Journey Begins with a Single Step RARE on the Road Takeaway (Postcard Project) Write down two to three takeaways from this event:

• Things you learned • Feelings about the meeting and community • Reminders

We will mail postcards back to you in 4-6 weeks. Enter a chance to win a trip to the Global Genes Summit and Rare Disease Week on Capitol Hill!!

Surveys must be filled out before you leave. Thank you!