Rare Disease Week 2021 Program Updated by Inbound Design

PRESENTED BY

J U LY

14-22

2021

#RareDC2021 @RareAdvocates

Thank You to Our Generous 2021 Rare Disease Week on Capitol Hill Sponsors PRESIDENTIAL

LEADERSHIP

RANKING

CHAMPION

CO-SPONSOR

GRASSROOTS

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

6 7

Thursday - Friday Legislative Conference Agenda Hill Day Issue Information Congressional Meeting Tips Social Media Advocacy Tips

10-13 16 17

About Rare Disease Legislative Advocates Rare Disease Week 2022 STAT Act EveryLife Foundation Rare Artist Newborn Screening Bootcamp RareVoice Awards #RAREis Scholarship Rare Giving Community Congress

25 25 26 26 27 28 28 29 29 30

10 year Anniversary Spread

19-20

Glossary Congressional Terms U.S. Federal Agencies

21-22 23

8-9

Welcome to Rare Disease Week

EveryLife Foundation for Rare Diseases Team OPERATIONS

Dear Rare Disease Advocate,

Lina Arslanian Finance and Operations Manager larslanian@everylifefoundation.org

Thank you for joining us for Rare Disease Week on Capitol Hill 2021. Ten years ago, 70 advocates traveled from around the country to participate in the Rare Disease Legislative Advocates first Hill Day event. Over the years, the day has evolved into a series of events we now know as Rare Disease Week on Capitol Hill. In 2020, more than 900 advocates participated, united in a common goal: To make their rare voices heard.

Julia Jenkins Executive Director jjenkins@everylifefoundation.org Mary Morlino Patient Engagement Consultant mmorlino@everylifefoundation.org Megan Pinegar Senior Director of Operations and Human Resources mpinegar@everylifefoundation.org Stephanie Riordan Patient Programs Manager sriordan@everylifefoundation.org

While we are gathering virtually this year and not on the Capitol steps, your engagement in this event is vital. The National Economic Burden of Rare Disease Study reinforces the urgency of our mission. This groundbreaking study found that the 379 rare diseases surveyed cost the U.S. an estimated $1 trillion in 2019 and that rare disease patients must wait an average of 6.3 years before receiving a confirmed diagnosis. We must make our rare voices heard to advance diagnostics, treatments, and cures. Because every voice matters.

Alyssa Terwall Associate Director of Special Events aterwall@everylifefoundation.org

DEVELOPMENT Ted Brasfield Senior Director of Development tbrasfield@everylifefoundation.org

aterwall@everylifefoundation.org Stephanie Goncalves Alliance Development Manager scongcalves@everylifefoundation.org

Elissa Taylor Associate Director of Alliance Development etaylor@everylifefoundation.org

POLICY, ADVOCACY, AND PATIENT ENGAGEMENT

Whether you are joining us for the first time or the tenth, we hope this week empowers and inspires you. The impact we make this week will help all rare disease patients for generations to come. This event would not be possible without your participation. Thank you for being an advocate!

Kylie Barber Medical Foods Policy Associate kbarber@everylifefoundation.org

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

Liesl Broadbridge Policy Fellow lbroadbridge@everylifefoundation.org

Katelyn Laws RDLA Program Coordinator klaws@everylifefoundation.org

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

Jack Meloro Community Congress Program Coordinator jmeloro@everylifefoundation.org

Swapna Kakani RDLA State Advocacy Fellow skakani@everylifefoundation.org

Sincerely, The EveryLife Foundation Team

Dylan Simon Newborn Screening and Diagnostics Policy Manager dsimon@everylifefoundation.org Jamie Sullivan Director of Public Policy jsullivan@everylifefoundation.org Shannon von Felden Director of Rare Disease Legislative Advocates svonfelden@everylifefoundation.org

Adrian Palau-Tejeda Diversity Inclusion Advocacy Fellow apalau-tejeda@everylifefoundation.org

COMMUNICATIONS & MARKETING Brenda Colmenares Communications Manager bcolmenares@everylifefoundation.org

#RareDC2021

Britta Dornan Senior Director of Communications and Marketing bdornan@everylifefoundation.org

#RareDC2021

Rare Disease Week on Capitol Hill 2021 Events

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

Mark Dant, Chair Executive Director, Ryan Foundation

Jennifer Bernstein, Executive Vice President, Horizon Government Affairs

Frank Sasinowski, MS, MPH, JD, Vice Chair Director, Hyman, Phelps & McNamara P.C.

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

Julia Jenkins, Secretary Executive Director, EveryLife Foundation

WEDNESDAY, JULY 14

•

6:00 P.M. – 7:30 P.M. ET Rare Disease Documentary Screening A Mystery to Me

Presented by:

THURSDAY, JULY 15

Presented by:

•

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

•

5:00 P.M. Rare Artist Gallery Opens

•

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

FRIDAY, JULY 16 Vicki Seyfert-Margolis, PhD, Treasurer Founder and CEO, MyOwnMed

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

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

Presented by:

1:00 P.M. – 5:00 P.M. ET Legislative Conference Day 2

•

5:15 P.M. – 6:00 P.M. ET YARR Leadership Academy Graduation

Presented by:

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6:00 P.M. – 7:00 P.M. ET YARR Meetup

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

"We can build tomorrow on the strength and the courage of our own gifts. The gift of your voice. The rare voice. The rare disease community is strong. Your voice can and will change tomorrow."

Presented by:

1:00 P.M. – 2:00 P.M. ET Rare Disease Congressional Caucus Briefing “Rare Disease Therapy Development: Existing Challenges and Proposed Innovative Solutions”

Presented by:

Social Media Wall Share your Rare Disease Week exerience by posting on Instagram or Twitter with the hashtag #RareDC2021. Posts made from July 7th - 30th, will appear on our Social Wall, located on the navigation pane. Click here

Thank you to our Social Media Wall Sponsor

Premier Technology Thank you to our Premier Technology Sponsor, whose support allows for closed captioning, translation and other accessibility features.

- Mark Dant, Rare Disease Week 2020

#RareDC2021

Rare Disease Week on Capitol Hill 2020 Events

Rare Disease Congressional Caucus Briefing

TUESDAY, JULY 20 9:00 A.M. – 5:00 P.M. ET Virtual Meetings with House Representatives

WEDNESDAY, JULY 14

•

1:00 P.M. – 2:00 P.M. ET

WEDNESDAY, JULY 21

RARE DISEASE THERAPY DEVELOPMENT: "EXISTING CHALLENGES AND PROPOSED INNOVATIVE SOLUTIONS"

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

•

THURSDAY, JULY 22 2:00 P.M. – 3:30 P.M. ET Diversity Roundtable

Presented by:

•

Virtual Photobooth

Moderator: Amrit Ray, MD, Board Member, EveryLife Foundation for Rare Diseases

Introduction: Eric Dube, CEO, Travere

Representative Gus Bilirakis (FL), Rare Disease Caucus Co-Chair

Senator Amy Klobuchar (MN), Rare Disease Caucus Co-Chair

Isabelle Lousada, Founder and CEO, Amyloidosis Research Consortium

Melissa Goetz, Co-President, Living with FCS

James Valentine, JD, MHS, Hyman, Phelps & McNamara, PC

Joanna Johnson, Board Member, Parent Project Muscular Dystrophy

KEYNOTES

SPEAKER PANEL

Thank you to our Presenting Sponsor

Additional support from

Share your experience during Rare Disease Week by taking a selfie in our Virtual Photobooth and posting it on social media with the hashtag #RareDC2021.

Thank you to our Virtual Photobooth Sponsor

#RareDC2021

Legislative Conference Agenda

Rare Disease Documentary Screening WEDNESDAY, JULY 14

THURSDAY, JULY 15

Presented by

Presented by:

1:00 P.M. – 5:00 P.M. ET

6:00 P.M – 7:30 P.M. ET

WELCOME

WELCOME RECEPTION

1:00 P.M. ET

6:00 P.M – 6:30 P.M. ET

• • • • • • • •

DOCUMENTARY SCREENING AND PANEL DISCUSSION

6:30 P.M – 7:30 P.M. ET

Welcome and 2021 Legislative Asks Welcome: Theresa Podrebarac, SVP of Clinical Development, Horizon Welcome and Moderator: Shannon von Felden, RDLA Program Director, EveryLife Foundation for Rare Diseases Jamie Sullivan, Director of Public Policy, EveryLife Foundation for Rare Diseases Dylan Simon, Newborn Screening and Diagnostics Policy Manager, EveryLife Foundation for Rare Diseases Sally Farrington Thompson, Global Health Policy Advisory, Office of Senator Roger Wicker Jay Eberle, Legislative Assistant, Office of Senator John Barrasso Caitlin Van Sant, Legislative Director, Office of Representative G. K. Butterfield

PREPARING FOR SUCCESSFUL HILL MEETINGS 2:00 P.M. ET

• •

Brenda Colmenares Moderator, EveryLife Foundation for Rare Diseases, Communications Manager

Sarah-Lloyd Stevenson, Director, Faegre Drinker Consulting Shannon von Felden, RDLA Program Director, EveryLife Foundation of Rare Diseases

DEEP POLICY DIVE #1—SPEEDING THERAPY ACCESS TODAY ACT 3:00 P.M. ET

• • • • •

GUEST PANEL

Moderator: Nicholas Manetto, Principal, Faegre Drinker Consulting Jamie Sullivan, Director of Public Policy, EveryLife Foundation for Rare Diseases Dan Whiting, Director, Community Impact, National Fragile X Foundation Paul Howard, Director of Public Policy, Amicus Therapeutics Caitlin Van Sant, Legislative Director, Office of Representative G. K. Butterfield

NETWORKING HAPPY HOUR

4:00 P.M. ET

Ben Strang

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Director, A Mystery to Me Documentary Film Series

• • • Glenn Phillips

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Featured in A Mystery to Me Documentary Film Series

#RareDC2021

Newborn Screening and Diagnostics Dylan Simon, Newborn Screening and Diagnostics Policy Manager, EveryLife Foundation for Rare Diseases Annie Kennedy, Chief of Policy and Advocacy, EveryLife Foundation for Rare Diseases State Advocacy Shannon von Felden, RDLA Program Director, EveryLife Foundation for Rare Diseases Swapna Kakani, RDLA State Advocacy Fellow, EveryLife Foundation for Rare Diseases Access State Policy Issues Jamie Sullivan, Director of Public Policy, EveryLife Foundation for Rare Diseases Rare Disease Advocacy Beyond COVID Lindsey Cundiff, Associate Director of Patient Engagement, EveryLife Foundation for Rare Diseases Abbey Hauser, Young Adult Representative of RDLA Rare Diversity: Increasing Advocacy in Underserved Communities Adrian Palau- Tejeda, Diversity Inclusion Advocacy Fellow, EveryLife Foundation for Rare Diseases #RareDC2021

Hill Day Issue Information

FRIDAY, JULY 16

1:00 P.M. – 4:45 P.M. ET

STATE NETWORKING BRUNCH: JOIN TO NETWORK WITH ADVOCATES IN YOUR STATE AND COORDINATE YOUR HILL MEETINGS 1:00 P.M. ET

Legislative Ask #1

DEEP POLICY DIVE #2—NEWBORN SCREENING SAVES LIVES REAUTHORIZATION ACT 2:00 P.M. ET

• • • •

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

Speaker Introduction: Kate Tighe, Vice President, US Public Affairs and Patient Advocacy—Rare Diseases, Sanofi Genzyme Moderator: Dylan Simon, Newborn Screening and Diagnostics Policy Manager, EveryLife Foundation for Rare Diseases Garrett Devenney, Health Policy Advisory, Senate Committee on Health, Education, Labor, and Pensions Dean Suhr, President and Co-founder, MLD Foundation

BREAKOUT SESSIONS: 3:00 P.M. ET DEEP POLICY DIVE #3—THE BETTER EMPOWERMENT NOW TO ENHANCE FRAMEWORK AND IMPROVE TREATMENTS (BENEFIT) ACT

• • • • YOUNG ADULTS ADVOCACY SESSION Yale, Young Adult Representatives of RDLA • Hannah • Grant Bonebrake, Young Adult Representatives of RDLA

Moderator: Annie Kennedy, Chief of Policy and Advocacy, EveryLife Foundation for Rare Diseases Ryan Fischer, Chief Advocacy Officer, Parent Project Muscular Dystrophy Sarah-Lloyd Stevenson, Director, Faegre Drinker Consulting Buddy Cassidy, PPMD Adult Advisory Committee

TALKING POINTS  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 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, 

HOT TOPICS IN RARE DISEASE POLICY 4:00 P.M. ET

• • • • •

Moderator: Sung Hee Choe, Senior Director, FasterCures Annie Kennedy, Chief of Policy and Advocacy, EveryLife Foundation for Rare Diseases Sloane Salzburg, Vice President, Council for Affordable Health Coverage John Richardson, Director, Policy and Government Relations, National Society of Genetic Counselors Saira Sultan, JD, President and CEO, Connect 4 Strategies and Policy Consultant for Haystack Project

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

 

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

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



Networking Lounge



Click here

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.

Thank you to our Networking Lounge sponsor

Download One Pager

#RareDC2021

Hill Day Issue Information

Hill Day Issue Information Legislative Ask #3

Legislative Ask #2

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

Cosponsor the Newborn Screening Saves Lives Reauthorization Act, S. 350/H.R. 482

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. 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 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.

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

KEY BILL PROVISIONS  

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

  

BACKGROUND  Federal newborn screening programs expired on September 30, 2019.  The House and Senate both introduced legislation that used the same language to re-authorize 



and build upon the current federal newborn screening program. The House passed the legislation on June 23rd and the Senate legislation is still pending.

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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 advocacy 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.

#RareDC2021

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Hill Day Issue Information

Legislative Ask #4

About the Rare Disease Congressional Caucus

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

TALKING POINTS

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

TALKING POINTS

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

 The Rare Disease Caucus is a bipartisan, bicameral caucus that works to raise awareness of rare diseases.

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 5,000 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.

     

ACCESS TO GENETIC COUNSELOR SERVICES ACT

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 Enact S. 1450/H.R. 2144 to update Medicare law to improve the lives of up to 60 million Americans,

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.

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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#RareDC2021

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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 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 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 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 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 IL-5 Jamie 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 Glenn Thompson PA-5 Rashida Tlaib MI-13 Paul Tonko NY-20 Lori Trahan MA-3

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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

SENATE

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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.

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Make a specific “ask”. Give Congress the solution. You don’t have to be an expert on legislation.

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

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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.

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Email the Congressional staffer with a one-pager on your asks as well as your contact information.

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Remember rare disease issues are nonpartisan. Don’t talk about politics in your meetings.

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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

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Virtual meetings with your legislators on Zoom can be just as productive and informative as in-person meetings.

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Start each meeting by thanking the Member/staffer for

Representatives

119 Democrats

Senators

1 Independent

63 Republicans

Thank you to our Resources Section Sponsor

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. Make it a group screenshot and do it quickly.

#RareDC2021

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 #RareDC2021 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 #RareDC2021 using the “@” symbol before the name. If you don’t know your legislator’s social media handle, check their official website.

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 @repgusbilirakis for #RareDC2021 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.

Share Your Rare Experience Follow us and share your #RareDC2021 pictures for a chance to win a travel stipend for Rare Disease Week 2022. Tip: Be Creative! @RareAdvocates

#RareDC2021

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:

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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 #RareDC2021”.

Thank you to our Resources Section Sponsor

#RareDC2021

THANK YOU FOR

YEARS OF ADVOCACY!

Glossary Congressional Terms

Glossary

Thank you to our Glossary Sponsor

Congressional Terms

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.

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

Majority Party – The political party having electoral strength sufficient to permit it to win control of a government.

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.

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”.

Nonpartisan – Not associated with a single political party or caucus. 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. 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. 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). 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. Click here to download full glossary terms

Lobbyist – A person who attempts to influence legislation on behalf of a specific interest group.

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Petition – A formal written request submitted by anyone other than the legislature (individuals, boards, commissions, cities, etc.). 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. Recess – The temporary break of a daily session. Can refer to breaks within the same day, overnight, or longer breaks (such as over holidays). 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. Sponsor – A member of the legislature who submits a bill or other measure for consideration. Other interested members can join as cosponsors. 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.

Click here to download full glossary terms

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Glossary

U.S. Legislative Process

Health-Related Federal Government Agencies 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)

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)

Administers healthcare/reimbursement programs including Medicare, Medicaid, and the Children’s Health Insurance Program (CHIP).

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.

U.S. Legislative Process

DEPARTMENT OF EDUCATION The department of the U.S federal government that administers federal programs related to education.

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.

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).

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 products that emit radiation.

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)

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

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The Foundation’s Community Congress program is a strategic advisory council that brings patient organizations and industry together to provide advice and insight on urgent policy issues.

Community Congress Working Groups and Efforts Public Policy

Focused on closing the innovation gap for the 93% - 95% of rare diseases that have no FDA-approved treatment and ensuring patients receive early access to treatment opportunities. STAT Act, disseminating and following up on the Economic Burden of Rare Disease Study, rare disease appropriations priorities

JOIN COMMUNITY CONGRESS

Regulatory Focused on improving the regulatory process and advancing regulatory science for rare disease therapies. PDUFA and MDUFA engagement, 21st Century Cures 2.0 input, advancing potential of digital health tools Roundtable Series

Newborn Screening and Diagnostics

Focused on ensuring patients receive earliest access to lifesaving diagnostic opportunities through newborn screening. State RUSP Alignment Legislation, Advancing the Newborn Screening Saves Lives Act and legislation to improve coverage for and study the benefits of DNA sequencing services in Medicaid

Access Focused on accelerating patients’ access to approved therapies. ICD Code Roadmap Project, 6 Access Priority Subgroups, Accelerated Approval Challenges

Ad-Hoc COVID-19 Focused on ensuring that the needs and experiences of the rare disease community are heard in relation to healthcare decisions made during the current pandemic. Advocating for rare disease patients’ needs in the COVID vaccine roll out, addressing vaccine efficacy for immunocompromised populations and monitoring rollback of COVID related flexibilities like telehealth

ONE RELENTLESS ILLNESS. THREE UNSTOPPABLE PEOPLE.

A MYSTERY TO ME A MYASTHENIA GRAVIS DOCUSERIES

Membership is free for qualified patient organizations. For more information please contact Jack Meloro at jmeloro@everylifefoundation.org

For all other organizations (industry, consultants, health professional associations) please contact Elissa Taylor at etaylor@everylifefoundation.org

JOINCOMMUNITYCONGRESS.ORG

We’re kicking off Rare Disease Week on Capitol Hill 2021 with a special virtual screening of A Mystery to Me. • A Mystery to Me is the first-ever documentary film series about life with myasthenia gravis (MG), a rare autoimmune disease that causes profound muscle weakness. • One of the short films from the series will be screened online, followed by a panel discussion with the director and one of the patients featured in the docuseries.

VIRTUAL SCREENING: July 14th 6 p.m E.T.

US-NON-21-00203 V1 06/2021

• To stream all three films and get a behind-the-scenes look at production, visit mg-united.com/a-mystery-to-me.

A proud sponsor of Rare Disease Week on Capitol Hill 2021

www.vrtx.com

www.janssen.com

Horizon Therapeutics is proud to support the 2021 Rare Disease Week on Capitol Hill. Horizon is focused on researching, developing and commercializing medicines that address critical needs for people impacted by rare, autoimmune and severe inflammatory 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. horizontherapeutics.com

Together we are delivering Together we are working to transform the treatment of rare diseases that were previously thought to be untreatable. For patients and families seeking information about rare diseases, gene therapy or clinical trials, educational resources from trusted organizations can provide a better understanding and help support discussions with your clinical team. Visit helpful resources for the rare disease community. © 2021 Novartis Gene Therapies, Inc. US-UNB-21-0051 06/2021

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 2021. travere.com | @TravereRare | #InRareForLife

A proud sponsor of the Fast Forward for RARE Advocate Training Webinar

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)

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

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.

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.

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 #RareDC2021

Rare Disease Week 2021 Program Updated

Articles inside

Hill Day Issue Information

Documentary Screening

Congressional Caucus Briefing

Legislative Conference Agenda