Rare Disease Week on Capital Hill - February 25th - 28th, 2020

PRESENTED BY

FEBRUARY

25-28

2020

Washington, DC

#RareDC2020 @RareAdvocates

POWERED BY THE EVERYLIFE FOUNDATION

Thank You to Our Generous 2020 Rare Disease Week on Capitol Hill Sponsors

PRESIDENTIAL

LEADERSHIP

RANKING

CHAMPION

CO-SPONSORS

GRASSROOTS

THIS HANDBOOK IS GENEROUSLY UNDERWRITTEN BY:

Table of Contents Introduction Welcome 1 EveryLife Foundation Team 2 EveryLife Foundation Board of Directors 3 Schedule of Events 4-5 Tuesday Congressional Caucus Briefing Documentary Screening

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Wednesday Advocate Services 8 Legislative Conference Agenda 9-13 Fast Forward for Rare 14 Legislative Conference Guest Speakers 15-26 YARR Young Adult Meetup 27 Thursday Hill Day Breakfast 28 Hill Day Issue Information 29-35 Congressional Meeting Tips 36 Social Media Advocacy Tips 37 Rare Artist Reception 38 Friday Rare Disease Day at NIH

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About Rare on the Road 39 Rare Across America 40 Rare Giving 40 Rare Voice 41 Newborn Screening 41 Glossary Congressional Terms 42 U.S. Federal Agencies 43 Legislative Process 44 Sponsors 45-48 EveryLife Foundation 49 Rare Disease Legislative Advocates 49 Notes 50-51 Maps Accessibility Resources 52 Metro 53 Reagan Center 54 Capitol Hill Resources 55 US Capitol 56

#RareDC2020

Welcome to Rare Disease Week

Dear Rare Disease Advocate, Thank you for joining us for Rare Disease Week on Capitol Hill. This event is a success because of your participation and determination to be an effective advocate for yourself or loved ones. We are honored that you have chosen to advocate with us in DC. As you know, Washington is often gridlocked with partisan bickering. However, rare diseases are not a partisan issue. Rare diseases do not discriminate; they affect Republicans and Democrats. We are not elephants or donkeys, we are zebras. We are the rare party and we are 30 million strong!

It is truly an honor to help our community by giving patients a voice on Capitol Hill. We hope this week is everything you imagined and that you continue to use your voices year-round to ensure rare disease patients are heard.

Sincerely, The EveryLife Foundation Team

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EveryLife Foundation for Rare Diseases Team OPERATIONS Julia Jenkins Executive Director jjenkins@everylifefoundation.org

Lauren Grinnals Rare Hub Office Manager lgrinnals@everylifefoundation.org

Megan Pinegar Senior Director of Operations and Human Resources mpinegar@everylifefoundation.org

Alyssa Terwall Special Events Manager aterwall@everylifefoundation.org

DEVELOPMENT Carol Kennedy Chief Development Officer ckennedy@everylifefoundation.org

Ted Brasfield Director of Development tbrasfield@everylifefoundation.org

POLICY & ADVOCACY Annie Kennedy Chief of Policy and Advocacy akennedy@everylifefoundation.org

Kylie Barber Medical Foods Policy Fellow kbarber@everylifefoundation.org

Steve Silvestri Director of Public Policy ssilvestri@everylifefoundation.org

Dylan Simon Newborn Screening Policy Fellow dsimon@everylifefoundation.org

Shannon von Felden Director of Rare Disease Legislative Advocates svonfelden@everylifefoundation.org

PUBLIC RELATIONS & PATIENT ENGAGEMENT

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Britta Dornan Senior Director of Public Relations and Patient Engagement bdornan@everylifefoundation.org

Lindsey Cundiff Associate Director of Patient Engagement lcundiff@everylifefoundation.org

Brenda Colmenares Communications Manager bcolmenares@everylifefoundation.org

Mary Morlino Patient Engagement Consultant mmorlino@everylifefoundation.org

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EveryLife Foundation for Rare Diseases Board of Directors The EveryLife Foundation Board of Directors is comprised of a diverse group of individuals who are both personally and professionally dedicated to the development of treatment and diagnostic opportunities for rare disease patients. With decades of experience in the government, nonprofit, finance, science, medicine, industry, and academic sectors, EveryLife’s Board provides valuable guidance on policy. Several of our board members are the parents of children with a rare disease, enabling them to offer firsthand knowledge of the challenges facing the rare disease community.

Mark Dant, Chair Executive Director, Ryan Foundation Frank Sasinowski, MS, MPH, JD, Vice Chair Director, Hyman, Phelps & McNamara P.C. Julia Jenkins, Secretary Executive Director, EveryLife Foundation Vicki Seyfert-Margolis, PhD, Treasurer Founder and CEO, MyOwnMed Ritu Baral, Member Managing Director/Senior Biotechnology Analyst, Cowen and Company

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Jennifer Bernstein, Member Executive Vice President, Horizon Government Affairs Richard S. Finkel, Member Director of Experimental Neurotherapeutics in Translational Neuroscience Program, St. Jude Amrit Ray, MD, MBA, Member Global President, Research, Development & Medical, Upjohn, Pfizer, Inc. Emil Kakkis, MD, PhD, Founder President/CEO, Ultragenyx

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Rare Disease Week on Capitol Hill 2020 Events TUESDAY, FEBRUARY 25 1:00 PM – 2:00 PM Rare Disease Congressional Caucus Briefing and Group Photo Capitol Hill, Russell Senate Office Building, Room 325 Washington, DC 20004 Metro Stop: Union Station (Senate side) or Capitol South (House side)

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Presented by:

2:30 PM - 4:30 PM Patient Advocacy Listening Session Hosted by the NYU Grossman School of Medicine Working Group on Pediatric Gene Therapy & Medical Ethics 1012 14th Street, NW, Washington, DC Email jamie.webb@nyu.edu for more information

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5:30 PM – 9:00 PM Rare Disease Documentary Screening Ronald Reagan Building and International Trade Center 1300 Pennsylvania Ave NW, Washington, DC 20004 Metro Stop: Federal Triangle

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Presented by:

WEDNESDAY, FEBRUARY 26 8:00 AM – 4:45 PM Legislative Conference Ronald Reagan Building and International Trade Center - Atrium Hall 1300 Pennsylvania Ave NW, Washington, DC 20004 Metro Stop: Federal Triangle

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6:00 PM - 8:00 PM Young Adult Meetup Rare Hub, 1012 14th Street NW, Suite 500, Washington, DC 20005 Metro Stop: McPherson Square or Farragut North

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Presented by:

Presented by:

5:30 PM – 7:30 PM Research!America Reception “Celebrating Rare Disease Research Advocates: Strong Together”* The W Hotel , 515 15th Street, NW, Washington, DC 20004 Metro Stop: Federal Triangle or Metro Center

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*Hosted by an outside organization and listed for informational purposes.

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Rare Disease Week on Capitol Hill 2020 Events THURSDAY, FEBRUARY 27 7:00 AM – 8:30 AM Hill Day Breakfast Ronald Reagan Building and International Trade Center - Atrium Ballroom 1300 Pennsylvania Ave NW, Washington, DC 20004 Metro Stop: Federal Triangle

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9:00 AM – 5:00 PM Scheduled Meetings with Members of the House and Senate on Capitol Hill Capitol Hill, Washington, DC 20004

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10:30 AM - 4:00 PM Available Advocate Service: Hospitality Room Capitol Hill, Russell Senate Office Building, Room 485 Washington, DC 20004 Metro Stop: Union Station (Senate side) or Capitol South (House side)

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5:00 PM – 7:00 PM Rare Artist Reception Capitol Hill, Hart Senate Office Building, Room 902 Washington, DC 20004 (Use “North” elevators) Metro Stop: Union Station (Senate side) or Capitol South (House side)

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Presented by:

Presented by:

FRIDAY, FEBRUARY 28 8:30 AM – 4:00 PM Rare Disease Day at the National Institutes of Health* National Institutes of Health 9000 Rockville Pike, Building 45, Main Auditorium, Natcher Conference Center, Bethesda, MD 20892 Metro Stop: Medical Center Station To learn more visit: events-support.com/events/NIH_Rare_Disease_Day

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*Hosted by an outside organization and listed for informational purposes.

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Rare Disease Congressional Caucus Briefing

TUESDAY, FEBRUARY 25 1:00 PM – 2:00 PM

ACCELERATING RARE DISEASE INNOVATION ACROSS THE PIPELINE: FROM DISCOVERY TO ACCESS

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Russell Senate Office Building, Room 325 Rare Disease Legislative Advocates and the Rare Disease Congressional Caucus invite you to a Rare Disease briefing.

Presented by:

SPEAKER PANEL Moderator: Frank Sasinowski, MS, MPH, JD, Director, Hyman, Phelps & McNamara Introduction: Eric Dube, Ph.D., President and CEO, Retrophin Welcome: Representative Gus Bilirakis (FL), Co-Chair of the Rare Disease Congressional Caucus

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Rare Disease Diagnostics Adeola Adesina, Legislative Assistant, Office of Representative Eric Swalwell Debbie Jessup, Policy Advisor, Office of Representative Lucille Roybal-Allard

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Regulatory Therapeutic Challenges and Solutions Thomas Rice, Legislative Director, Office of Representative Michael McCaul Larry Bauer, Senior Regulatory Drug Expert, Hyman, Phelps, & McNamara

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Rare Disease Documentary Screening TUESDAY, FEBRUARY 25

Presented by:

5:30 PM - 9:00 PM Ronald Reagan Building, 1300 Pennsylvania Ave NW, Washington, DC 20004 Tin Soldiers shines a light on the victims of a debilitating disease, the hunt to find a cure, and the search for those still locked in darkness.

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GUEST PANEL Moderator: Mark Dant, Chair, Board of Directors, EveryLife Foundation for Rare Diseases; Founder, The Ryan Foundation Christian Rubio, VP of Strategic Advancement, Global Genes Amanda Cali, Executive Producer, Tin Soldiers documentary; Trustee, The Radiant Hope Foundation; FOP Parent and Advocate Melissa Bryce Gamble, Co-Founder and Executive Director, Global Foundation for Peroxisomal Disorders Carlos Cristi, IgA Nephropathy (Berger’s Disease) Patient Advocate Eve Dryer, Executive Director, Patient Advocacy, Retrophin

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Legislative Conference Advocate Services Please take advantage of these resources generously provided by our sponsors. See map on page 54 for reference.

Nursing Room Atrium Ballroom A A comfortable private room available for any mother’s needs; located within Atrium Ballroom A, on the left side.

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Family Room Atrium Gallery The room is located in the Atrium Gallery and intended for families to comfortably gather while being able to enjoy any of the presentations happening in the main hall via livestream.

The Family Room will be available all day except from 1:15 p.m. – 2:15 p.m. when families are required to attend the “Preparing for Successful Meetings” session. We also encourage families to eat in the Atrium with their state teams.

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Medical Station

Presented by:

Atrium Gallery Inside the Family Room there will be a nurse available if any medical issues arise.

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Nap Nook

Presented by:

Atrium Gallery For those in need of rest, the nap nook is a comfortable napping space located just outside the Atrium Gallery.

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All-Day Beverage Station

Presented by:

Atrium Drinks available throughout the Legislative Conference.

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Legislative Conference Agenda

WEDNESDAY, FEBRUARY 26

Presented by:

8:00 AM – 4:45 PM Ronald Reagan Building and International Trade Center - Atrium 1300 Pennsylvania Ave NW, Washington, DC 20004

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REGISTRATION AND BREAKFAST 8:00 AM – 8:45AM Atrium

Breakfast Presented by:

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WELCOME 8:45 AM – 9:30 AM Atrium Hall and via Livestream Welcome: Brent Fisk, Director of Federal Government Affairs and Policy, Horizon Shannon von Felden, Director of Rare Disease Legislative Advocates, EveryLife Foundation for Rare Diseases Keynote: Mark Dant, Chair, Board of Directors, EveryLife Foundation for Rare Diseases

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FAST FORWARD FOR RARE VIDEO SUITE 9:30 AM – 4:45 PM (CLOSED 1:15 PM - 2:15 PM) Atrium Ballroom Coat Room

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HEALTH POLICY FORECAST 9:30 AM – 10:00 AM Atrium Hall and via Livestream Healthcare continues to be one of the most challenging issues for Americans. Hear from former Congressional staffers about how Congress will impact healthcare policy in 2020, and how you can help shape these trends.

Introduction: Catherine Jackson, RN, BSN, MPH, Director of Advocacy Relations, Mallinckrodt Pharmaceuticals Moderator: Cheryl Jaeger, Principal, Williams and Jensen Remy Brim, Ph.D., Vice President, BGR Government Relations Mary-Sumpter Lapinski, Vice President, Global Government Affairs, Greenwich Biosciences

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Wifi Network Access Network Name: Ronald Reagan Building Wifi Username: RareDC2020 Password: Takeda Presented by:

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2020 LEGISLATIVE ASKS 10:00 AM - 11:00 AM Atrium Hall and via Livestream We encourage advocates to make one specific “ask” during their meetings with Members of Congress.

The panel highlights some potential “asks” that are priorities for the rare disease patient community.

Moderator: Nicholas Manetto, Principal, Faegre Drinker Consulting Shayne Woods, Senior Policy Advisor, Office of Representative Gus Bilirakis Dylan Simon, Newborn Screening Fellow, EveryLife Foundation for Rare Diseases Wen-Hann Tan, BMBS, Assistant Professor of Pediatrics, Harvard Medical School Caitlin Van Sant, Senior Policy Advisor, Office of Representative G.K. Butterfield Ryan Fischer, Senior Vice President of Community Engagement, Parent Project Muscular Dystrophy Kylie Barber, Medical Foods Policy Fellow, National PKU Alliance

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BREAK 11:00 AM – 11:15 AM Atrium

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BREAKOUT SESSIONS 11:15 AM – 12:00 PM

TRACK 1: DEEP DIVE POLICY - ACCELERATING TREATMENTS FOR RARE DISEASES

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Atrium Hall and via Livestream Learn how a Rare Disease Center of Excellence within the FDA would elevate rare disease expertise within the agency and help to remove communication barriers across centers and divisions to accelerate and improve rare disease therapy development.

Moderator: Annie Kennedy, Chief of Policy and Advocacy, EveryLife Foundation for Rare Diseases Steve Silvestri, Director of Public Policy, EveryLife Foundation for Rare Diseases Larry Bauer, Senior Regulatory Drug Expert, Hyman, Phelps, & McNamara Lisa Carlton, Ph.D., Director of Regulatory Affairs, REGENXBIO Nicholas Manetto, Principal, Faegre Drinker Consulting

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TRACK 2: HOW TO ENGAGE IN STATE ADVOCACY

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Atrium Ballroom A This session is designed for advocates who want to become more involved in rare disease advocacy in their state and build opportunities at the state level.

Moderator: Julia Jenkins, Executive Director, EveryLife Foundation for Rare Diseases Angela Ramirez Holmes, Founder and President, CAL RARE Khrystal Davis, Founder, Texas Rare Alliance Swapna Kakani, Founder and Director, Alabama Rare Nikia Vaughan, Cimone and Friends Melissa Bowie, Senior Director, State Government Affairs, Takeda

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TRACK 3: LOBBYING 101 - MOCK MEETING*

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Atrium Ballroom B This is a must-attend session for those individuals who are new to advocacy. Hear from professional lobbyists about how legislation is passed and how to be most impactful in Congressional meetings.

Seasoned advocates will present some dos and don’ts in a mock Congressional meeting skit. A great opportunity to get questions answered by experienced rare disease advocates.

Jennifer Bernstein, Vice President, Horizon Government Affairs Ryan Fischer, Senior Vice President of Community Engagement, Parent Project Muscular Dystrophy Tabatha Mitchell, Emes Army Monica Weldon, Bridge the Gap—Syngap

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GROUP PHOTO 12:05 PM Atrium Stairs

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NETWORKING LUNCH

Presented by:

12:30 PM – 1:15PM Atrium We encourage families to eat in the Atrium with their state team.

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PREPARING FOR SUCCESSFUL MEETINGS* 1:15 PM – 2:15 PM Atrium Hall & via Livestream *Mandatory for advocates participating in Hill Day meetings. (Family Room will be closed at this time) Advocates receive their Hill Day schedules and strategize with their teams to make the most of their meetings on Capitol Hill.

Mike Gula, Co-Founder, Prime Advocacy Shannon von Felden, Director of Rare Disease Legislative Advocates, EveryLife Foundation for Rare Diseases

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NYU GROSSMAN SCHOOL OF MEDICINE WORKING GROUP ON PEDIATRIC GENE THERAPY AND MEDICAL ETHICS (PGTME) INDUSTRY LISTENING SESSION BREAKOUT 1:15 PM – 2:15 PM Atrium Ballroom A

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SNACK BREAK

Presented by:

2:15 PM – 2:45 PM

#RareDC2020 Expectations “I am very excited to get to meet other advocates like myself, who can as a team make the biggest impact! I want to learn everything I possibly can and do well when sharing my story and my priorities.”

Robin Alexix - NY Like • Reply • Message

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BREAKOUT SESSIONS 2:45 – 3:30 PM

TRACK 1: DEEP DIVE POLICY – IMPROVING ACCESS TO TREATMENTS FOR RARE DISEASES

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Presented by:

Atrium Hall & via Livestream As more treatments for rare diseases are moving into regulatory review at the FDA, there is an increasing need to ensure that patient-centered data is used to inform decision-making in the payer environment. This session will explore opportunities for innovation and outcomes-based data collection - across stakeholders - to inform decision making and facilitate favorable access environments for rare disease patients.

Introduction: James Wu, M.Sc., MPH, Principal Health Economist, Global Health Economics, Amgen Moderator: Jennifer Bernstein, Vice President, Horizon Government Affairs Ryan Fischer, Senior Vice President of Community Engagement, Parent Project Muscular Dystrophy Dr. Sean Tunis, MD, MSc., Principal, Rubix Health and Senior Advisor, FDA Vicki Seyfert-Margolis, PhD, Founder & CEO MyOwnMed, former Senior Advisor for Science Innovation and Policy in the Office of the Commissioner, FDA

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TRACK 2: ADVOCACY FOR YOUNG ADULTS: YOUNG ADULT REPRESENTATIVES OF RDLA (YARR)

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Atrium Ballroom A This session is for young adults ages 16 – 30 years old. In this session young advocates will learn how to make a difference on Capitol Hill and how to have an effective meeting with their Member of Congress.

Allison Herrity, YARR Representative Dan Pezzetta, YARR Representative

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TRACK 3: LOBBYING 101 - PRACTICE YOUR PITCH

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Atrium Ballroom B This session is for new advocates who are looking for some extra practice before their Hill meetings.

Advocates will learn how to tell their story, cohesively tie in their “ask”, refine their elevator pitch and finetune their legislative talking points.

Chris Smith, President and Chief Executive Officer, SmithSolve Joel White, Founder and President, Horizon Government Affairs

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TRACK 4: MEDICAL FOODS EQUITY ACT 2:45 PM – 4:30 PM Continental C This session is for advocates interested in supporting the Medical Nutrition Equity Act. Sarah Chamberlin, National PKU News Kristen Vanags, Georgia PKU Connect Amy Oliver, Intermountain PKU and Allied Disorders

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BREAK 3:30 – 3:45 PM Atrium

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BREAKOUT SESSIONS 3:45 PM – 4:30 PM

TRACK 1: DEEP DIVE POLICY—HOW FEDERAL FUNDING SUPPORTS RARE DISEASE PATIENTS

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Atrium Hall & via Livestream This session will explore the appropriations process, how Congress decides to fund programs, and the different programs of importance to the rare disease community.

Moderator: Steve Silvestri, Director of Public Policy, EveryLife Foundation for Rare Diseases Mark Vieth, Senior Vice President, CRD Associates Eric Gascho, Vice President, Policy and Government Affairs, National Health Council Wen-Hann Tan, BMBS, Assistant Professor of Pediatrics, Harvard Medical School

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TRACK 2: ADVOCACY FOR YOUNG ADULTS - PRACTICE MEETINGS WITH CONGRESSIONAL STAFF

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Atrium Ballroom A This session is for young adults (ages 16-30) to practice telling their stories and asks to Congressional staffers. Attendees will learn tips and tricks for successful meetings on the Hill from seasoned advocates.

Moderator: Stefanos Koutsoukos, YARR Representative Juan Gomez, Legislative Aide, Office of Senator Corey Booker (NJ) Katherine Sears, Communications Director, Office of Representative David Joyce (OH) Shannon von Felden, Director of Rare Disease Legislative Advocates, EveryLife Foundation for Rare Diseases

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TRACK 3: HOT LEGISLATIVE TOPICS

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Atrium Ballroom B Learn about hot legislative topics on Capitol Hill from Hill staffers and advocates and how you can help move the needle on these issues.

Moderator: Ritu Baral, Managing Director, Senior Biotechnology Analyst, Cowen & Co. Adeola Adesina, Legislative Assistant, Office of Representative Eric Swalwell Dylan Simon, Newborn Screening Fellow, EveryLife Foundation for Rare Diseases Luke Hatzis, Principal, Capitol Counsel Jennifer Dexter, Director of Policy, National Health Council Annie Kennedy, Chief of Policy and Advocacy, EveryLife Foundation for Rare Diseases

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CLOSING REMARKS

The Family Room Ronald Reagan Building, Atrium Gallery The Family Room will be available for families all day except from 1:15 p.m. – 2:15 p.m., when families are required to attend the “Preparing for Successful Meetings” session.

We also encourage families to eat in the Atrium with their state teams.

4:30 PM - 4:45 PM

NOTE: Agenda schedule is subject to change.

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Every Voice Matters!

Come practice your pitch at Fast Forward for RARE, a new video suite hosted by RDLA in partnership with the communication specialists at SmithSolve. Receive personalized feedback to help you tell your story with impact and inspire action.

Stop

• Visit our video suite outside the Atrium Ballroom at the Rare Disease Week Legislative Conference on Wed., Feb. 26

Record

• Share your story on camera • Get immediate feedback and tips

Fast Forward for Rare • Apply what you learn on Capitol Hill and beyond!

• Receive a digital copy of your video and photo to share

Together We Will

Meet Your Coaches

• Advance important legislation • Improve understanding of rare diseases • Support development of new treatments Katie Burns

Britta Dornan

Dan Salvo

Mary Morlino

Chris Smith

Fast Forward for RARE is sponsored by SmithSolve & Rare Disease Legislative Advocates. SmithSolve specializes in rare disease communication, patient advocacy, and community engagement.

If you are unable to visit our video suite, please attend the Practice Your Pitch breakout led by Chris Smith and Joel White at 2:45 p.m. in Atrium Ballroom B. 14

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Legislative Conference Guest Speakers Adeola Adesina Legislative Assistant, Office of Representative Eric Swalwell Originally from Arlington, Texas, Adeola earned her BS in communications and journalism from Texas A&M University and currently pursuing a master’s degree at Georgetown University. Adeola Adesina is the healthcare legislative assistant for Congressman Eric Swalwell. She previously served as the healthcare legislative correspondent for Democratic Leader Chuck Schumer. Her internships include the offices of Reps. David Scott and Bill Flores, Texas State Rep. John Raney, and Bryan-College Station Chamber of Commerce. In her free time, Adeola likes to attend music festivals, brew kombucha, and tend to her unsuccessful garden.

Ritu Baral Managing Director, Senior Biotechnology Analyst, Cowen and Company Ritu has been in the financial investment industry for most of her career. She has been with Cowen & Company since August of 2014 and has been on the Board of Directors of the EveryLife Foundation since September of that year. Ms. Baral was Managing Director and Senior Analyst at Biotechnology Equity Research from 2009 until 2014 at which time Biotechnology Research acquired Canaccord Genuity where she was a Senior Associate from 2007 until 2009. Ms. Baral also served on the Board of Directors of the Pulmonary Fibrosis Foundation from 2011 until 2014.

Kylie Barber Medical Foods Policy Fellow, National PKU Alliance Originally from Arizona, Kylie Barber moved to Washington D.C. after completing her graduate school programs in Chicago. Kylie holds a Master of Jurisprudence in Children’s Law and Policy, as

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well as a Master of Science in Child Development with specializations in Infancy and New Language Learners. Kylie has completed work in early childhood education policy and child health policy on both state and federal levels. Kylie is currently acting as the Medical Foods Policy Fellow at the EveryLife Foundation for Rare Diseases where she advocates on behalf of the National PKU Alliance and develops advocacy strategies for the passage of the Medical Nutrition Equity Act (MNEA).

Larry Bauer Sr. Regulatory Drug Expert, Hyman, Phelps, and McNamara, P.C. Larry assists medical product industry and patient advocacy organization clients in a wide range of regulatory matters, including new drug and biologic development and approval issues. Prior to this position he worked at the FDA in CDER’s Rare Diseases Program working on policy, education, and science related to rare disease drug development. He has expertise in Rare Pediatric Disease priority review vouchers and designations, expedited programs, and patient engagement including extensive experience guiding patient advocacy groups. He also serves on the National Organization for Rare Disorders (NORD) Advocacy Committee.

#RareDC2020 Expectations “I look forward to connecting with rare disease advocates from across the country and learning about their diseases and advocacy priorities. But the best part is helping patients and caregivers who are first-time attendees feel comfortable sharing their story and asking for change in the meetings with Members and staff.”

Stephanie - Pennsylvania Like • Reply • Message

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Legislative Conference Guest Speakers Jennifer Bernstein Vice President, Horizon Government Affairs

Remy Brim, Ph.D. Vice President, BGR Government Relations

Jennifer joined Horizon Government Affairs in December 2008 as Vice President, focusing on legislative and regulatory developments within the pharmaceutical, biotechnology and medical device sectors.

Remy Brim is the Vice President of Regulatory Policy and Strategy, and the Health and Life Sciences Practice Co-Lead at the BGR Group.

In this role she focuses on policy development and advocacy support for clients with FDA regulated products navigating the complex regulatory and political landscapes necessary to advance their discovery, development, and delivery.

Prior to joining Horizon, Jennifer was Vice President of Healthcare Research at a mid-sized healthcare advisory and financial services firm. In this capacity, she managed over 100 clients, including hedge funds, mutual funds, investment advisers and healthcare corporations and was responsible for anticipating and translating legislative and regulatory catalysts within all sectors of the healthcare marketplace.

Jennifer also spent several years on Capitol Hill as a legislative assistant to former Congressman James Greenwood (R-PA).

Jennifer received her Bachelor of Arts in Political Science and History from Millersville University of Pennsylvania, and Master’s Degrees in American Government and International Relations from Temple University. A proud Chicago native, Jennifer and her family now reside in Virginia, where they are still searching for good pizza.

Melissa Bowie Senior Director, State Government Affairs, Takeda Melissa runs the State Public Affairs team at Takeda Pharmaceuticals America, with responsibility for the Mid-West/Mid-Atlantic region.

Her passion is engaging with state policymakers to ensure access for patients to medicines. Starting her health policy career at the Centers for Medicare & Medicaid Services provided firsthand experience to Medicaid drug policy and the Medicare prescription drug benefit. She also worked in the U.S. Senate on the Senate Finance and Senate Help committees.

While at Vanderbilt University, she earned a Bachelor of Arts in Political Science and French, as well as a law degree from The Catholic University of America. She is creative and loves crafty projects, especially with her many nieces and nephews.

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Prior to joining the BGR Group, Remy was the Senior FDA Policy Advisor to Senate HELP Committee Ranking Member Patty Murray (D-WA), where she led negotiations for Democrats on the 21st Century Cures Act and the FDA Reauthorization Act. Previously, she managed Sen. Elizabeth Warren’s (D-MA) comprehensive health care portfolio.

Remy earned a B.S. in Microbiology and Molecular Genetics from Michigan State University, a Ph.D. in Pharmacology from the University of Michigan, where she helped develop an investigational, Breakthroughdesignated drug, and served as a Bioethics PostDoctoral Fellow at the National Institutes of Health.

Share Your Rare Experience #RareDC2020 whitneyangellcarter • Follow Washington D.C.

Trip to DC was a success! Rare Disease Week followed by the National Lupus Advocacy Summit with LADA (Lupus Allied Diseases Association) and @lupusorg #RareDC2019 #LupusAdvocacy #risingvoices #lupuswarrior #RareDisease

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Legislative Conference Guest Speakers Mark Dant Chair, Board of Directors, EveryLife Foundation for Rare Diseases Mr. Dant comes from the Carrolton, Texas Police Department where he retired as the Assistant Chief of Police. Mark and his wife Jeanne are the parents of Ryan, 31-years-old, diagnosed with MPS I. Over 15 years ago, Mr. Dant and his wife, Jeanne were the parents that spearheaded the funding for the first MPS Enzyme Replacement Therapy, Aldurazyme, through their Foundation, the Ryan Foundation. In partnership with Dr. Emil Kakkis, Mr. Dant and his family were also key advocates speaking to the FDA about the importance and significant impact of ERT for the treatment of mucopolysaccharidoses.

In 2009, Mr. Dant and his family successfully championed Congress to pass the Ryan Dant Health Care Opportunity Act, H.R. 1441-111.

Khrystal Davis Founder, Texas Rare Alliance Khrystal joined the rare disease community in 2011 when her newborn son, Hunter, was diagnosed with Spinal Muscular Atrophy (SMA) Type 1, the leading genetic cause of mortality in children under the age of two. In May of 2016, Khrystal advocated alongside the FAST Movement (Families for the Acceleration of Spinal Muscular Atrophy Treatments) in a meeting with top FDA representatives for access to Spinraza, an SMA treatment in clinical trials at the time. Together with other FAST members, she asked the FDA to stop placebo trials, provide a means of access for the weakest SMA patients, accelerate the approval, and approve the treatment for all SMA patients regardless of age or type of SMA. In an interim look completed on August 1, 2016, the FDA found the treatment met trial objectives. An Expanded Access Program for SMA Type 1 patients commenced August 12, 2016, and the FDA approved Spinraza for children and adults with SMA on December 23, 2016.

Khrystal advocates for whole-genome sequencing of newborns, the expansion of newborn screening programs, insurance policies that conform to FDA labels for orphan drugs, and clinical trials and real-world evidence that include endpoints and data points that are meaningful to patients and caregivers.

Jennifer Dexter Director of Policy, National Health Council Jennifer has been working in the public policy field for more than 20 years. In her role as Director of Policy, Dexter helps the National Health Council and its member organizations develop policy positions that seek to improve the lives of people with chronic diseases and disabilities and advocates for these policies on Capitol Hill and within the executive branch.

Prior to joining the NHC in September 2019, Dexter spent two and a half years as a product director at Anthem, Inc. helping them develop and shape Medicaid solutions that supported people with disabilities, older adults and children in foster care.

Prior to that, Dexter led Easterseals’ efforts with Congress annually to create and assure funding for federal programs serving people with disabilities and older, particularly in long-term services and supports.

Dexter earned her BA in political science from Washington University in St. Louis.

#RareDC2020 Expectations “Watching to see who y’all find that’s really in our corner for better insurance approval processes, awareness of what it’s like to live with uncertain paths to treatment, and who really understands how difficult it is to navigate healthcare.”

Pam - Texas Like • Reply • Message

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Legislative Conference Guest Speakers Ryan Fischer Senior Vice President of Community Engagement, Parent Project Muscular Dystrophy Ryan serves as the Sr. Vice President of Community Engagement for Parent Project Muscular Dystrophy (PPMD) and has been with the organization for 16 years. PPMD is the leading organization in the fight to end Duchenne.

Within PPMD, Ryan oversees strategic community engagement, patient-preference research, and grassroots advocacy, guiding the patient community on advocating with members of Congress and federal agencies. Ryan has led legislative efforts toward passage of the MD-CARE Act Reauthorization of 2008, the MD-CARE Act Amendments 2014, and the Patient Focused Impact Assessment Act (PFIA, 2015).

Ryan leads PPMD’s national grassroots outreach network, PPMD CONNECT. These parent-led state specific groups serve to connect families with local support and work to turn the catastrophic nature of the diagnosis of Duchenne into advocacy and action to impact outcomes in Duchenne. Ryan has led PPMD’s pioneering work in patient preference research, with the aim of advancing the science of patient input, conducting six patient preference studies to date.

Eric Gascho Vice President, Policy and Government Affairs, National Health Council Eric is the Vice President, Policy and Government Affairs.

He has been with the National Health Council since August 2009. In his role, Gascho helps the National Health Council and its member organizations develop policy positions that seek to improve the lives of people with chronic diseases and disabilities and advocates for these policies on Capitol Hill and within the executive branch. Prior to joining the NHC, he worked in the government relations department of the Society of Teachers of Family Medicine. Gascho earned his BA degrees in government and business management from the University of Redlands in California.

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Juan Gomez Legislative Aide, Office of Senator Corey Booker Juan Gomez was born and raised in New Jersey and attended Montclair State University where he studied Psychology and Political Science. He has worked for Senator Cory Booker for the past five years and currently serves as the Senator’s Legislative Aide covering labor, health care, and immigration issues.

Mike Gula Co-founder, Prime Advocacy Mike is the co-founder of Prime Advocacy, the leading Washington, D.C. Advocacy Day firm. Since 2009, Gula Graham has raised over $250,000,000 for House and Senate members of Congress. Since 2010, Prime Advocacy has scheduled over 26,000 meetings for 30,000 attendees. When Congress was in session, Prime Advocacy secured a meeting with the actual member of Congress 39% of the time in 2018 and confirmed meetings with 96% of the Congressional offices where meetings were requested. A native of Austin, Texas, Mike received his Bachelor of Arts degree from the University of Richmond, where he also served as captain of the baseball team.

#RareDC2020 Expectations “In the short time since my diagnosis in October 2019, I have learned that I must be my own/best advocate.

I am looking forward to learning and sharing so I can continue to fight and hopefully help others as well.”

Linda - Maine Like • Reply • Message

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Legislative Conference Guest Speakers Luke Hatzis Principal, Capitol Counsel Lucas “Luke” Hatzis is a principal with Capitol Counsel, LLC. Hatzis brings to clients nearly a decade of congressional experience serving senior members of the House Republican Caucus, with an emphasis on health care policy and the legislative process. Both on and off the Hill, Hatzis has worked with senior Ways and Means and Energy and Commerce staff to advance legislation. He has also worked for several Illinois campaigns, including the Illinois Victory Program and the 2008 14th District Special Election.

Allison Herrity YARR Representative Allison is a 21-year old rare disease patient and health policy student at the George Washington University in Washington, DC. Her passion for rare disease advocacy is drawn from her personal experiences as a rare disease patient, as well as those of her younger sister. Allison currently serves as co-chair of the YARR policy committee, President of RARE GW, and Community Engagement Liaison for the Virginia Rare Action Network (RAN). She has previously served as a policy fellow at the National Organization for Rare Disorders (NORD).

Angela Ramirez Holmes Founder and President, CAL RARE Angela started her advocacy in rare diseases when her then 8 year old son was diagnosed with a rare brain condition, an arteriovenous malformation (AVM). Angela is the Founder and President of California Action Link for Rare Diseases (Cal Rare), a non-profit membership coalition of rare disease stakeholders that began in 2017 and is dedicated to improving the lives of rare disease patients in California. Cal Rare started the California Rare Disease Legislative Caucus that now includes

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25% of the State Legislature. In 2018, Angela was the recipient of the Rare Voice Award for State Advocacy from the Rare Disease Legislative Advocates. In 2019, she was named a Top 100 Social Media Advocate for Rare Disease and in 2020, a Top 50 Rare Disease Awareness Advocate by the International Pain Foundation. Angela has over twenty years of political and legislative experience on the national, state, and local levels.

Catherine Jackson, RN, BSN, MPH, Director of Advocacy, Mallinckrodt Pharmaceuticals Cathy earned her nursing degree and Master’s degree in Public Health from the University of South Florida in Tampa. Cathy’s professional experience includes a variety of positions in nursing including Nurse Manager at Johns Hopkins Blaustein Pain Treatment Center. She has been in the pharmaceutical industry for the last 19 years working in Medical Affairs and Advocacy. Cathy resides in St. Petersburg, FL.

Cheryl Jaeger Principal, Williams & Jensen Cheryl brings extensive experience to Williams and Jensen having served as a senior policy advisor for the House Republican leadership for nine years, most recently with the former Majority Leader Eric Cantor (R-VA).

In this role, she led the advancement of health care reform legislation to improve patient choice, lower health care costs, and expand innovation. Jaeger previously served in the Office of the Majority Whip as a senior advisor to now Senator Roy Blunt (RMO). Prior to serving as a leadership aide, Jaeger worked as a professional staff member for the Committee on Energy and Commerce. She was the lead staff negotiator of the NIH Reform Act of 2006, the Gabriella Miller Kids First Research Act, and several other pieces of legislation. Jaeger began her Capitol Hill career as a legislative aide to former Congressman Christopher Cox (R-CA).

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Legislative Conference Guest Speakers Julia Jenkins Executive Director, EveryLife Foundation for Rare Diseases Julia has worked for the EveryLife Foundation for 10 years. She was appointed Executive Director and Board Secretary in 2013 after serving as the Director of Public & Government Relations since the Foundation was founded in 2009. Prior to working at EveryLife, Julia worked as a Political Communications Consultant with the Lew Edwards Group, creating strategic plans to organize communities to support local funding measures. Julia was Legislative Director and registered California State lobbyist for Public Employees Union Local #1. She coordinated all political action at local, state, and federal levels, including efforts to ensure every Californian would have access to affordable quality healthcare. She led efforts for the San Francisco Democratic Party, helping to defeat the anti-labor propositions in the 2005 Special Election and worked for House Majority Leader Nancy Pelosi. Prior to working in politics, Julia was a fundraiser for the Muscular Dystrophy Association.

Julia did her Masters work in Political Science at San Francisco State University and her undergraduate work in Communications at St. Mary’s College of California.

Swapna Kakani Founder and Director, Alabama Rare Swapna diagnosed with Short Bowel Syndrome, a chronic GI rare disease, at birth, has lived with supplemental intravenous nutrition and a feeding tube for 30 years. In 2014, she had a small intestine organ transplant. Swapna through her platform, Swapna Speaks, has given several presentations across the world to various healthcare events, including, a TEDx talk. Swapna, does healthcare advocacy work at the federal and state level for the Short Bowel Syndrome and broader rare disease community. In 2017, she founded and now directs Alabama Rare, a grassroots coalition to unite Alabama around the rare disease population.

In 2017, Swapna on behalf of Alabama Rare, was

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instrumental in creating the Alabama Rare Disease Advisory Council. In March 2019, Swapna was awarded the consumer advocacy award by the American Society for Parenteral and Enteral Nutrition, and in July 2019, was nominated by Wego Health as a patient leader hero.

Annie Kennedy Chief of Policy and Advocacy, EveryLife Foundation for Rare Diseases Focused on improving health outcomes for people living with rare diseases by advancing the development of treatment and diagnostic opportunities for rare disease patients through science-driven public policy, Annie’s work includes building strong partnerships with policy makers, federal agencies, Industry, and alliances. Current areas of emphasis include leading the national Burden of Rare Disease Study, 21st Century Cures Act and PDUFA VII engagement, national newborn screening program engagement and state RUSP alignment legislation, and innovating around therapy valuation and access issues.

Annie has served within the community for nearly three decades through her roles with Parent Project Muscular Dystrophy (PPMD) and the Muscular Dystrophy Association (MDA). She helped lead legislative efforts around passage and implementation of the MD-CARE Act (2001, 2008, 2014), the Patient Focused Impact Assessment Act (PFIA) which became the Patient Experience Data provision within the 21st Century Cures Act (sec 3001), engagement with the FDA and Industry around regulatory policy and therapeutic pipelines.

#RareDC2020 Expectations “Giving my daughter and son a voice to fight for MPS1 Hurler syndrome.

Gain experience networking and using my voice to make a change!”

Allie - Tennessee Like • Reply • Message

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Legislative Conference Guest Speakers Stefanos Koutsoukos YARR Representative Stefanos Koutsoukos has dedicated his young career to the advancement of translational research for rare diseases, including his own.

He has a rare congenital skin condition called ichthyosis which is characterized by extremely dry and scaly skin. His living with this disease, in addition to his great interest in the life sciences, has inspired his pursuit of translational research for his own and other rare diseases.

Stefanos plans to identify and study the underlying genetic causality of his disease as well as develop novel therapeutics for its treatment and ultimately its cure. He plans to pursue graduate education in genetics after the completion of his fellowship at the National Human Genome Research Institute.

Mary-Sumpter Lapinski VP, Global Government Affairs, Greenwich Biosciences Mary-Sumpter previously served as Counselor to the Secretary for Public Health and Science at the Department of Health and Human Services from March 2017-October 2018, she worked closely with the CDC, FDA, NIH, and SAMHSA. Prior to HHS, Mary-Sumpter spent ten years working for Senator Lamar Alexander (R-TN) first as Health Policy Advisor and then four years as his Senate HELP Committee Health Policy Director, overseeing the Committee’s health policy work, including drafting, negotiation, and passage of landmark legislation, including the 21st Century Cures Act.

Nicholas Manetto Principal, Faegre Drinker Consulting Nick designs, directs and implements successful health care policy advocacy campaigns. He represents a wide variety of clients, with an emphasis on patient advocacy organizations and children’s hospitals. He has particular experience advocating on issues related to medical research and public health, children’s health, rare disease, patient-focused drug development, and coverage and payment policy.

In more than 15 years in health policy, government affairs and strategic communications, Nick has led efforts that have resulted in legislative, executive branch and regulatory wins for clients. His work has helped enact multiple pieces of legislation into law, and regularly he maintains strong relationships with key congressional health-focused committees.

Nick has successfully represented client interests on nearly every significant piece of health care legislation over the past decade, including the 21st Century Cures Act, prescription drug user fee bills, numerous Medicare packages, the Affordable Care Act (ACA), and a number of annual appropriations bills, particularly the Labor, HHS and Education spending bill.

In addition to her public sector experience, MarySumpter lobbied for Bristol-Myers Squibb, both in the United States and abroad, as well as for Jeffrey J. Kimbell and Associates in D.C. A native Tennessean and graduate of Vanderbilt University, Mary-Sumpter began her career working in the Tennessee Congressional delegation working for Rep. Van Hilleary and then Senator Bill Frist.

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Legislative Conference Guest Speakers Dan Pezzetta YARR Representative Dan is a 20 year old with Aortic Stenosis. When he was born he only had two of the four aortic heart valves that pump blood to the rest of the body. He had surgeries to put in replacement valves, but it is still like trying to drive a car with two flat tires; he gets very tired very easily, due to how fast his heart has to pump. He was inspired by Claire Wineland to become an advocate and to fight for people with rare diseases in the hopes to make changes to bills and to make the lives of everyone with a rare disease easier. Dan is currently in college studying public policy, and intends to attend graduate school to pursue a doctorate in public policy.

Chris Smith President & Chief Executive Officer, SmithSolve Chris has nearly 30 years of experience in healthcare communications, including leadership positions with global pharmaceutical, biotechnology, and public relations firms. As an expert in rare disease communication, he knows how to address the scientific, medical, clinical, social, and economic aspects of orphan drug development. His work spans more than a dozen biopharma companies and includes patient advocacy support, patient panels and meet-ups, media relations, investor events, patient days, clinical trial recruitment, corporate branding and website development, pipeline communications, data announcements, and more. Chris has worked across diverse therapeutic categories and has represented many leading healthcare companies, including Alexion, AstraZeneca, Johnson & Johnson, Pfizer, and Sanofi.

He is a founding member of the Rare Collective®, a group of trusted independent advisors in orphan drug development.

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Katherine Sears Communications Director, Office of Representative David Joyce Born and raised in California, Katherine came to Washington, D.C. at 17 years-old to attend American University. During college, Katherine held four internships (including with a congressional office and a press internship at TSA). After graduating with a bachelor’s degree in Communications, Law, Economics, and Government, Katherine enrolled in AU’s Political Communication master’s program. Shortly thereafter, she was hired to run the University’s Mentorship Programs abroad.

Following the 2016 election, Katherine began working on the Hill. In December of 2018, Katherine was hired as Rep. Dave Joyce’s Communications Director. In this role she is the Congressman’s official spokesperson and develops, implements and manages all of the Congressman’s communications, including social media, official statements, press releases, interviews, speeches, e-newsletters, op-eds, etc.

Steve Silvestri Director of Public Policy, EveryLife Foundation for Rare Diseases Steve drives the EveryLife Foundation’s federal and state policy priorities to accelerate access to rare disease therapies and diagnostics. Additionally, he manages the Foundation’s Community Congress and Scientific Workshop programs which brings together stakeholders from patient groups, government, and industry. Before joining EveryLife, Steve was Senior Legislative Assistant to Congressman Rodney Frelinghuysen (NJ), former Chairman of the House Appropriations Committee. He was responsible for overseeing the Chairman’s health policy agenda, including the enactment of robust funding increases for the National Institutes of Health and new funding authorized by the 21st Century Cures Act to accelerate drug development at the FDA.

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Legislative Conference Guest Speakers Dylan Simon Newborn Screening Policy Fellow, EveryLife Foundation for Rare Diseases Dylan Simon joined the EveryLife Foundation as the Newborn Screening Policy Fellow in September 2019. Prior to that he interned for the Senate HELP Committee and worked at advocacy organizations. He began at Research America, where he advocated for increased support of policies that would allow for faster biomedical progress. After his time there, he moved to VeteransAgainstAlzheimer’s, where he worked to help launch the network, which focuses on improved treatment and caregiving for veterans who suffer from Alzheimer’s disease.

Dylan received his Master’s degree in Molecular Microbiology and Immunology from the University of Maryland, Baltimore in May 2016. He earned his Bachelor’s degree in Biochemistry from Case Western Reserve University in May 2014.

Vicki Seyfert-Margolis, PhD Founder & CEO MyOwnMed Vicki has published and presented in top journals and numerous conferences about the transformative nature of digital technologies and novel clinical trial designs. Prior to founding My Own Med, Inc., Vicki was appointed in the Obama Administration as the Senior Advisor for Science Innovation and Policy in the Office of the Commissioner of the US Food and Drug Administration. While at the FDA, she worked on a myriad of regulatory and leading-edge science issues, including mobile technologies, innovation in drug development, companion diagnostics, and a whole range of regulatory science initiatives and policies working with industry and academic leaders. Prior to the FDA, she served as Chief Scientific Officer at the Immune Tolerance Network (ITN), a non-profit consortium of researchers seeking new treatments for diseases of the immune system.

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Sean R. Tunis, MD, MSc. Principal, Rubix Health Sean consults with public and private sector organizations on issues at the interface of regulatory and reimbursement policy, market access, comparative effectiveness, outcomes measurement and health technology assessment. He is also a senior advisor at the FDA, in the Office of Clinical Policy and Programs. Dr. Tunis was the Founder and CEO with the Center for Medical Technology Policy in Baltimore, Maryland from 2016 to 2019. CMTP is an independent, nonprofit organization that provides a neutral platform for multi-stakeholder collaborations that are focused on improving the quality, relevance, and efficiency of clinical research. Dr. Tunis is the immediate past President of Health Technology Assessment International.

Through September of 2005, Dr. Tunis was the Director of the Office of Clinical Standards and Quality and Chief Medical Officer at the Centers for Medicare and Medicaid Services (CMS). In this role, he had lead responsibility for clinical policy and quality for the Medicare and Medicaid programs.

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Legislative Conference Guest Speakers Caitlin Van Sant Senior Policy Advisor, Office of Representative G.K. Butterfield Caitlin advances Representative Butterfield’s health care, education, and labor priorities, with a focus on childhood cancer and rare diseases. Prior to joining the office of Representative Butterfield in June 2019, Caitlin was a manager of federal affairs at the Children’s Hospital Association, where she advocated on behalf of 220 children’s hospitals across the country on Medicaid and CHIP policy. Caitlin also worked for the American Academy of Pediatrics as a federal lobbyist and the Biotechnology Innovation Organization, working on state policy.

Caitlin graduated cum laude from George Mason University School of Law and was a notes editor of the George Mason Law Review. She holds a B.A. in International Affairs and a B.B.A. in International Business from James Madison University. Caitlin is licensed to practice law in the Commonwealth of Virginia.

Nikia Vaughan Cimone and Friends A dedicated community leader in Baltimore, Maryland, Nikia has a passion for healthy living, community and ending stigmas in the AfricanAmerican community. When her daughter Cimone was diagnosed with sickle cell disease SS in 2012, Nikia created a sickle cell awareness group, Cimone and Friends, to advocate on behalf of, and provide education for, families with children who suffer from sickle cell disease.

In 2018, Nikia testified as a parent for SB 161, requiring the State Department of Education and the Maryland Department of Health to establish certain guidelines by December 1, 2018, for public schools regarding the administration of health care services to students with sickle cell disease and requiring the State Department of Education and the Maryland Department of Health by December 1, 2018, to

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provide technical assistance on implementation of the guidelines and to develop a process to monitor implementation of the guidelines. The bill was passed and signed into law by Governor Larry Hogan in May 2018. Nikia resides in Baltimore City with her husband Charmon and their two children, Langston (10) and Cimone (7).

Mark Vieth Senior Vice President, CRD Associates Since 2014, Mark has served as the coordinator of the Defense Health Research Consortium. The consortium is managed at Cavarocchi Ruscio Dennis Associates, LLC (CRD Associates), where Mark currently serves as a Senior Vice President. Mark also represents the interests of other patient advocacy organizations, professional scientific societies and coalitions.

Prior to joining CRD Associates in 2002, Mark served for 14 years as a professional staff member in the House of Representatives. As Chief of Staff for Congressman Robert A. Borski (D-PA), Mark worked extensively on a wide range of economic and urban development issues, including surface transportation, aviation, port and waterfront revitalization, defense conversion, and brownfields cleanup. He also managed a successful, multi-year appropriations strategy that channeled in millions of dollars for highpriority infrastructure projects into Philadelphia, PA. Mark is also past President and current active board member of the Pennsylvania Society of Washington, D.C. He earned his B.A. in international relations from Syracuse University.

#RareDC2020Expectations “Experience doing rare disease advocacy work and networking with others who are passionate about improving the lives of those living with rare diseases.”

Susan - Washington State Like • Reply • Message

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Legislative Conference Guest Speakers Shannon von Felden Director of Rare Disease Legislative Advocates, EveryLife Foundation for Rare Diseases Shannon manages the Rare Disease Legislative Advocates program at the EveryLife Foundation for Rare Diseases where she works with advocates from across the country to engage at the local, state, and federal level. She began her career on Capitol Hill as a Legislative Assistant for Congresswoman Shelley Berkley (NV) working on health care and veterans affairs issues. Shannon has worked with national nonprofit organizations to further their policy and advocacy goals including the Juvenile Diabetes Research Foundation and National Osteoporosis Foundation.

She received her Master of Public Policy from American University.

Monica Weldon Founder and President, Bridge the Gap—Syngap In November of 2012, Monica’s twin son, Beckett, was the first to be diagnosed at Texas Children’s Genetics Clinic with the gene mutation SYNGAP1 (6p21.3). When Beckett was four months old, she noticed he was not meeting the same milestones as his twin sister. Monica then began a journey to find answers to help her son. She started to blog about his progress and this led to building a community of parents and caregivers that are now a strong support group. She is the Founder and President/CEO of Bridge the Gap – SYNGAP Education and Research Foundation. Her focus is on building the programs and mission of Bridge the Gap – SYNGAP Education and Research Foundation.

She is the Primary Investigator on the SYNGAP1 (MRD5) Registry and Natural History Study. She is a life member of the Worldwide Association of Female Professionals and a member of the first class of 2017 Illumina Ambassadors established in the United States.

Joel White Founder and President, Horizon Government Affairs Joel is the Founder and President of Horizon Government Affairs. He is the President of the Council for Affordable Health Coverage and its campaigns on transparency and medication adherence (Clear Choices and Prescriptions for a Healthy America) as well as a service organization called Arlington Bridge Builders. In addition, Joel is the Executive Director of the Health Innovation Alliance, an HGA effort to expand adoption and use of health IT to improve outcomes and lower costs. He is on the Board of Directors of the Newborn Foundation and Samaritan Inns.

Joel spent twelve years on Capitol Hill as professional staff, where he helped enact nine laws, including the 2002 Trade Act, which created health care tax credits, the 2003 law that established the Medicare prescription drug benefit and Health Savings Accounts, the 2005 Deficit Reduction Act, and the 2006 Tax Reform and Health Care Act, which reformed Medicare payment policies.

Share Your Rare Experience Follow us and share your #RareDC2020 pictures for a chance to win an $800 travel stipend for Rare Disease Week 2021.

Tip: Be Creative! @RareAdvocates #RareDC2020

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Legislative Conference Guest Speakers Shayne Woods Senior Policy Advisor, Office of Representative Gus Bilirakis

a grants reviewer for the past seven years and was a member of the National Patient-Centered Clinical Research Network (PCORnet) Industry Working Group for over four years.

Shayne currently serves as a Legislative Assistant for the Hon. Gus M. Bilirakis of Florida. In this capacity, he plans and develops legislative initiatives while tracking legislation through the legislative process related to Public Health, Energy, Foreign Affairs, Trade, Agriculture, Education & the Workforce, Natural Resources, and Oversight & Government Reform. Additionally, Mr. Woods prepares public statements, drafts correspondence, and meets with constituents and advocacy groups in regard to the aforementioned issues. Mr. Woods also founded and ran a healthcare consulting practice that advised key industry stakeholders on the Affordable Care Act while providing business development assistance.

Mr. Woods holds a B.A. from Washington University in St. Louis.

Before joining Amgen, Jim held various global roles in health economics, market access/reimbursement, contracting/pricing, and commercial operations for several biotech manufacturers including Genentech and Abbott Vascular. Prior to industry, Jim worked at Brown & Toland Medical Group (an original Medicare Pioneer ACO based in San Francisco, CA), where he developed his background in managed care contracting, claims/benefits/eligibility administration, and electronic medical/health records, data warehousing, and provider reporting/auditing.

James J. Wu, M.Sc., M.P.H. Principal Health Economist, Global Health Economics, Amgen Inc.

Jim holds a Master of Science in Immunology from Brown University (Providence, RI), a Master of Public Health in Medical Informatics / Public Policy from New York Medical College (Valhalla, NY), and a Bachelor of Science in Cellular Biology, Molecular Biology, and Biochemistry from the University of Michigan (Ann Arbor, MI).

Share Your Rare Experience #RareDC2020 chronicallyawesome1 • Follow Washington D.C.

Jim Wu is Principal Health Economist in the Health Technology Assessment Center of Excellence (HTA CoE), Global Health Economics (GHE) at Amgen, Inc., where for the past six years he has supported multiple products in the oncology, cardiovascular, and inflammation therapeutic areas (several with orphan indications), providing strategy and direction for HTA submissions to Amgen’s global affiliates. Since 2017, Jim has also been a Scholar-in-Residence with the Friends of Cancer Research, a patient advocacy organization based in Washington, DC, where he provides health economic expertise for US regulatory and access/reimbursement policy.

From 2013 thru 2018, Jim was a member of the Patient Centered Outcomes Research Institute’s Rare Disease Advisory Panel (PCORI RDAP), having served an inaugural three year appointment, and followed by re-appointment for another two years. He has also served the PCORI Improving Methods Program as

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Thanks to our California Representatives, Senators and their staff for meeting with us! Rare Disease Week is every week for me and the 30 million other people in the US living with a rare disease. #RareDC2019 #RareAdvocates #autoimmuneautonomicganglionopathy

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#HearUsYARR #HearUsYARR #HearUsYARR #HearU YARR #HearUsYARR #HearUsYARR #HearUsYARR #HearUsYARR #HearUsYARR #HearUsYARR #HearU YARR #HearUsYARR #HearUsYARR #HearUsYARR #HearUsYARR#HearUsYARR #HearUsYARR #HearU YARR #HearUsYARR #HearUsYARR #HearUsYARR #HearUsYARR #HearUsYARR #HearUsYARR #HearU YARR #HearUsYARR #HearUsYARR #HearUsYARR #HearUsYARR #HearUsYARR#HearUsYARR #HearU YARR #HearUsYARR #HearUsYARR #HearUsYARR #HearUsYARR #HearUsYARR #HearUsYARR #HearU YARR #HearUsYARR #HearUsYARR #HearUsYARR

To join us for the Young Adult Meetup at Rare Hub Wednesday, February 26 • 6:00 p.m. - 8:00 p.m.

Thank you to our sponsor

1012 14th Street NW, Suite 500, Washington D.C. 20005 HEARUSYARR.org

@HEARUSYARR

CELEBRATING RARE DISEASE RESEARCH ADVOCATES: STRONG TOGETHER The W Hotel, Washington D.C. 515 15th St NW, Washington, D.C. 20004 February 26, 2020 5:30 p.m. to 7:30 p.m.

Join Research!America and partners for a reception honoring advocates for the critical role they play in faster progress against rare diseases. The program will feature a brief, interactive conversation about rare disease research, caregiving, and advocacy.

Throughout the reception and program, join the conversation online by sharing your reasons for research at #RareDC2020 #RAREis #RareDiseaseWeek.

Register Now: bit.ly/2urSVkZ Supported in part by

In partnership with

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Thursday Hill Day Breakfast THURSDAY, FEBRUARY 27

7:00 AM – 8:30 AM Ronald Reagan Building and International Trade Center - Atrium Ballroom 1300 Pennsylvania Ave NW, Washington, DC 20004 Metro Stop: Federal Triangle Join fellow rare disease advocates to get fueled up for your meetings on Capitol Hill.

Attendees will receive any updates or last-minute changes to their Hill Day schedules. EveryLife staff and Prime Advocacy representatives will be available for questions.

• • •

FEATURING ADDRESS BY Joel Wood Senior Vice President, Government Affairs, The Council of Insurance Agents & Brokers

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Joel is the chief lobbyist at The Council, which represents 200 firms that collectively sell more than 90 percent of all commercial property/casualty premiums, and more than 70 percent of employee benefit premiums nationally. Over the course of his career, Wood has been deeply engaged in many issues impacting the commercial insurance brokerage industry – from health insurance reform efforts, to terrorism reinsurance, to regulatory reforms both small and large, at both the federal and state level – and has received numerous accolades as one of the top lobbyists in the financial services industry. In 2000, he and his wife Dana formed the Foundation to Eradicate Duchenne following the diagnosis of their son James with Duchenne Muscular Dystrophy, the world’s most lethal childhood genetic disease. Their advocacy and charitable work has led to hundreds of millions of dollars’ worth of scientific research into DMD, and a corresponding decade-long increase in life expectancy for those afflicted with the disorder.

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Hospitality Room Join us between meetings from 10: 30 AM - 4:00 PM in our Hospitality Room.

Rusell Senate Office Building, Room 485 Presented by:

#RareDC2020

Hill Day Issue Information Legislative Ask #1 Support Creation of The Rare Disease Center of Excellence at the U.S. Food and Drug Administration (FDA)

TALKING POINTS ✓ A Center of Excellence (COE) within the FDA would elevate rare disease expertise within the agency and help ✓ ✓ ✓ ✓

to remove communication barriers across centers and divisions to accelerate and improve the rare disease therapy development process at FDA. FDA already has authority under the 21st Century Cures Act to establish Centers of Excellence.

Three years ago, the FDA established the first FDA COE, focused on oncology; the Oncology Center of Excellence which has been extremely successful in bringing new cancer therapies to patients.

Two years ago, the EveryLife Foundation hosted a Scientific Workshop on this topic with case studies from patient organizations and industry that demonstrated the value of having such a COE. Given the challenges and, therefore, the unique expertise needed to advance the development and review of products for rare diseases, innovators have long believed that a Rare Disease Center of Excellence would provide the necessary resources and support to allow offices across FDA to more consistently and efficiently review novel products for these rare conditions.

RARE DISEASE CENTER OF EXCELLENCE VISION ✓ The COE would serve as a consultative and cross-cutting body to build knowledge and capacity and consult ✓

✓ ✓

with review divisions in reviewing applications for rare-disease therapies.

It would identify and address current and emerging challenges and opportunities in rare disease therapy development, including needs pertaining to development of therapies for individual or very small populations.

The COE would not supplant any authorities held by FDA review divisions.

The Rare Disease Center of Excellence would also include a dedicated program focused on the many unique needs associated with developing treatments for individual or very small populations (e.g., N of 1).

FOOD AND DRUG ADMINISTRATION (FDA) ✓ The FDA plays a critical role in bringing cures into the hands of patients through the review and approval of innovative medicines and devices.

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#RareDC2020

Hill Day Issue Information Legislative Ask #2 Enact the Newborn Screening Save Lives Reauthorization Act, H.R. 2507 / S. 2158

TALKING POINTS ✓ Federal newborn screening programs expired as of September 30, 2019 as a result of the failure to pass the Newborn Screening Saves Lives Reauthorization Act.

✓ The House has done its job, passing the Reauthorization Act by voice vote in July 2019. Currently, the bill is held up in the Senate.

✓ This bill would reauthorize critical existing programs that provide assistance to states to improve and ✓ ✓

expand their newborn screening programs, support parent and provider education, and ensure laboratory quality and surveillance for newborn screening.

Diagnosis through newborn screening enables early intervention that can reduce the progression of irreversible harm and significantly reduce long term treatment costs.

Newborn screening SAVES LIVES.

KEY BILL PROVISIONS ✓ Reauthorizes the Health Resources and Services Administration (HRSA) grants to states to expand and ✓

improve their screening programs, educate parents and health care providers, and improve follow-up care for infants with a detected condition.

Reauthorizes the Secretary’s Advisory Committee on Heritable Disorders in Newborns and Children, which provides states with a Recommended Uniform Screening Panel (RUSP) to help ensure every infant is screened for conditions which have a known treatment.

NEWBORN SCREENING FACTS ✓ Of the four million babies born in the U.S. each year, one in 300 are found to have a potentially devastating condition through newborn screening.

✓ 12,000 newborns benefit from the early detection and delivery of life-saving treatments. ✓ Newborn screening is the practice of testing every newborn for certain genetic, metabolic, hormonal, and functional conditions that are not otherwise apparent at birth.

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#RareDC2020

Hill Day Issue Information Legislative Ask #3 Support Increased Funding for the U.S. Food and Drug Administration (FDA) Orphan Products Clinical Trial Grants Program and the Natural History Grants Program

TALKING POINTS ✓ Due to increases in the cost of clinical trials, the FDA appropriated grant funds are covering less and less of ✓ ✓

the total cost for conducting these trials.

FDA is currently only able to grant a small number of the total applications they receive.

We request an increase in the annual discretionary funding for the Food and Drug Administration (FDA) Orphan Products Clinical Trials Grants Program and Natural History Grants Program in the Fiscal Year 2021 Agriculture Appropriations Act.

ABOUT APPROPRIATIONS REQUESTS ✓ Every Fiscal Year (October- September) Congress allocates funding to support federal agencies and programs. The annual appropriations process is an opportunity to advocate for funding that supports rare disease patients.

FOOD AND DRUG ADMINISTRATION (FDA) ✓ The FDA plays a critical role in bringing cures into the hands of patients through the review and approval of innovative medicines and devices.

FDA ORPHAN PRODUCTS CLINICAL TRIAL GRANTS PROGRAM ✓ This FDA grant program supports new and continuing extramural research projects that test the safety and efficacy of promising new drugs, biologics, devices, and medical foods through human clinical trials in extremely vulnerable populations often with life-threatening conditions.

FDA NATURAL HISTORY GRANTS PROGRAM ✓ This FDA grant program supports studies that advance rare disease therapy development through:

• •

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characterization of the natural history of rare diseases and development and/ or validation of clinical outcome measures.

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Hill Day Issue Information Legislative Ask #4 Join the Rare Disease Congressional Caucus

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Co-Chairs: Representatives G.K. Butterfield (NC) and Gus Bilirakis (FL) and Senators Roger Wicker (MS) and Amy Klobuchar (MN)

TALKING POINTS ✓ The Rare Disease Caucus is a bipartisan, bicameral caucus that works to raise awareness of rare diseases. ✓ Rare diseases affect more than 30 million Americans and their families. ✓ One in ten Americans has a rare disease. ✓ There are more than 7,000 known rare diseases but unfortunately, the vast majority (over 90%) do not yet have a treatment approved by the FDA.

✓ Rare or orphan diseases are defined as diseases affecting fewer than 200,000 people in the U.S. ✓ More than 80% of rare diseases are considered ultra-rare, affecting fewer than 6,000 people. Some ✓

32

diseases affect fewer than 100.

Rare diseases include rare cancers, tropical or neglected diseases, genetic diseases and many pediatric diseases including cancers. Many of these diseases are life-threatening and have no treatment options.

#RareDC2020

HOUSE

• • • • • • • • • • • • • • • • • • • • • • • • • • • • • • • • • • • • • • •

Mark Amodei NV-2 Cindy Axne IA-3 Andy Barr KY-6 Joyce Beatty OH-3 Ami Bera CA-7 Eddie Bernice Johnson TX-30 Donald Beyer Jr. VA-8 Gus Bilirakis* FL-12 Sanford Bishop, Jr. GA-2 Lisa Blunt Rochester DE Suzanne Bonamici OR-1 Mo Brooks AL-5 Julia Brownley CA-26 Vern Buchanan FL-16 Michael Burgess TX-26 Cheri Bustos IL-17 G.K. Butterfield* NC-1 Salud Carbajal CA-24 Andre Carson IN-7 John Carter TX-31 Sean Casten IL-6 Steve Chabot OH-1 Judy Chu CA-27 David Cicilline RI-1 Lacy Clay MO-1 Steve Cohen TN-9 James Comer KY-1 Gerald Connolly VA-11 Jim Cooper TN-5 Jason Crow CO-6 Susan Davis CA-53 Rodney Davis IL-13 Peter DeFazio OR-4 Diana DeGette CO-1 Suzan DelBene WA-1 Ted Deutch FL-21 Debbie Dingell MI-12 Mike Doyle PA-14 Eliot Engel NY-16

• • • • • • • • • • • • • • • • • • • • • • • • • • • • • • • • • • • • • • • • • •

Anna Eshoo CA-18 Abby Finkenauer IA-1 Brian Fitzpatrick PA-8 Jeff Fortenberry NE-1 Ruben Gallego AZ-7 John Garamendi CA-3 Josh Gottheimer NJ-5 Garret Graves LA-6 Danny Heck WA-10 Jaime Herrera-Beutler WA-3 Kevin Herns OK-1 Jim Himes CT-4 Kendra Horn OK-5 Jared Huffman CA-2 Hank Johnson GA-4 David P. Joyce OH-14 Marcy Kaptur OH-9 Joseph Kennedy, III MA-4 Ro Khanna CA-17 Derek Kilmer WA-6 Andy Kim NJ-3 Ron Kind WI-3 Peter King NY-2 Raja Krishnamoorthi IL-8 Darin LaHood IL-18 Jim Langevin RI-2 Susie Lee NV-3 Mike Levin CA-49 Dan Lipinski IL-3 Dave Loebsack IA-2 Zoe Lofgren CA-19 Alan Lowenthal CA-47 Blaine Luetkemeyer MO-3 Tom Malinowski NW-7 Carolyn Maloney NY-12 Sean Patrick Maloney NY-18 Kenny Marchant TX-24 Brian Mast FL-18 Doris Matsui CA-6 Ben McAdams UT-4 Michael McCaul TX-10 Jim McGovern MA-2

• • • • • • • • • • • • • • • • • • • • • • • • • • • • • • • • • • • • • • • • •

David McKinley WV-1 Cathy McMorris Rodgers WA-5 Grace Meng NY-6 Markwayne Mullin OK-2 Seth Moulton MA-6 Richard Neal MA-1 Donald Norcross NJ-1 Eleanor Holmes Norton DC Steven Palazzo MS-4 Frank Pallone NJ-6 Jimmy Panetta CA-20 Chris Pappas NH-1 Bill Pascrell NJ-9 Donald Payne, Jr. NJ-10 Scott Peters CA-52 Collin Peterson MN-7 Chellie Pingree ME-1 Bill Posey FL-8 David Price NC-04 Mike Quigley IL-5 Jamie Raskin MD-8 Kathleen Rice NY-4 Max Rose NY-11 David Rouzer NC-7 Dutch Ruppersberger MD-2 John Rutherford FL-4 Jan Schakowsky IL-9 Brad Schneider IL-10 David Scott GA-13 Mikie Sherrill NJ-11 Mike Simpson ID-2 Albio Sires NJ-8 Elissa Slotkin MI-8 Adam Smith WA-9 Chris Smith NJ-4 Jason Smith MO-8 Lloyd Smucker PA-16 Darren Soto FL-9 Jackie Speier CA-14 Chris Stewart UT-2 Steve Stivers OH-15

• • • • • • • • • • • • • • • • •

Eric Swalwell CA-15 Glenn Thompson PA-5 Rashida Tlaib MI-13 Paul Tonko NY-20 Lori Trahan MA-3 Fred Upton MI-6 Juan Vargas CA-51 Nydia Velazquez NY-7 Peter Visclosky IN-1 Ann Wagner MO-2 Greg Walden OR-2 Debbie Wasserman-Schultz FL-23 Bonnie Watson Coleman NJ-12 Joe Wilson SC-2 Robert Wittman VA-1 John Yarmuth KY-3 Lee Zeldin NY-1

SENATE

• • • • • • • • • • • • • • • • • • • • • •

John Barrasso WY John Boozman AR Maria Cantwell WA Shelley Moore Capito WY Christopher Coons DE Tom Cotton AR John Hoeven ND Cindy Hyde-Smith MS James Inhofe OK John Kennedy LA Angus King ME Amy Klobuchar* MN Edward Markey MA Jeff Merkley OR Gary Peters MI James Risch ID Kyrsten Sinema AZ Jeanne Shaheen NH Tina Smith MN Debbie Stabenow MI Chris Van Hollen MD Roger Wicker* MS

161 Members 139 22

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Representatives

108 Democrats

Senators

1 Independent

52 Republicans

#RareDC2020

Hill Day Issue Information

Legislative Ask #5 Support the Better Empowerment Now to Enhance Framework and Improve Treatments (BENEFIT) Act

TALKING POINTS ✓ While much progress has been made to incorporate patient experience data into the regulatory review ✓ ✓

✓

process at FDA through previous legislative efforts, some significant gaps remain.

One such gap is a requirement for FDA to include any patient experience or patient-focused drug development (PFDD) data within its benefit-risk framework.

This means that the agency’s signature tool for evaluating a product’s benefit-risk does not have to include data from the patient perspective that could be critical to informing the agency’s evaluation and, ultimately, decision on whether or not to approve a product.

This legislation will amend the Food, Drug and Cosmetic Act (FDCA) to ensure that patient experience, PFDD, and related data – including information developed by a product sponsor or a third party such as a patient advocacy organization or academic institution – be considered as part of the benefit-risk assessment.

PATIENT EXPERIENCE DATA & THE FDA REGULATORY REVIEW PROCESS ✓ Many significant statutory and regulatory advances have been made in the past decade to ensure that ✓ ✓

34

patient experience is meaningfully incorporated into product development and regulatory review processes.

BENEFIT represents the evolution, following on critical provisions coming out of PDUFA V, VI, and the 21st Century Cures Act that have been implemented by the FDA and embraced by stakeholders.

The BENEFIT Act (S. 1052) was previously introduced in the Senate by Sen. Roger Wicker (R-MS) and Sen. Amy Klobuchar (D-MN) – and passed in the Senate in August of 2017.

#RareDC2020

Hill Day Issue Information

Legislative Ask #6 Co-Sponsor the Medical Nutrition Equity Act, H.R. 2501

• •

Senate Lead Sponsor: Senator Bob Casey (D-PA) House Sponsors: Representatives Jim Mcgovern (D-MA), and Jaime Herrera- Beutler (R-WA)

TALKING POINTS ✓ Specialized medical nutrition is medically necessary for the safe and effective management of digestive

✓

✓

and inherited metabolic disorders. Without these specialized foods infants, children, and adults suffer adverse health consequences including hospitalization, intellectual impairment, behavioral dysfunction, inadequate growth, nutrient deficiencies and even death.

While this specialized nutrition is medically essential for patients, they are often expensive and not uniformly reimbursed by health insurance leaving many families with a large financial burden they cannot afford.

The Medical Nutrition Equity Act would ensure that public and private insurance cover the medically necessary nutrition required to prevent severe disabilities and death so that children and adults with digestive and inherited metabolic disorders can achieve optimal health outcomes and thrive in society.

Senator

Bob Casey (PA)

35

Representative

Jim McGovern (MA)

Representative

Jaime Herrera-Beutler (WS)

#RareDC2020

Congressional Meeting Tips ✓ Start each meeting by thanking the Member/ ✓

✓ ✓

staffer for meeting with you.

Share your personal story and explain why a specific issue is important to you. Explain the problem and how your “ask” can improve or solve it.

Make a specific “ask”. Give Congress the solution.

You don’t have to be an expert on legislation.

If you are asked a question that you are not sure how to answer, write it down and be sure to follow up.

✓ Respect the time of the Member, staffer and

✓ ✓ ✓ ✓

fellow advocates by limiting your story to no more than a minute or two. Typical meetings will last 15 minutes in total.

Leave behind a one-pager with a summary of each issue as well as your contact information.

It’s ok to ask for a photo with a Member of Congress. Make it a group shot and do it quickly.

Report back to RDLA staff on how the meeting went.

Follow up with a thank you note/ email reinforcing your asks.

RDLA Congressional Scorecard Encourage your legislator to join the Rare Disease Caucus and cosponsor rare disease legislation to improve their score.

You can view your state specific scorecard by visiting: rareadvocates.org/legislativescorecard

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#RareDC2020

Social Media Advocacy Tips What is a hashtag? “#”

•

On Twitter, Facebook and Instagram the pound sign (#) turns any word or group of words that directly follow it into a searchable link. This allows you to organize content and track discussion topics based on those keywords. For instance, if you want to post about Rare Disease Week on Capitol Hill, you would include #RareDC2020 to join the conversation. You could then click the hashtag to see other posts on Rare Disease Week on Capitol Hill.

How do I ‘mention’ someone on Twitter, Facebook or Instagram? “@”

•

Many Congressional offices have Social Media accounts to keep in touch with constituents. If you know your legislator’s handle, you can mention him or her in your post about #RareDC2020 using the “@” symbol before the name. If you don’t know your legislator’s Social Media handle, check his or Elena Silva her official website. @elena_m_silva

Before your meeting:

• •

Create a post tagging the Member’s office and the issue you will be talking about, for example:

“We are excited to meet with @amyklobuchar for #RareDC2020 to talk about ways to bring

more treatments to #Raredisease patients.”

This is a good way to introduce yourself and your issue to the staff. This will add a face to the upcoming meeting and will help them remember you.

Rare Diseases include Huntington, Sickle Cell, and thousands more you’ve never heard of. Combined, they affect 1 in 10 people (not so rare) and could unlock cures for the more common #RareDC2019 @RareAdvocates @CureCMD @ncats_nih_gov @childrenshealth 31

12:05 PM - February 27, 2019

During the meeting:

• •

Ask to take a photo, preferably towards the end of the meeting.

Write down any notes that might make for good tweets or quotes on your Facebook page.

After the meeting:

•

37

Post your picture with a thank you note on Twitter, Facebook or Instagram re-emphasizing the ask or any key points you discussed during the meeting, for example:

“Thank you @amyklobuchar for joining the

Rare Disease Congressional Caucus and supporting #RareDisease legislation!

Share Your Rare Experience Follow us and share your #RareDC2020 pictures for a chance to win an $800 travel stipend for Rare Disease Week 2021.

Tip: Be Creative! @RareAdvocates

#RareDC2020

#RareDC2020

Rare Artist Reception Thursday, February 27, 2020 5:00 p.m. - 7:00 p.m.

Capitol Hill, Hart Senate Office Building, Room 902, Washington, DC (Use “North” elevators)

 Metro Stop: Union Station (Senate side) or Capitol South (House side)  Enjoy works of art by the rare disease community.  Complimentary food and drink.  Free and open to the public.

Thank you to our presenting sponsor

RAREARTIST.ORG

February 28, 2020 #RDDNIH 8:30 am - 4:30 pm

Natcher Conference Center ● Bethesda, MD https://bit.ly/39wu9ir

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#RareDC2020

Rare Disease

Educating the next generation of rare disease advocates.

MARCH 28

RARE ON THE ROAD STOP #1

2020

Learn

Raleigh, North Carolina

APRIL 17

RARE ON THE ROAD STOP #2

Recognize

2020

Burlingame, California

MAY 30

RARE ON THE ROAD STOP #3

2020

Connect

Minneapolis, Minnesota

JUNE 27

RARE ON THE ROAD STOP #4

Discover

2020

New Orleans, Louisiana

A LIMITED NUMBER OF $150 TRAVEL STIPENDS ARE AVAILABLE

Register Now at RARETOUR.ORG

FOR MORE INFORMATION, PLEASE CONTACT: The Global Genes Events Team - events@globalgenes.org Britta Dornan - bdornan@everylifefoundation.org

39

#RAREontheRoad

#RareDC2020

ADVOCATE IN YOUR HOMETOWN!  We set up meetings with Members of Congress close to your home to advocate for legislation impacting the rare disease community.  Build relationships with legislators and their staff.  Share your rare disease story.

AUGUST 3 THROUGH SEPTEMBER 7

POWERED BY THE EVERYLIFE FOUNDATION

REGISTER ONLINE MAY 4 - JULY 3 AT RAREACROSSAMERICA.ORG

More than 650 patient advocates applied for travel stipends to attend Rare Disease Week on Capitol Hill.

Thanks to our Rare Giving sponsors we were able to grant 105 stipends.

If you are a stipend recipient, please see the Foundation table set up near registration during the Legislative Conference to pick up your check.

Please remember to bring your photo ID.

The most impactful act a rare disease advocate can make is to tell their story to policymakers. Rare Giving is here to help.

APPLY ONLINE FOR SUPPORT AT RAREGIVING.ORG

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#RareDC2020

October 17th • Des Moines, Iowa Join us for a full-day Advocacy Bootcamp!

Hear from experts on how to advance newborn screening for your disease. Learn how to: • Build coalitions • Develop and validate your diagnostic assay • Create a state pilot program • Nominate your conditions for federal Recommended Uniform Screening Panel (RUSP)

FOR FINDING THE ZEBRAS APHL Newborn Screening & Genetic Testing Symposium FALL 2020 CAMPAIGN

LEARN MORE AT RARESCREENING.ORG

save the date

rare disease legislative advocates presents

December 9, 2020

washington, dc a celebration to honor advocates who give rare disease patients a voice in state and federal policy RAREVOICEAWARDS.ORG

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#RareDC2020

Glossary

Congressional Terms

Glossary Presented by:

Bill Sponsor – A Representative or Senator who introduces a bill.

Bill Cosponsor – A Representative or Senator who formally signs on to support a bill. Only the first-named Member is the sponsor, all others are cosponsors, even those whose names appeared on the measure at the time it was submitted.

Bicameral bill – A bill that has been introduced in both the House and Senate.

Bipartisan bill – A bill that has at least one cosponsor from both parties.

Congressional Budget Office (CBO) – Agency within the legislative branch that produces independent analyses of budgetary and economic issues to support the Congressional process. Often calculates the cost or savings from enacting a specific bill. This is referred to as a “score”.

Committee – A panel with members from the House or Senate tasked with conducting hearings, examining and developing legislation, and conducting oversight.

The Senate and House have separate versions of each committee, but occasionally a joint committee is made of members from both chambers. The Energy and Commerce Committee, Ways and Means Committee, and Appropriations Committee in the House and Health, Education, Labor and Pensions Committee (HELP), Finance Committee, and Appropriations Committee in the Senate have most of the jurisdiction over healthcare issues.

•

Subcommittee – A subpanel of a committee with a more specific jurisdiction. For example, the House Energy and Commerce Committee has a Health Subcommittee.

Chair – The member of the majority party on a committee or subcommittee who has formal responsibility over the panel’s agenda and resources, presides at its meetings, and can, in some circumstances, act on the committee’s behalf.

Ranking Member – The most senior (though not necessarily the longest-serving) member of the minority party on a committee or subcommittee. The ranking member typically oversees minority committee staff and may coordinate involvement of the minority party members in committee activities.

Passed – When a bill is approved in one chamber by a majority vote.

Enacted – When a bill is passed by both chambers and signed into law by the President.

Hearing – A formal meeting of a congressional committee (or subcommittee) to gather information from witnesses for use in its activities.

Markup – Meeting by a committee or subcommittee during which committee members offer, debate, and vote on amendments to a bill or other measure.

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#RareDC2020

Glossary

Government Agencies

Glossary Presented by:

DEPARTMENT OF HEALTH AND HUMAN SERVICES (HHS) Secretary Alex Azar II, JD A cabinet-level department of the U.S. federal government with the goal of protecting the health of all Americans and providing essential human Services.

This Department includes the below agencies, among others.

NATIONAL INSTITUTES OF HEALTH (NIH) Director Francis Collins, MD, PhD The nation’s medical research agency, tasked with making discoveries that improve health and save lives.

FOOD AND DRUG ADMINISTRATION (FDA) Commissioner Stephen Hahn, M.D. Responsible for protecting the public health by ensuring the safety, efficacy, and security of human and veterinary drugs, biological products, and medical devices; and ensuring the safety of our nation’s food supply, cosmetics, and products that emit radiation.

HEALTH RESOURCES AND SERVICES ADMINISTRATION (HRSA) Administrator Thomas Engels The primary federal agency for improving access to health care services for people who are uninsured, isolated or medically vulnerable. This agency administers a number of newborn screening programs.

CENTERS FOR DISEASE CONTROL AND PREVENTION (CDC) Director Robert Redfield, MD Tasked with protecting the nation from health, safety and security threats, both foreign and in the U.S. Monitors reported disease and maintains information databases on prevalence, region, etc.

CENTERS FOR MEDICARE AND MEDICAID SERVICES (CMS) Administrator Seema Verma, MPH Administers healthcare/ reimbursement programs including Medicare, Medicaid, and the Children’s Health Insurance Program (CHIP).

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#RareDC2020

U.S. Legislative Process

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#RareDC2020

Everyone has a story to tell.

Share yours at RAREisCommunity.com Horizon is focused on researching, developing and commercializing medicines that address critical needs for people impacted by rare and rheumatic diseases. Our pipeline is purposeful: we apply scientific expertise and courage to bring clinically meaningful therapies to patients. At Horizon, we believe science and compassion must work together to transform lives.

A program by

45

#RareDC2020

FOCUSED ON DEVELOPING SPECIALTY TREATMENTS for debilitating diseases that are often difficult to diagnose and treat, providing hope to patients and their families.

Proud to support EveryLife Foundation for Rare Diseases and Rare Disease Week on Capitol Hill www.sanofigenzyme.com SAUS.GZ.18.03.1378

03/2018

WHERE OTHERS SEE COMPLEXITY, WE SEE HOPE FOR PATIENTS AND FAMILIES At Mallinckrodt, our focus is to improve the lives of patients worldwide. Making a difference is what drives us every day as we work to develop innovative therapies and cutting-edge technologies for underserved patients with severe and critical conditions.

We see challenges as opportunities to change lives.

It is our passion. It is Mallinckrodt.

Learn more at Mallinckrodt.com.

Mallinckrodt is proud to support Rare Disease Week on Capitol Hill 2020.

Mallinckrodt, the “M” brand mark and the Mallinckrodt Pharmaceuticals logo are trademarks of a Mallinckrodt company. © 2020 Mallinckrodt.

46

#RareDC2020

THE SCIENCE of POSSIBILITY

Vertex aims to create new possibilities in medicine to cure diseases and improve people’s lives.

We work with leading researchers, doctors, public health experts and other collaborators who share our vision for transforming the lives of people with serious diseases, their families and society.

Vertex is a proud sponsor of Everylife Foundation and Rare Disease Week 2020.

Vertex and the Vertex triangle logo are registered trademarks of Vertex Pharmaceuticals Incorporated Š 2020 Vertex Pharmaceuticals Incorporated 1/2020

www.vrtx.com

Providing Solutions for the Safety and Success of Clinical Trials WCG extends our best wishes to patient advocates heading to The Hill for Rare Disease Week 2020! We salute the EveryLife Foundation for Rare Diseases for its dedication to change, through science-driven public policy.

To learn more about WCG, visit us at www.wcgclinical.com and listen to our Patient Radio Podcast: www.wcgclinical. com/insights/podcast.

Better Health, Brighter Future

www.takeda.com

47

#RareDC2020

We proudly support Rare Disease Week on Capitol Hill At Retrophin, we come to work every day to do something extraordinary that can make a difference in the lives of people living with rare disease.

We are committed to collaborating with the rare disease community to identify, develop and deliver life-changing therapies as soon as possible.

Through our portfolio of approved products and promising pipeline in rare nephrology and hepatology, we strive to help people achieve the best possible outcomes today and tomorrow.

Retrophin.com | @RetrophinRare

WE LISTEN TO AND LEARN FROM PATIENTS EVERY DAY...

©2019 Ipsen Biopharmaceuticals, Inc. 01/2020 NON-US-001366 Pierre-Olivier / CAPA Pictures

Melanie Global Quality Dreux, France

We are dedicated to improving patients’ lives through innovative medicines in Oncology, Neuroscience and Rare Diseases. Because patients can’t wait. www.ipsenUS.com

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#RareDC2020

CHANGING POLICY, SAVING LIVES

We do not speak for patients.

We provide the training, education, resources and opportunities to make their voices heard.

By activating the patient advocate, we can change public policy and save lives. @EveryLifeOrg

Join Us. EVERYLIFEFOUNDATION.ORG

POWERED BY THE EVERYLIFE FOUNDATION

EVERY VOICE MATTERS

THE RDLA IS YOUR ADVOCACY PARTNER! Action Alerts

Legislative Scorecard

Event Support

Office Space in Washington, D.C.

One-on-One Policy Consulting

Monthly Newsletter

Advocacy Resources

Monthly Webinars

RAREADVOCATES.ORG

49

@RareAdvocates

#RareDC2020

Notes

TRANSFORMING THE LANGUAGE OF LIFE INTO VITAL MEDICINES

At Amgen, we believe that the answers to medicine’s most pressing questions are written in the language of our DNA. As pioneers in biotechnology, we use our deep understanding of that language to create vital medicines that address the unmet needs of patients fighting serious illness – to dramatically improve their lives.

For more information about Amgen, our pioneering science and our vital medicines, visit www.amgen.com

Amgen is proud to support Rare Disease Week on Capitol Hill. ©2020 Amgen Inc. All rights reserved.

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#RareDC2020

Notes

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#RareDC2020

Accessibility Resources GENERAL TRAVEL

• • •

visitthecapitol.gov/plan-visit/visitors-disabilities wheelchairtravel.org/washington-dc/ washington.org/dc-information/washington-dc-disability-information

TRANSPORTATION OPTIONS Metro (Subway) Capitol Shuttle wmata.com/service/accessibility/#main-content For your convenience, the Capitol Visitor Center provides an on-demand shuttle service for A list of out-of-service elevators is available at the individuals who use wheelchairs or who need information booth of every metro station. Check the mobility assistance. The shuttles run from the WMATA website or call 202-637-7000 for outages bus drop-off and pick-up areas on the West prior to leaving. side of the Capitol to the Capitol Visitor Center Ride App Services entrance at the center of the Capitol’s East UberWAV : uber.com/us/en/ride/uberwav/ Plaza. They operate continuously, as needed, Lyft Access: help.lyft.com from 8:30 a.m. – 4:30 p.m., Monday - Saturday.

• •

•

• •

CAPITOL HILL BUILDINGS, ENTRANCES AND SERVICES The Office of Congressional Accessibility Services (OCAS) Transportation Security Administration (TSA) aoc.gov/accessibility-services Information for travelers with disabilities, medical (202) 224-4048 devices, medical conditions. tsa.gov (855) 787-2227

• •

•

Capitol Buildings visitthecapitol.gov/plan-visit/visitors-disabilities

Cannabis consumption is NOT permitted in any public space. mpdc.dc.gov/marijuana

•

VEHICLES, SCOOTERS AND WHEELCHAIR RENTALS

• • • •

• 52

Wheelchair Accessible Van Rentals and Sales Ride-Away - (888) 743-3292 Wheelchair Van Rentals - (800) 910-8267 Accessible Vehicles - (301) 838-970 1119 Taft Street, Rockville, MD Scootaround - (888) 441-7575 Scooter and wheelchair rentals available daily, weekly or longer periods of time. Take a tour of DC and the National Mall on a mobility scooter.

• • •

DC Tours - (888) 878-9870.

Rent a mobility scooter or a manual wheelchair.

Daily rates.

Bike and Roll - (202) 842-BIKE. Electric scooters and manual wheel chairs available.

Two-hour, half-day, daily, and multi-day rentals.

Lenox Medical - (202) 387-1960.

Provides short-term scooter, wheelchair and knee walker rentals to tourists and local residents.

See maps on pages 54 and 56 for handicap accessible entrances to the Capitol and the Ronald Reagan Center. #RareDC2020

Station Features

Red Line • Glenmont / Shady Grove

Bus to Airport

Orange Line • New Carrollton / Vienna

Parking

Blue Line • Franconia-Springfield / Largo Town Center

Hospital

Green Line • Branch Ave / Greenbelt

Airport

Yellow Line • Huntington / Greenbelt

Connecting Rail Systems

Silver Line • Wiehle-Reston East / Largo Town Center

C

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Glenmont

o

wmata.com Information: 202-637-7000 | TTY: 202-962-2033 Metro Transit Police: 202-962-2121 | Text: MYMTPD (696873)

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Wheaton Shady Grove

D

Station in Service

Under Construction

C

Legend

n io at St er sf an Tr

System Map

D or n

King St-Old Town

St Alexandria

Eisenhower Ave

Fairfax Co

Franconia-Springfield

Huntington

Metro is accessible.

Map is not to scale

WASHINGTON METROPOLITAN AREA TRANSIT AUTHORITY © 2019

No Smoking

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No Eating or Drinking

No Animals (except service animals)

No Audio (without earphones)

No Littering or Spitting

No Dangerous or Flammable Items

#RareDC2020

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6 Atrium Lobby North Elevator

4

Atrium Lobby South Elevator

Gallery

Panera Bread

5 Atrium Ballroom

8 9

10

Atrium

2

3

Atrium Hall

12

1

11 7 14th Street Lobby North Elevator

14th Street Lobby South Elevator

Food Court

Food Court

14th Street CONCOURSE LEVEL Lower Level

1 2 3 4 5 6

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Registration Main Sessions Breakfast, Lunch, Snack Break & All-Day Beverage Service Track 2 Breakout Sessions Track 3 Breakout Sessions Medical Foods Breakout Session

Food Court

7 8 9 10 11 12

Group Photo Location Family Room Medical Station Nap Nook Nursing Room Fast Forward for Rare Video Suite

14th Street Deli Bassett’s Original Turkey California Tortilla City Lights of China Express Flamers Charbroiled Hamburgers and Chicken Forever Young Caf Gelatissimo Great Wraps

Kabuki Sushi & Teriyaki Larry’s Cookies & Ice Cream Nook Quick Pita R&B Steak and Grill Sbarro Smoothie King Subway

#RareDC2020

Capitol Hill Resources FOOD SERVICE OPTIONS ON CAPITOL HILL

• • • • • •

Longworth Cafeteria – B223—Basement level of Longworth House Office Building Rayburn Cafeteria – 2063—Ground level of Rayburn House Office Building Dirksen Cafeteria – SD-G26—Ground level of Dirksen Senate Office Building The Coffee Shop – SD-BR8—Basement level of Dirksen Senate Office Building Southside Buffet – SD-BR8—Basement level of Dirksen Senate Office Building Dirksen North Cafe – SD-BR7—Basement level of Dirksen Senate Office Building

UNDERSTANDING ROOM NUMBERS IN THE HOUSE AND SENATE OFFICE Rooms with 3 digits: The first digit in the number indicates the floor level of the room.

Example: 231 Cannon House Office Building. The room is located on the 2nd floor Example: 104 Hart Senate Office Building. The room is located on the 1st floor

• • •

Rooms with 4 digits: The first digit indicates the building. Rayburn and Longworth are the only building with 4-digit room numbers.

1 is the first number for all Longworth rooms.

Example: 1365 Longworth House Office Building 2 is the first number for all Rayburn rooms.

Example: 2145 Rayburn House Office Building The second digit in these room numbers indicates the appropriate floor level.

Example: 1365 Longworth House Office Building is located on the 3rd floor Example: 2145 Rayburn House Office Building is located on the 1st floor

• • • •

• • • •

Public Wifi Network Access House of Representatives Office Buildings Network Name: HousePublic Password: HousePublic

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Senate Office Buildings Network Name: Senate_Guest Password: constituent

#RareDC2020

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#RareDC2020

Every Voice

MATTERS

The EveryLife Foundation for Rare Diseases is a 501(c)(3) nonprofit, nonpartisan organization dedicated to advancing the development of treatment and diagnostic opportunities for rare disease patients

everylifefoundation.org @EveryLifeOrg

POWERED BY THE EVERYLIFE FOUNDATION

1012 14th Street NW, Suite 500, Washington, D.C. 20005 Office: (202) 697-RARE(7273) rareadvocates.org @RareAdvocates #RareDC2020