RDW Program 2022 Rev. by Inbound Design

PRESENTED BY

VIRTUAL EVENT

#RareDC2022 @RareAdvocates

Thank You to Our Generous 2022 Rare Disease Week on Capitol Hill Sponsors

Table of Contents Introduction Welcome 1 EveryLife Foundation Team 2 EveryLife Foundation Board of Directors 3 Schedule of Events 4-5

LEADERSHIP

RANKING

Tuesday Congressional Caucus Briefing Documentary Screening

6 7

Wednesday - Thursday Legislative Conference Agenda

8-9

Glossary Congressional Terms 19-20 U.S. Federal Agencies 21 Legislative Process 22 Sponsors 28-34

CHAMPION

CO-SPONSOR

ADVOCATE

GRASSROOTS

#RareDC2022

Welcome to Rare Disease Week

EveryLife Foundation for Rare Diseases Team

Dear Rare Disease Advocate,

OPERATIONS

Welcome to Rare Disease Week on Capitol Hill 2022! Over the years, we have been humbled to hear from attendees saying that this week jump-started their advocacy journey and changed their lives.

Lina Arslanian

Finance and Operations Manager larslanian@everylifefoundation.org

Julia Jenkins

Executive Director jjenkins@everylifefoundation.org

For some, it was the first time that they had interacted with other people who share their disease. For others, it was an eye-opening opportunity to learn about the policy issues that affect the lives of rare disease patients every day. But for all, Rare Disease Week was when they discovered the power of their voice - the patient voice.

Megan Pinegar

Chief Operating Officer mpinegar@everylifefoundation.org

Stephanie Riordan

Patient Programs Manager sriordan@everylifefoundation.org

We are honored that hundreds of advocates keep coming back each year to make their voices heard and that hundreds of new advocates continue to join us. Whether you are a first-time attendee or a veteran, we are here to walk alongside you. We are your advocacy partners.

Alyssa Terwall

Together, we will raise our voices in harmony, making policymakers understand that rare disease is not someone else’s problem. It is a public health issue that we all must care about and prioritize.

Director of Patient Programs and Events aterwall@everylifefoundation.org

Courtney Felle

Jamie Sullivan

DEVELOPMENT

Annie Kennedy

Shannon von Felden

Katelyn Laws

COMMUNICATIONS & MARKETING

Ted Brasfield

Senior Director of Development tbrasfield@everylifefoundation.org

Stephanie Siddiqi

Alliance Development Manager ssiddiqi@everylifefoundation.org

This event would not be possible without your participation. Thank you for joining us. While we look forward to seeing you in person at future events, we are heartened that hosting this event virtually enables many more advocates to embrace advocacy. The impact we make this week may change not only our lives, but the lives of rare disease patients for generations to come.

Elissa Taylor

Assoc. Director of Alliance Development etaylor@everylifefoundation.org

POLICY, ADVOCACY, AND PATIENT ENGAGEMENT

Sincerely, The EveryLife Foundation Team

#RareDC2022

Chief of Policy and Advocacy akennedy@everylifefoundation.org

RDLA Program Manager klaws@everylifefoundation.org

Baillie McGowan

State Policy Manager bmcgowan@everylifefoundation.org

Jack Meloro

Community Congress Program Coordinator jmeloro@everylifefoundation.org

Lindsey Cundiff

Rachelle Raudes

Assoc. Director of Patient Engagement lcundiff@everylifefoundation.org

State Advocacy Fellow rraudes@everylifefoundation.org

Claire Ellis

Dylan Simon

Newborn Screening and Diagnostics Policy Fellow cellis@everylifefoundation.org

Patient Engagement Fellow cfelle@everylifefoundation.org

Associate Director of Public Policy dsimon@everylifefoundation.org

Director of Public Policy jsullivan@everylifefoundation.org

Senior Director of Advocacy svonfelden@everylifefoundation.org

Brenda Colmenares

Communications Manager bcolmenares@everylifefoundation.org

Britta Dornan

Senior Director of Communications and Marketing bdornan@everylifefoundation.org

#RareDC2022

EveryLife Foundation for Rare Diseases Board of Directors

Rare Disease Week on Capitol Hill 2022 Events

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 treatments 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 rare diseases, enabling them to offer firsthand knowledge of the challenges facing the rare disease community.

TUESDAY, FEBRUARY 22

Mark Dant, Chair Executive Director, Ryan Foundation

1:00 P.M. – 2:30 P.M. ET Rare Disease Congressional Caucus Briefing “The Accelerated Approval Pathway: Reflecting the Rare Disease Community’s Priorities of Rigor, Safety, and Urgency”

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5:30 P.M. – 7:30 P.M. ET Rare Disease Documentary Screening Do Something: The Jeffrey Modell Story

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Jennifer Bernstein, Secretary Executive Vice President, Horizon Government Affairs

WEDNESDAY, FEBRUARY 23 Frank Sasinowski, MS, MPH, JD, Vice Chair Director, Hyman, Phelps & McNamara

Julia Jenkins, Board President Executive Director, EveryLife Foundation

Abbey Hauser, Young Adult, Rare Disease Advocate

Ritu Baral, Managing Director, Senior Biotechnology Analyst, Cowen and Company

Richard S. Finkel Dir. of Experimental Neurotherapeutics in Translational Neuroscience Program, St. Jude Children's Research Hospital

Presented by:

1:00 P.M. – 5:00 P.M. ET Legislative Conference Day 1

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5:00 P.M. ET Rare Artist Gallery Opens

Presented by:

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Amrit Ray, MD, MBA Senior Advisor, Bain Capital Life Sciences

Vicki Seyfert-Margolis, PhD, Treasurer Founder and CEO, MyOwnMed

Presented by:

THURSDAY, FEBRUARY 24 12:00 P.M. – 12:45 P.M. ET Coffee Chat with NCATS Acting Director Joni Rutter, Ph.D.

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Emil Kakkis, MD, PhD, Founder President/CEO, Ultragenyx

Presented by:

1:00 P.M. – 4:45 P.M. ET Legislative Conference Day 2

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5:15 P.M. – 6:00 P.M. ET Young Adult Representatives of RDLA (YARR) Meetup

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Stephen C. Groft, Pharm. D. Special Volunteer, National Center for Advancing Translational Sciences at NIH

Social Media Wall

Rare Disease Week gives us the unique opportunity to use our rare disease stories as means to push forward important legislative policy. The connections we build with lawmakers in these meetings make a real difference for the future of Rare. This week is a powerful experience in anyone’s rare disease journey.

Share your Rare Disease Week experience by posting on Instagram or Twitter with the hashtag #RareDC2022. Post by March 11 to have your post appear on our Social Wall in the navigation pane.

Thank You to Our Social Media Wall Sponsor

- Abbey Hauser, Rare Disease Week 2021

#RareDC2022

Rare Disease Congressional Caucus Briefing

Rare Disease Week on Capitol Hill 2022 Events

TUESDAY, FEBRUARY 22

TUESDAY, MARCH 1 9:00 A.M. – 5:00 P.M. ET Virtual Meetings with U.S House of Representatives

1:00 P.M. – 2:30 P.M. ET

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THE ACCELERATED APPROVAL PATHWAY: REFLECTING THE RARE DISEASE COMMUNITY’S PRIORIES OF RIGOR, SAFETY AND URGENCY

WEDNESDAY, MARCH 2

Presented by:

9:00 A.M. – 5:00 P.M. ET Virtual Meetings with U.S Senators

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Virtual Photo Booth

Moderator: Frank Sasinowski, M.S., M.P.H., J.D Hyman, Phelps & McNamara & Everylife Foundation for Rare Diseases, Board Vice Chair

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Introduction: Eric Dube, CEO, Travere Therapeutics

SPEAKER PANEL

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Share your experience during Rare Disease Week by taking a selfie in our Virtual Photobooth and posting it on social media with the hashtag #RareDC2022.

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Thank you to our Virtual Photobooth Sponsor

Ellis Unger, MD former Director, FDA OCHEN, ODE

Pat Furlong Founder and CEO, Parent Project Muscular Dystrophy (PPMD)

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Teonna Woolford, CEO , Sickle Cell Reproductive Health Education Directive

GK Raju, PhD, MS Light Pharma Inc.

RECORDED REMARKS BY

2021 Snapshots

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#RareDC2022

Representative G.K Butterfield (NC)

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Senator Roger Wicker (MS)

Senator Amy Klobuchar (MN)

#RareDC2022

Rare Disease Documentary Screening

Legislative Conference Agenda

TUESDAY, FEBRUARY 22

WEDNESDAY, FEBRUARY 23

Presented by:

1:00 P.M. – 5:00 P.M. ET

5:30 PM - 7:30 PM ET

Presented by:

WELCOME AND 2022 LEGISLATIVE ASKS

WELCOME RECEPTION

1:00 P.M. ET Welcome: Heidi Evita Moore, Senior Director, Diversity & Inclusion, Horizon Therapeutics Shannon von Felden, EveryLife Foundation for Rare Diseases Caitlin Van Sant, Office of Representative G. K. Butterfield Debbie Jessup, Office of Representative Lucille Roybal-Allard Kirby Miller, Office of Senator Roger Wicker Jay Eberle, Office of Senator John Barrasso

5:30 P.M – 6:15 P.M. ET

• • • • • •

DOCUMENTARY SCREENING AND PANEL DISCUSSION 6:15 P.M – 7:30 P.M. ET

GUEST PANEL

LANDMARK LEGISLATIVE VEHICLES IN 2022: PDUFA VII AND CURES 2.0

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2:00 P.M. ET Moderator: Nicholas Manetto, Faegre Drinker Eric Gascho, National Health Council Sherie Lou Santos, Office of Representative Dianna DeGette Mark Ratner, Office of Representative Fred Upton Annie Kennedy, EveryLife Foundation for Rare Diseases

• • • • •

Moderator: Tilea West Rare Disease Patient, Regulatory Research Coordinator- Phase 1 Oncology

DEEP POLICY DIVE #1—SPEEDING THERAPY ACCESS TODAY ACT 3:00 P.M. ET Moderator: Dan Whiting, National Fragile X Foundation Chris Jones, Office of Representative Gus Bilirakis Jill Wood, Phoenix Nest Bio Tech Kelly Maynard, Little Hercules Jamie Sullivan, EveryLife Foundation for Rare Diseases

• • • • •

Vicki & Fred Modell Co-Founders Jeffrey Modell Foundation

STATE/REGIONAL NETWORKING HAPPY HOUR 4:00 P.M. ET Join advocates from your state or region to network and socialize.

THURSDAY FEBRUARY 24

12:00 P.M. – 4:45 P.M. ET

COFFEE CHAT WITH NCATS ACTING DIRECTOR JONI RUTTER, PH.D. 12:00 P.M. ET Moderator: Shannon von Felden, EveryLife Foundation for Rare Diseases

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Networking Lounge Drop in and say hello in our Virtual Networking Lounge! Take part in group chats with other advocates and speakers throughout Rare Disease Week

JOIN THE NETWORKING LOUNGE

DEEP POLICY DIVE #2—NEWBORN SCREENING: REAUTHORIZATION AND BEYOND 1:00 P.M. ET Moderator: Kate Tighe, Sanofi Dylan Simon, EveryLife Foundation for Rare Diseases Garrett Devenney, Senate Committee on Health, Education, Labor, and Pensions Zhanzhi Hu, Project GUARDIAN Don Bailey, RTI International

Lounge Presented by:

• • • • • #RareDC2022

#RareDC2022

Hill Day Issue Information

BREAKOUT SESSIONS

2:00 PM ET

LEGISLATIVE HOT TOPICS

• • • •

Moderator: Liesl Broadbridge, ScM, CGC, Rutgers University Ryan Fisher, Parent Project Muscular Dystrophy John Richardson, National Society of Genetic Counselors Annie Kennedy, EveryLife Foundation for Rare Diseases

Legislative Ask #1

YOUNG ADULT REPRESENTATIVES OF RDLA (YARR): DEEP POLICY DIVE

• • •

Cosponsor the Speeding Therapy Access Today Act of 2021, H.R. 1730/S. 670

Moderator: Courtney Felle, EveryLife Foundation for Rare Diseases Christina Brundage, YARR Laura Romano, YARR

BREAKOUT SESSIONS

• • • • •

Presented by:

 More than 30 million Americans are living with one or more rare disease.  Between 93% and 95% of the more than 7,000 known rare diseases have no U.S. Food and Drug

Introductions: Tamar Thompson, Alexion Pharmaceuticals Moderator: Linda Blount, Black Women’s Health Imperative Lauren Citron, Office of Representative Bobby Rush Shayne Woods, Office of Senator Tim Scott Daisy Kim, Asian and Pacific Islander American Health Forum

  

HOW DATA CAN POWER RARE DISEASE ADVOCACY

• • • • •

Moderator: Jamie Sullivan, EveryLife Foundation Eric Sid, National Center for Advancing Translational Sciences Melissa Haendel, University of Colorado Anschutz School of Medicine Ryan Shay, Faegre Drinker Marc Yale, International Pemphigus and Pemphigoid Foundation

Administration approved therapy. The development process for a rare disease drug takes an average of 15 years. Traditional regulatory processes have become more complex involving combinations of therapies, genomics, novel diagnostic tests, multi-systemic diseases, small patient populations, and precision medicine. As a result, numerous parts of the regulatory system need to cohesively work together. When new therapies for rare diseases are approved, patients often face unnecessary delays and barriers to access, resulting in avoidable deterioration in health.

STAT ACT VISION  The STAT Act will enact targeted, impactful, and attainable policy reforms at the Food and Drug

NETWORKING HAPPY HOUR ON ADVOCACY AND POLICY TOPICS 4:00 P.M. ET State Advocacy • Moderators: Rachelle Raudes and Shannon von Felden, EveryLife Foundation for Rare Diseases

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How to Engage with State DURs and P&T Committees • Moderators: Baillie McGowan and Jack Meloro, EveryLife Foundation for Rare Diseases

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Newborn Screening RUSP Alignment • Moderators: Dylan Simon and Claire Ellis, EveryLife Foundation for Rare Diseases

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SPEEDING THERAPY ACCESS TODAY

TALKING POINTS

3:00 PM ET

HOW CONGRESS CAN WORK TO REDUCE BARRIERS TO CARE AND HEALTH DISPARITIES

STAT Act

Starting Your Advocacy Journey as a New Rare Disease Advocate • Moderators: Sarah Tompkins; Rare Disease Week Chair, Sarita Edwards; Rare Disease Week Vice Chair and Katelyn Laws, EveryLife Foundation for Rare Diseasesw

Administration (FDA) to accelerate development of therapies across the spectrum of rare diseases and disorders and facilitate patient access to such therapies. Improve rare disease coordination, stakeholder engagement, and policy development within FDA by expanding existing authority to create a Rare Disease Center of Excellence. Inform rare disease policies and actions by creating a Rare Disease and Condition Drug Advisory Committee. Fund regulatory science and related activities to support development of therapies to treat very small rare disease populations. Strengthen rare disease patient access to FDA-approved therapies in both public and commercial plans through enhanced FDA and Centers for Medicare and Medicaid Services coordination, proactive engagement of pay.

YOUNG ADULT REPRESENTATIVES OF RDLA (YARR) MEETUP 5:15 P.M. ET YARR Members will gather following the Legislative Conference to discuss the day’s events. Anyone 16-30 years old is welcome to attend whether or not they are a member of YARR. DOWNLOAD

#RareDC2022

Hill Day Issue Information Legislative Ask #2 Cosponsor the Newborn Screening Saves Lives Reauthorization Act, S. 350/H.R. 482

Hill Day Issue Information Legislative Ask #3

Newb

Cosponsor the Better Empowerment Now to Enhance Framework and Improve Treatments (BENEFIT) Act, S. 373

Screening

TALKING POINTS

 Of the four million babies born in the U.S. each year, 1 in 300 are found to have a potentially devastating

 While much progress has been made in driving forward policies and procedures to ensure the patient

condition through newborn screening.

functional conditions that are not otherwise apparent at birth.  Diagnosis through newborn screening saves lives, improves healthcare outcomes, and reduces long-term healthcare costs by allowing for detection and intervention at the earliest moment possible.  Newborn screening is the most successful public health program in the history of our country.

perspective is considered by FDA, reviewers evaluating candidate drugs and other medical products, some significant gaps remain.  One such gap is the lack of any requirement in law today that the FDA include patient experience or patient-focused drug development (PFDD) data as a part of its risk-benefit framework.  This means that the agency’s signature tool for evaluating risk-benefit does not have 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.

KEY BILL PROVISIONS

THE BENEFIT ACT

 Reauthorizes the Health Resources and Services Administration (HRSA) state grants to expand and

 The BENEFIT Act will enhance an important transparency and accountability provision included in the

 20,000 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

 

improve screening programs, provide educational resources to 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 advises the Secretary of Health and Human Services on newborn and childhood screening policies and priorities to enhance state health agencies to ensure screening is available to every eligible infant. Includes the evidence-based federal Recommended Uniform Screening Panel (RUSP).

21st Century Cures Act by requiring the FDA to share how such patient experience and PFDD data was considered within the risk-benefit assessment for any approved therapies.  This will provide additional learnings to all stakeholders, particularly patients, and help further refine and develop such tools going forward.  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 advocac organization or academic institution – be considered as part of the risk-benefit assessment.  Many significant statutory and regulatory advances have been made in the past decade to ensued that patient experience is meaningfully incorporated into product development and regulatory review processes.  The BENEFIT Act 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.

BACKGROUND  In 2008, Congress passed the original Newborn Screening Saves Lives Act (P.L. 110-204), which  

established national newborn screening guidelines and helped facilitate comprehensive newborn screening in every state. The Act was first reauthorized in 2014. Prior to this act, the number and quality of newborn screening tests varied greatly by state. In 2007, only 10 states and the District of Columbia required infants to be screened for all the recommended disorders. Today, all 50 states and the District of Columbia require screening for at least 31 treatable conditions, as recommended by the Department of Health and Human Services. .

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#RareDC2022

DOWNLOAD

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Hill Day Issue Information

Legislative Ask #4

About Rare Disease Congressional Caucus About thethe Rare Disease Congressional Caucus

Cosponsor the Access to Genetic Counselor Services Act, S. 1450/H.R. 2144

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

TALKING POINTS TALKING POINTS

 Genetic counselors are experts with advanced training in medical genetics and counseling, uniquely equipped 

 

to support patients, families, and providers throughout the genetic testing process. Genetic counselors improve patient care and healthcare costs. The services of the more than 5,800 certified genetic counselors in the U.S. are increasingly important to patients and providers given advances in genetic testing, with approximately ten new genetic tests becoming available daily, and as genetic testing is becoming more complex. Limited access to genetic counseling services can result in increased wait-times for genetic testing and subsequent delays in diagnosis. Current Medicare policy denies Medicare beneficiaries direct access to highly trained, board-certified genetic counselors to navigate complex factors affecting their health.

 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.  There are more than 7,000 known rare diseases but unfortunately, 93-95% 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 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.  The Rare Disease Congressional Caucus helps bring public and Congressional awareness to the unique needs of the rare disease community (including patients, physicians, scientists, and industry), and creates opportunities to address barriers to the development of and access to life-altering treatments.  The Caucus gives a permanent voice to the rare disease community on Capitol Hill. Working together we can find solutions that transform hope into therapies and cures.

ACCESS TO GENETIC COUNSELOR SERVICES ACT  Enact S. 1450/H.R. 2144 to update Medicare law to improve the lives of up to 60 million Americans, including    

those with rare diseases. Provide beneficiaries direct access to genetic counselors through direct access and billing. S. 1450/H.R. 2144 will enable genetic counselors to bill Medicare directly and be reimbursed for services delivered to Medicare beneficiaries at 85% of physician payment levels. Improve care for all patients seeking genetic counseling by facilitating the ability of additional physician practices and hospitals to employ genetic counselors. Help to address health disparities and inequities that affect those with lower incomes, improving care for vulnerable, dual eligible Medicare-Medicaid beneficiaries.

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#RareDC2022

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Congressional Meeting Tips HOUSE

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Mark Amodei NV-2 Jake Auchincloss MA-4 Cindy Axne IA-3 Don Bacon NE-2 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-At Large 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 Emanuel Cleaver, II MO-5 Steve Cohen TN-9 Jenniffer González Colón PR-AL James Comer KY-1 Gerald Connolly VA-11 Jim Cooper TN-5 Jason Crow CO-6 Rodney Davis IL-13 Sharice Davids KS-03 Peter DeFazio OR-4 Diana DeGette CO-1 Rosa De Lauro CT-3 Suzan DelBene WA-1 Antonio Delgado NY-19 Ted Deutch FL-21 Debbie Dingell MI-12 Mike Doyle PA-14 Tom Emmer MN-6 Anna Esho CA-18 Brian Fitzpatrick PA-1

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Lizzie Fletcher TX-7 Jeff Fortenberry NE-1 Ruben Gallego AZ-7 John Garamendi CA-3 Josh Gottheimer NJ-5 Garret Graves LA-6 Brett Guthrie KY-2 Jaime Herrera-Beutler WA-3 Kevin Hern OK-1 Jim Himes CT-4 Richard Hudson NC-8 Jared Huffman CA-2 Dusty Johnson SD-AL Hank Johnson GA-4 Mondaire Jones NY-17 David P. Joyce OH-14 Marcy Kaptur OH-9 Ro Khanna CA-17 Derek Kilme WA-6 Andy Kim NJ-3 Ron Kin WI-3 Raja Krishnamoorthi IL-8 Darin LaHood IL-18 Jim Langevin RI-2 John Lason CT-1 Bob Latta OH-5 Susie Lee NV-3 Teresa Leger Fernández NM-3 Debbie Lesko AZ-08 Andy Levin MI-09 Mike Levin CA-49 Ted Lieu CA-33 Zoe Lofgren CA-19 Alan Lowenthal CA-47 Blaine Luetkemeyer MO-9 Stephen Lynch MA-8 Tom Malinowski NJ-7 Nicole Malliotakis NY-11 Carolyn Maloney NY-12 Sean Patrick Maloney NY-18 Kathy Manning NC-6 Brian Mast FL-18 Doris Matsui CA-6 Michael McCaul TX-10 Jim McGovern MA-2 David McKinley WV-1 Cathy McMorris Rodgers WA-5

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Grace Meng NY-6 Markwayne Mullin OK-2 Seth Moulton MA-6 Richard Neal MA-1 Donald Norcross NJ-1 Ralph Norman SC-5 Eleanor Norton DC Tom O'Halleran AZ-1 Steven Palazzo MS-4 Frank Pallone NJ-6 Jimmy Panetta CA-20 Chris Pappas NH-01 Bill Pascrell NJ-9 Donald Payne, Jr. NJ-10 Scott Peters CA-52 Dean Phillips MN-03 Chellie Pingree ME-1 Katie Porter CA-45 Bill Posey FL-8 David Price NC-04 Mike Quigley ILJamie Raskin MD-8 Kathleen Rice NY-4 Martha Roby AL-2 Deborah Ross NC-2 David Rouzer NC-7 C.A. Dutch Ruppersberger MD-2 John Rutherford FL-4 Mary Gay Scanlon PA-5 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 Chris Smith NJ-4 Adam Smith WA-9 Jason Smith MO-8 Lloyd Smucker PA-16 Darren Soto FL-9 Jackie Speier CA-14 Chris Stewart UT-2 Steve Stivers OH-15 Marilyn Strickland WA-10 Thomas Suozzi NY-03 Eric Swalwell CA-15

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Glenn Thompson PA-5 Rashida Tlaib MI-13 Paul Tonko NY-20 Lori Trahan MA-3 Fred Upton MI-6 Jeff Van Drew NJ-2 Juan Vargas CA-51 Nydia Velazquez NY-7 Ann Wagner MO-2 Debbie Wasserman-Schultz FL-23 Bonnie Watson Coleman NJ-12 Bruce Westerman AR-4 Jennifer Wexton VA-10 Susan Wild PA-7 Joe Wilson SC-2 Robert Wittman VA-1 John Yarmuth KY-3 Lee Zeldin NY-1

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John Barrasso WY John Boozman AR Mike Braun IN Maria Cantwell WA Shelley Moore Capito WV Christopher Coons DE Tom Cotton AR Steve Daines MT Chuck Grassley IA 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 Jerry Moran KS Alex Padilla CA Gary Peters MI Jame Risch ID Kyrsten Sinema AZ Jeanne Shaheen NH Tina Smith MN Debbie Stabenow MI Chris Van Hollen MD Roger Wicker* MS

 Start each meeting by thanking the Member/staffer 

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SENATE

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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. Email the Congressional staffer with a one-pager on your asks as well as your contact information. Remember rare disease issues are nonpartisan. Don’t talk about politics in your meetings. Report back to RDLA on how the meeting went by filling out the Online Meeting Feedback Form. Follow-up with a thank you note/ email reinforcing your asks.

VIRTUAL TIPS  Virtual meetings with your legislators on Zoom can

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be just as productive and informative as in-person meetings. Before your scheduled Zoom meetings, check your lighting and background, and turn on your camera for your meeting if you can. Take a screenshot with a Member of Congress. Ask permission first. Make it a group screenshot and do it quickly.

185 27

Representatives

122 Democrats

Senators

1 Independent

Resources Presented by:

63 Republicans

#RareDC2022

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

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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 #RareDC2022 to join the conversation. You could then click the hashtag to see other posts about Rare Disease Week on Capitol Hill.

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

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Many Congressional offices have social media accounts to keep in touch with constituents. If you know your legislator’s handle, you can mention them in your post about #RareDC2022 using the “@” symbol before the name. If you don’t know your legislator’s social media handle, check their official website.

Before your meeting:

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Create a post tagging the Member’s office and the issue you will be talking about, for example: “We are excited to meet with @repgusbilirakis for #RareDC2022 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.

During the meeting:

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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:

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Post your picture with a thank you note on social media "re-emphasizing" the 'ask' or any key points you discussed during the meeting, for example: “Thank you @repgusbilirakis for joining the Rare Disease Congressional Caucus and supporting #RareDisease legislation #RareDC2022.”

ANNOUNCEMENT!

Share Your Rare Experience Share your Rare Disease Week experience and include how has RDW changed your life or if new to RDW why are you attending? Include #RAREDC2022 for a chance to win a $100 Gift Card of your choice.

Are you ready to take your advocacy to the next level? Tune in during the Legislative Conference on Wednesday, February 23 to learn about a NEW opportunity for experienced advocates.

Resources Presented by:

The Scavenger Hunt code is: RareDiseaseWeek2022 17

#RareDC2022

Glossary Congressional Terms

Glossary Presented by:

Act – A bill that has passed both house of congress and has been enacted into a law.

Constituent – Citizens within a district of a legislator; the voters that elect a representative.

Adoption – Formally approve of; usually in reference to a change or amendment.

“Died in committee” – A bill that was considered and rejected by a committee; not returned to the house for action.

Amendment – A formal change (or proposed change) made to a piece of legislation.

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

Appeal – Asking a higher court to change or reverse the decision of a lower court.

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

Appropriation – The allocation of funds for a specific purpose within government. Allows for funds to be spent but is not an actual expenditure.

Joint session – Meeting of both the House and the Senate in one chamber.

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

Legislator – An elected official of a legislative body.

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.

Legislature – The branch of government responsible for enacting laws. Lobbyist – A person who attempts to influence legislation on behalf of a specific interest group. Majority Party – The political party having electoral strength sufficient to permit it to win control of a government.

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.

Minority Party – A political party whose electoral strength is so small as to prevent its gaining control of a government except in rare and exceptional circumstances.

Cabinet – A body consisting of the Vice President of the United State and the heads of the executive branch’s federal executive departments

Motion – A proposal asking for the Senate or House to take an action. Nonpartisan – Not associated with a single political party or caucus.

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”.

Partisan – Being associated with a single political party or caucus. Passed – When a bill is approved in one chamber by a majority vote (most legislation requires a 60-vote majority in the Senate).

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

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Petition – A formal written request submitted by anyone other than the legislature (individuals, boards, commissions, cities, etc.).

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.

Quorum – The minimum number of members of the legislature necessary to conduct business. Ranking Member – The most senior (though not necessarily the longest-serving) member of the minority party on a committee or subcommittee.

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

Recess – The temporary break of a daily session. Can refer to breaks within the same day, overnight, or longer breaks (such as over holidays).

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.

Resolution – An official communication of an intent by a legislature or a chamber. Session – The period of time that the legislature meets and carries out its regular business.

Caucus – An informal meeting of members of a body of government (typically belonging to the same political party and/or another common interest such as the Rare Disease Caucus).

Sponsor – A member of the legislature who submits a bill or other measure for consideration. Other interested members can join as cosponsors.

Companion Bill – A bill introduced in either the House or Senate which has identical or similar language to another bill introduced in the other chamber.

Veto – Rejection of a bill or resolution by the president or governor. The bill can still become law if the House and Senate both vote (by a two-thirds majority) to override the veto. DOWNLOAD

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Glossary

U.S. Legislative Process

Health-Related Federal Government Agencies

The U.S. Legislative Process

AGENCY FOR HEALTHCARE RESEARCH AND QUALITY (AHRQ) The Agency for Healthcare Research and Quality (AHRQ) was established to fund and conduct research aimed at improving the quality of healthcare. Additionally, AHRQ is tasked with disseminating this evidence and translating it into clinical practice.

CENTERS FOR DISEASE CONTROL AND PREVENTION (CDC)

HOUSE OF REPRESENTATIVES

SENATE

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.

A bill is introduced by a member of the Senate

CENTERS FOR MEDICARE AND MEDICAID SERVICES (CMS)

It starts with an idea

A bill is introduced by a member of the House

Administers healthcare/reimbursement programs including Medicare, Medicaid, and the Children’s Health Insurance Program (CHIP). A committee meets to discuss, amend and vote on the bill

DEPARTMENT OF DEFENSE (CDMRP)

The Department of Defense hosts the Congressionally Directed Medical Research Programs. CDMRP was created in 1992 via a Congressional appropriation to foster novel approaches to biomedical research in response to the expressed needs of its stakeholders. Stakeholders include the American public, the military and Congress.

A bill must pass both chambers of Congress

A committee meets to discuss, amend and vote on the bill

A conference committee, made up of members of both chambers, reconciles any differences between the Senate and the House versions of the bill

DEPARTMENT OF EDUCATION The department of the U.S federal government that administers federal programs related to education. Bill proceeds to the full Senate, where it is debated/amended and brought to a vote

SOCIAL SECURITY ADMINISTRATION (SSA)

An independent agency of the U.S. federal government that administers Social Security, a social insurance program consisting of retirement, disability, and survivor benefits.

Bill proceeds to the full House, where it is debated/amended and brought to a vote Both chambers vote on the final bill

DEPARTMENT OF HEALTH AND HUMAN SERVICES (HHS)

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 (12 total). Congress can override the veto by a 2/3 vote of both chambers

FOOD AND DRUG ADMINISTRATION (FDA)

Responsible for protecting 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 productsthat emit radiation.

The president vetoes the bill and sends it back to Congress

HEALTH RESOURCES AND SERVICES ADMINISTRATION (HRSA)

The primary federal agency for improving access to health care services for people who are uninsured, isolated or medically vulnerable. This agency administers several newborn screening programs.

NATIONAL INSTITUTES OF HEALTH (NIH)

Thank you to Faegre Drinker for their support of the rare disease community’s policy and legislative priorities.

The nation’s medical research agency tasked with making discoveries that improve health and save lives. Comprised of 27 institutes and centers.

The president signs the bill into law

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THERE ARE 400 MILLION AROUND THE WORLD LIVING WITH A RARE DISEASE. We believe it is our responsibility to listen to, understand, and change their lives, and seek opportunities to better collaborate with everyone involved in the patient journey, including those who provide care and access. Alexion.com

We are in rare for life. At Travere Therapeutics, we come together every day to help patients, families and caregivers of all backgrounds as they navigate life with a rare disease. We know the need for treatment options is urgent — that is why our global team works with the rare disease community to identify, develop and deliver life-changing therapies. Travere Therapeutics is proud to support Rare Disease Week on Capitol Hill 2022.

travere.com | @TravereRare | #InRareForLife

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WHERE OTHERS SEE COMPLEXITY, WE SEE HOPE FOR PATIENTS AND FAMILIES

Interested in learning about gene therapy? Welcome home. Whether you are just beginning or have already started your journey toward understanding gene therapy—genehome is your go-to source for educational resources.

A proud sponsor of Rare Disease Week on Capitol Hill 2022

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 patients with severe and critical conditions.

START EXPLORING

Mallinckrodt is proud to support Rare Disease Week on Capitol Hill 2022.

www.bio.org

Precision that moves We are proud to support Virtual Rare Disease Week on Capitol Hill 2022.

At Blueprint Medicines, we work together in pursuit of one common goal: to dramatically improve the lives of people with genomically defined cancers and rare diseases.

Together we are delivering

For more information, visit blueprintmedicines.com/patients/

Together we are working to transform the treatment of rare genetic diseases that were previously thought to be untreatable.

Rob T. Living with GIST

Diane L. Living with NSCLC

Visit helpful resources for the community on rare genetic diseases and the potential of gene therapy.

Creating a future where rare disease is a thing of the past. SM = systemic mastocytosis NSCLC = non-small cell lung cancer GIST = gastrointestinal stromal tumor

The image depicted contains models and is being used for illustrative purposes only. Janssen Research & Development, LLC © JRD, LLC 2022

cytokinetics.com

thegenehome.com

Kristine F. Living with SM

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USBP-PRNP-22.004.1

We are Janssen, the Pharmaceutical Companies of Johnson & Johnson. Bold Thinkers. Big Dreamers. Fearless advocates on behalf of patients. So that one day, the world’s most daunting rare immune-mediated diseases will be found only in the pages of history books. Learn more at www.janssen.com

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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 Virtual Rare Disease Week on Capitol Hill 2022 www.vrtx.com Vertex and the Vertex triangle logo are registered trademarks of Vertex Pharmaceuticals Incorporated. ©2022 Vertex Pharmaceuticals Incorporated | 2/2022

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WHAT IS AADC DEFICIENCY? Aromatic L-amino Acid Decarboxylase (AADC) deficiency is a disorder that interferes with the way the cells in the nervous system talk to each other through substances called neurotransmitters. In AADC deficiency, a genetic mutation (change within a gene) leads to a decrease in the amount of neurotransmitters made.

The most common symptoms are: Low muscle tone, also called “hypotonia,” is the most common symptom Delayed development. A child with AADC deficiency may be unable to: – Lift and control his or her head – Crawl, sit, or stand without support

Other common symptoms you might notice are: Excessive sweating Drooling Drooping eyelids Stuffy or runny nose

– Walk – Babble or say words Other involuntary movements, such as sudden jerking, flailing, or twisting

These symptoms may also be present: Seizures Difficulty sleeping Behavior problems (irritability or excessive crying) Problems with digestion – Diarrhea, constipation, reflux Problems with feeding – Trouble swallowing without choking or coughing

Involuntary eye movements (called oculogyric crises) are moments when a child’s eyes suddenly roll upward involuntarily. They can last anywhere from a few seconds to hours, and can happen several times a day or several times a week. When this happens, it can look like the child is having a seizure.

If you want to learn more about AADC deficiency and receive helpful resources, you can email AADCpatientengagement@ptcbio.com or visit AboutAADC.com.

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Every Voice MATTERS

The EveryLife Foundation for Rare Diseases is a 501(c)(3) nonprofit, nonpartisan organization dedicated to empowering the rare disease patient community to advocate for impactful, science-driven legislation and policy that advances the equitable development of and access to lifesaving diagnoses, treatments and cures.

everylifefoundation.org

@EveryLifeOrg

1012 14th Street NW, Suite 500, Washington, D.C. 20005 Office: (202) 697-RARE(7273) rareadvocates.org @RareAdvocates #RareDC2022

RDW Program 2022 Rev.

Articles inside

Documentary Screening

Legislative Conference Agenda