RDW 2024 Program Booklet

Presented by

FEBRUARY 25 - 28

To Our Generous 2024 Rare Disease Week on Capitol Hill Sponsors

Table of Contents

Leadership

Ranking

This

Presidential Champion

3 Welcome Letter

4 Board of Directors

5-6 EveryLife Team

7-8 Schedule of Events

Sunday

9 Documentary Screening & Reception

Monday

10-13 Legislative Conference Agenda

Wednesday

Advocate

14 Rare Disease DEIA Discussion & Congressional Caucus Briefing

15 Rare Artist Reception

16 Notes

Legislative Asks & Tips

17 Ask #1: Interagency Coordinating Committee on Rare Disease

18 Ask #2: Accelerating Kids’ Access to Care Act

19 Ask #3: Creating Hope Reauthorization Act

20 Ask #4: Safe Step Act

21 Ask #5: Ask your Member of Congress to Join the Caucus

22

Welcome

to Rare Disease Week on Capitol Hill hosted by the EveryLife Foundation for Rare Diseases.

Unified. Amplified. Stronger than ever.

This week marks the 15th anniversary of the EveryLife Foundation and the 13th year of Rare Disease Week on Capitol Hill.

15 years of impact. 15 years of landscape-changing policy. 15 years of ensuring the roar of the rare community is heard. 15 years of life-changing innovation. This progress is only possible thanks to your dedication and passion for rare disease advocacy.

And we are just getting started. Our community’s needs are greater than ever.

Together, we must ensure that the promise of today’s innovations and development pipelines are transformed into life-changing diagnostics and therapies that can be accessed by all who need them.

We are thrilled to welcome attendees from all 50 states, Washington, D.C., and Puerto Rico. We recognize and appreciate the sacrifice and effort required to join us in Washington this week. Your stories and influence as rare disease ambassadors hold immense importance this year. Rare diseases pose a significant public health crisis, with the cost reaching a staggering $1 trillion dollars in 2019. Prioritizing and accelerating access to treatments and timely diagnoses is crucial in saving lives and mitigating this crisis.

As we celebrate the 15th anniversary of the EveryLife Foundation for Rare Diseases, we reflect on the incredible progress made possible by your unwavering dedication and advocacy. Together, we form an extraordinary team united in our mission to drive positive change for the rare disease community, and we have so much important work ahead of us.

As we embark on this week of advocacy and collaboration, let us recognize the power of our collective efforts. On behalf of the EveryLife Team, our volunteers, and partners, we wish you an exceptional and impactful week in Washington, DC.

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 the 30 million Americans living with a rare disease. With decades of experience in the government, nonprofit, finance, science, medicine, industry, and academic sectors, EveryLife board members provide valuable guidance to the Foundation toward achieving its mission. In addition, 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.

Jennifer

Cristina Casanova Might, BS, MBA CEO, Welcomed Co. Patient and Parent Advocate

Merrill Friedman Regional Vice President, Inclusive Policy & Advocacy

Lisa Carlton, PhD Lisa Carlton Consulting Parent Advocate

Abbey Hauser Young Adult Rare Disease Patient Advocate

Frank Sasinowski, MS, MPH, JD - Vice Chair Director, Hyman, Phelps & McNamara P.C. Patient and Parent Advocate

Vicki Seyfert-Margolis, PhD Chair Founder and CEO, Respond Health Family Advocate

Bernstein Secretary Executive Vice President, Horizon Government Affairs Parent Advocate

Stephen C. Groft Treasurer Pharm.D

EveryLife Foundation for Rare Diseases Team

Communications & Marketing

Policy, Advocacy & Patient Engagement

Development

Operations

The EveryLife Foundation’s team works every day to improve the lives of the 30 million Americans living with one of 10,000 known rare diseases. We do this by educating and activating the patient community to ensure they are heard by policy makers in government and the healthcare industry.

Ted Brasfield Vice President of Corporate Giving

Stephanie Siddiqi Associate Director of Development

Collin Sovie Alliance Development Manager

Brenda Colmenares Associate Director of Communications

Margo Metzger Senior Director of Marketing and Public Relations

Lindsey Cundiff Associate Director of Policy Programs

Shannon von Felden Senior Director of Advocacy

Courtney Felle Young Adult Rare Representatives Program Manager

Kendly Jones State Advocacy Manager

Chrissy Lanham Event Coordinator

Annie Kennedy Chief of Policy, Advocacy & Patient Engagement

Katelyn Laws Associate Director of RDLA

Baillie McGowan Associate Director of Policy & Research

Priscilla Rodriguez Diversity Inclusion Advocacy Manager

Dylan Simon Director of Policy

Emily Stauffer State Policy Manager Jamie Sullivan Vice President of Policy

Lina Arslanian Associate Director of Finance & Salesforce

Megan Pinegar Chief Operating Officer

Alyssa Terwall Senior Director of Operations & Events

Stephanie Riordan Director of Patient Programs

Mariela Romero Executive Assistant

Thank you to the Presidential Sponsor of Rare Disease Week on Capitol Hill

Schedule of Events

SUN 25 MON

26

• Rare Disease Documentary Screening and Reception

5:15 p.m. - 8:30 p.m. EST

Ronald Reagan Building and International Trade Center

1300 Pennsylvania Ave, NW, Concourse Level, Washington DC, 20004

Metro Stop: Metro Center and Federal Triangle

• Legislative Conference

9:00 A.M. - 5:00 P.M. EST

Ronald Reagan Building and International Trade Center

Metro Stop: Metro Center and Federal Triangle

Young Adult Rare Representatives (YARR) Meetup

5:00 P.M. - 6:30 P.M. EST

Ronald Reagan Building and International Trade Center

Presented by:

Presented by:

1300 Pennsylvania Ave, NW, Concourse Level, Washington DC, 20004

Presented by:

TUE 27 WED 28

• Meetings with Members of Congress

10:00 a.m. - 5:00 p.m. EST

Capitol Hill, Washington DC, 20004

Metro Stop: Union Station (Senate side) or Capitol South (House side)

• Rare Disease Week 2024 DEIA Discussions

10:00 a.m. - 12:00 p.m. EST

Capitol Hill, Cannon House Office Building, Room 390

Metro Stop: Union Station (Senate side) or Capitol South (House side)

• Rare Disease Congressional Caucus Briefing

1:00 p.m. - 2:30 p.m. EST

Capitol Hill, Cannon House Office Building, Room 390

Metro Stop: Union Station (Senate side) or Capitol South (House side)

• Group Photo on Capitol Hill Steps

3:00 p.m. EST

Capitol Hill, Washington D.C 20004

East Steps

Energy Guide Key

Physical

Low: Little movement, ample seating available

Medium: Prolonged standing, some walking and some seating

Mental

Low: Optional participation, minimal to no participation

Medium: Might require participation, note taking or cognitive effort

High: Ample walking and standing, seating not guaranteed

High: Requires participation, note taking, and/or cognitive effort

Metro Stop: Union Station (Senate side) or Capitol South (House side)

• Rare Artist Reception

3:45 p.m. - 5:45 p.m. EST

Russell Senate Office Building, Kennedy Caucus Room (SR-325)

2 Constitution Ave NE, Washington, DC 20002

Metro Stop: Hill Center is two blocks east of the Eastern Market Metro stop on the Blue, Orange, and Silver lines.

Presented by:

Presented by:

Presented by:

Rare Disease

Documentary Screening

• Sunday, February 25

5:15 p.m. - 8:30 p.m. EST

Ronald Reagan Building and International Trade Center 1300 Pennsylvania Ave, NW, Concourse Level Washington DC, 20004

Chris Bombardier has never let severe hemophilia stop him from climbing some of the world’s tallest mountains. In 2017, Chris partnered with hemophiliac filmmaker Patrick James Lynch and his award-winning production team to film his journey through Nepal to summit the world’s tallest peak, Mount Everest. Bombardier Blood, highlights the physical, psychological and emotional struggle of acclimating to the mountain and preparing for the greatest challenge of Chris’ life.

• 5:15 p.m. - 6:30 p.m. EST: Reception

• 6:30 p.m. - 6:45 p.m. EST: Opening Remarks

• 6:45 p.m. - 8:05 p.m. EST: Documentary Screening

• 8:05 p.m. - 8:30 p.m. EST: Panel Discussion

Introduction Sponsor Remarks

Founder and CEO, Respond Health

Alison Clifford Director, Therapeutic Policy & Advocacy, Takeda Pharmaceuticals America, Inc.

Patrick James Lynch

Filmmaker and CEO, Believe Limited

Presented by:

Discussion Panel Featuring:

Chris Bombardier Executive Director, Save One Life

Jessica Bombardier

Scientist and Rare Disease Advocate

Legislative Conference Agenda

• Monday, February 26

Ronald Reagan Building and International Trade Center 1300 Pennsylvania Ave, NW, Concourse Level Washington DC, 20004

8:00 - 9:00 a.m. Registration and Breakfast

Atrium

9:00 - 9:45 a.m. Welcome

Atrium Hall and via Livestream

Annie Kennedy, EveryLife Foundation for Rare Diseases

Brett McReynolds, Amgen

Dylan Simon Director of Policy, EveryLife Foundation for Rare Diseases

Connie Montgomery

Patient and Family Advisor, PFCC Partners

Presented by:

Registration Presented by: Breakfast Presented by:

Frank Sasinowski, Hyman, Phelps, & McNamara and Vice Chair, EveryLife Foundation for Rare Diseases

Keynote

Abbey Hauser, Advocacy Chair of Rare Disease Week on Capitol Hill, EveryLife Foundation for Rare Diseases

Logistics Overview

Shannon von Felden, EveryLife Foundation for Rare Diseases

9:45 - 10:15 a.m. Legislative Outlook

Atrium Hall and via Livestream

Hear from experts on the 118th Congress and how Congress will impact healthcare policy in 2024.

Moderator: Ryan Shay, Faegre Drinker

Josh Trent, Leavitt Partners

Caitlin Van Sant, Mehlman Consulting

10:15 - 10:30 am Break

Family Space

Ballroom A is available for social distancing with livestream

Presented by:

Ronald Reagan Building, Oculus, Ground Level. The Family Space will be available for families all day except from 1:45-2:45, 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. Thank you to our Family Space sponsor, Biogen.

Vicki Seyfert-Margolis, PhD Chair, EveryLife Foundation for Rare Diseases

Legislative Conference Agenda

• Monday, February 26 - Continued

10:30 - 11:30 a.m. Deep Dive Policy Ask #1:

Interagency Coordinating Committee on Rare Disease

Atrium Hall and via Livestream

Moderator: Annie Kennedy, EveryLife Foundation for Rare Diseases

Matthew Ellinwood, Ph.D., National MPS Society

Stephen Groft, PharmD, NIH/NCATS, EveryLife Foundation Board of Directors

Marsha Henderson, Foundation for Sarcoidosis Research

Nisha Quasba, Faegre Drinker

11:30 - 12:00 p.m. Deep Dive Policy Ask #2:

Accelerating Kids’ Access to Care Act

Atrium Hall and via Livestream

Moderator: Paloma Juarez, RDLA Advisory Committee

Nicholas Manetto, Faegre Drinker

Kyle Underwood, RDLA Advisory Committee

12:05 pm Group Photo

Atrium Stairs

12:15 - 1:15 pm Networking Lunch

Atrium

Lunch

Presented by:

1:15 - 1:45 pm Deep Dive Policy Ask #3: Pediatric Priority Review

Voucher (PRV) Reauthorization

Atrium Hall and via Livestream

Moderator: Jamie Sullivan, EveryLife Foundation for Rare Diseases

Chris Jones, Office of Congressman Gus Bilirakis

Melissa Kennedy, International Rett Syndrome Foundation

1:45 - 2:45 pm Preparing for Successful Meetings*

Atrium Hall, Ballroom A & B, and via Livestream

*Mandatory for advocates participating in Hill Day (family room will be closed at this time). Advocates receive information on their Hill meeting schedule and their meetings and strategize with their teams to make the most of their meetings on Capitol Hill.

Katelyn Laws, EveryLife Foundation for Rare Diseases

Accessibility and Inclusion

Closed captioning, Spanish translation materials and important event updates via text are possible thanks to our Accessibility and Inclusion sponsor, Travere Therapeutics.

Reagan WiFi Network Access

Legislative Conference Agenda

Network Name: Ronald Reagan Building

WiFi Username: RareDC2024

Password: RDLA

2:45 - 3:15 pm Snack Break

Atrium

3:15 - 4:00 p.m. Breakout Sessions

Track 1:

Deep Dive Policy Ask #4: Safe Step Act

Atrium Hall and via Livestream

Moderator: Nisha Trivedi, RDLA Advisory Committee

Sarah Buchanan, National Psoriasis Foundation

Dylan Simon, EveryLife Foundation for Rare Diseases

Anita Wilkerson, HAE advocate

Track 2:

Advocacy for Young Adults

Atrium Ballroom B

This session is for young adults (ages 16-30) to learn more about the Young Adult Rare Representatives (YARR) community legislative ask.

Moderator: Courtney Felle, EveryLife Foundation for Rare Diseases

Rachelle Cook, YARR Member

Sophie Melancon, YARR Member

Legislative Conference Agenda

• Monday, February 26 - Continued

4:05 - 4:50 p.m. Breakout Sessions

Track 1:

Emerging Policy Issues for the Rare Disease Community

Atrium Hall and via Livestream

This session will share updates and information on policy issues impacting rare disease including the PROTECT Rare Act and newborn screening and how rare disease advocates can become involved.

Introductions: Duane Clark, Sanofi

Moderator: Jennifer Bernstein, Horizon Government Affairs

Amy Gaviglio, MS, CGC, Genetics and Public Health Consultant

Sarah Gilbert, Office of Representative

Neal Dunn

Brian Fahey, Office of Representative

Brett Guthrie

Track 2:

Practice Your Pitch

Atrium Ballroom B

*This is a must-attend session for those individuals who are new to advocacy. Learn how to develop and make your legislative ask during your meetings. The session will offer new advocates an opportunity to practice making their ask and telling their story with other advocates. This is a great opportunity to get questions answered by experienced rare disease advocates.

Moderator: Stephanie Riordan, EveryLife Foundation for Rare Diseases

4:50 - 5:00 p.m. Closing Remarks

Atrium Hall and Atrium Ballroom B

*Sessions and speakers are subject to change.

Rare Disease DEIA Discussion & Congressional Caucus Briefing

• Wednesday, February 28

Rare Disease Week 2024 DEIA discussion

10:00 a.m. - 12:00 p.m. EST

Capitol Hill, Cannon House Office Building, Room 390

Session 1: Access & Utilization of Healthcare Services

Access to Genetic and Genomic Testing in Hispanic/Latinx Communities, Amanda de Leon, Providence St. Jude Medical Center

Prior Authorization and Other Utilization Management Practice Challenges in Rural Communities, Michael Ward, Alliance for Aging Research

The Intersectionality of LGBTQIA+ and Rare Disease, William Romero, Disability Advocate

Session 2: Cell and Gene Therapy Disparities:

Development, Approval and Access, Ashley Valentine, Sick Cells

• Wednesday, February 28

Rare Disease Congressional Caucus Briefing

1:00 p.m. - 2:30 p.m. EST

Capitol Hill, Cannon House Office Building, Room 390

The Rare Disease Community & Congress –Changing the Status Quo

Rare Disease Legislative Advocates and the Rare Disease Congressional Caucus invite you to a rare disease briefing.

Guest Panel

Moderator: Ryan Fischer, Foundation for Angelman Syndrome Therapeutics

Introductions: Mary-Lacey Reuther, Head of North America Policy, Advocacy Government Affairs for CSL Behring

Presented by:

Presented by:

Additional support from:

Panelists:

Brigid Brennan, Friedreich’s Ataxia Research Alliance

Billy Cline, Rare Disease Patient Community Representative

Brian Denger, Parent Project Muscular Dystrophy

Hill Day Issue Information

Legislative Ask #1

Support the Creation of an Interagency Coordinating Committee for Rare Diseases

Background

Agencies across the federal government have long implemented policies and programs to support rare disease patients and their families. The expansion of newborn screening to include rare diseases, the FDA’s Accelerating Rare Disease Cures Program, and the recently announced CMS Cell and Gene Therapy Access demonstration project are all examples of federal initiatives that focus on the rare disease community. Beyond specific initiatives like these, federal agencies provide services, make policies, and prioritize resources on a host of issues that are relevant to the health and quality of life for people living with rare diseases. Considering the expansive scope of federal initiatives and the substantial unmet needs within the rare disease community, it is imperative to establish a mechanism that fosters optimal coordination and collaboration and ensures resources are efficiently utilized.

Interagency Coordinating Committee for Rare Diseases

• Interagency committees elevate the awareness of the associated issues, provide forums for collaboration across agencies and even departments and ensure effective and efficient resource utilization. Interagency coordinating committees also provide invaluable opportunities to engage non-governmental stakeholders.

• HHS can use its authorities under the Federal Advisory Committee Act to establish an interagency coordinating committee for rare diseases. The committee should include leaders from agencies within HHS including the National Institutes of Health (NIH), especially the National Center for Advancing Translational Sciences (NCATS), the Food and Drug Administration (FDA) and relevant offices and centers, the Centers for Medicare & Medicaid Services (CMS), Centers for Disease Control and Prevention (CDC), the HHS office of the Secretary, and many more. Departments and agencies outside of HHS, such as the Department of Education, the Social Security Administration, the Department of Veterans Affairs, and the Department of Defense’s medical research function, serve vital roles that affect rare disease patients and should be included as well.

• An interagency coordinating committee must include a role for external rare disease stakeholders including rare disease patient community representatives, clinicians, researchers, and social service providers who can share their experiences and expertise.

• The Committee should meet periodically, undertake an assessment of rare disease initiatives and services that acutely impact Americans living with rare diseases, identify opportunities for improvements in existing efforts and community needs yet to be met in the existing efforts and provide regular reports with outcomes to assess progress.

Hill Day Issue Information

Legislative Ask #2

Cosponsor the Accelerating Kids’ Access to Care Act (AKACA), H.R. 4758 / S. 2372

Talking Points

✓ Many children live in states where specialized care is not available locally. Even some states with leading medical centers lack health providers with expertise in some rare conditions.

✓ Children with complex medical needs like cancer often must travel outside their states to receive treatment.

✓ More than half of children in the U.S. rely on Medicaid and the Children’s Health Insurance Program (CHIP) for their health insurance coverage. But bureaucratic rules in these programs often limit kids’ ability to get timely treatment outside their home state.

✓ This process needlessly results in delays in treatment that can cause a child’s condition to worsen.

Accelerating Kids’ Access to Care Act (AKACA)

• Enact H.R. 4758 / S. 2372 to allow pediatric providers to enroll more efficiently in multiple state Medicaid programs for a five-year period, positioning them to provide essential, time-sensitive care to children who need it.

• The Accelerating Kids’ Access to Care Act will ensure better, faster healthcare for kids who need it most by reducing the burden on the doctors who treat them.

Hill Day Issue Information

Legislative Ask #3 Cosponsor the Creating Hope Reauthorization Act (H.R. 7384)

Talking Points

✓ Developing drugs for rare pediatric diseases is challenging due to the small populations affected, difficulties associated with conducting clinical trials for children, delays in diagnosis and more.

✓ About 70% of rare diseases are exclusively pediatric onset and overall, 95% of rare diseases have no approved treatments.

✓ The Creating Hope Act expanded the Rare Pediatric Disease Priority Review Voucher (PRV) program to include drugs that treat rare pediatric diseases as part of the Food and Drug Administration Safety and Innovation Act (FDASIA) in 2012.

✓ To be eligible for a PRV, the treatment must obtain Rare Pediatric Designation from the FDA, be eligible for priority review, and it must be the first approval for the drug’s active ingredient.

✓ After an eligible treatment is approved by the FDA, the company is issued a PRV. The opportunity to obtain a PRV is an important incentive in pediatric rare disease therapy development because:

• Earlier Review: a company who gets a PRV can use it on a future treatment that wouldn’t otherwise qualify for priority review, leading to about a 4-month reduction in review time.

• Revenue Generation: some companies chose to sell the PRV to another company, generating revenue for the seller that is often used to continue and expand their rare disease research and development programs.

✓ The program was last reauthorized in 2020 for a 4-year period. Without Congressional action, companies will have to receive a Rare Pediatric Designation by September 30, 2024, to be eligible. After September 30, 2026, FDA will no longer be able to award Rare Pediatric PRVs.

The Creating Hope Reauthorization Act

• H.R. 7384 will reauthorize the Rare Pediatric Disease Priority Review Voucher (PRV) program for four years, ensuring this vital incentive that has no cost to taxpayers can continue to spur innovation in rare diseases that disproportionately affect children.

Scan

Hill Day Issue Information

Talking Points

✓ A medication step therapy protocol establishes a specific sequence in which a group health plan or a health insurance issuer covers prescription drugs.

✓ Step therapy protocols may require patients to try and fail an insurer preferred medication before being covered by the physician-prescribed medication.

✓ Many insurers have instituted this practice to help control the costs of expensive medications. However, while this practice may initially reduce insurer costs, it can have devastating health consequences for patients and ultimately lead to more expensive health care costs in the long run.

✓ Patients who are denied first coverage of medications recommended by their physicians can end up with poor health outcomes due to adverse health events, which can lead to costly hospitalizations.

Safe Step Act

• Amends the Employee Retirement Income Security Act of 1974 (ERISA) to require a group health plan to establish an exception process to medication step therapy protocol:

• When the treatment is contraindicated;

• Expected to be ineffective;

• Likely to cause an adverse reaction;

• Expected to decrease the individual’s ability to perform daily activities or occupational responsibilities; or

• The individual is stable based on the prescription drugs already selected.

• This legislation would require that requests be granted in a timely manner — within 72 hours after receipt of the request or 24 hours when the protocol jeopardizes the life or health of the individual.

Hill Day Issue Information

Legislative Ask #5

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.

✓ More than 95% of the estimated 10,000 rare diseases do not have an FDA approved treatment.

✓ Rare or orphan diseases are defined as diseases affecting fewer than 200,000 people in the U.S.

✓ 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.

Co-Sponsors

Rare Disease Congressional Caucus Member List

House

• Mark Amodei NV-2

• Jake Auchincloss MA-4

• Don Bacon, NE-2

• Andy Barr KY-6

• Joyce Beatty OH-3

• Ami Bera CA-7

• Donald Beyer, Jr. VA-8

• Gus Bilirakis* FL-12

• Sanford Bishop, Jr. GA-2

• Lisa Blunt Rochester DE

• Suzanne Bonamici OR-1

• Julia Brownley CA-26

• Vern Buchanan FL-16

• Michael Burgess TX-26

• Ken Calvert CA-42

• Salud Carbajal CA-24

• Tony Cardenes CA-29

• Andre Carson IN-7

• John Carter TX-31

• Sean Casten IL-6

• Kathy Castor FL-14

• Yadira Caraveo, M.D CO-8

• Judy Chu CA-27

• Emanuel Cleaver, II MO-5

• Ben Cline VA-6

• Steve Cohen TN-9

• Jenniffer Gonzalez-Colon PR-AL

• James Comer KY-1

• Gerald Connolly VA-11

• Jason Crow CO-6

• Sharice Davids KS-3

• Diana DeGette CO-1

• Rosa DeLauro CT-3

• Suzan DelBene WA-1

• Mark DeSaulnier CA-10

• Debbie Dingell MI-12

• Lloyd Doggett TX-37

• Tom Emmer MN-6

• Anna Eshoo CA-18

• Brian Fitzpatrick PA-8

• Lizzie Fletcher TX-7

• Bill Foster IL-11

• Ruben Gallego AZ-7

• John Garamendi CA-3

• Josh Gottheimer N-J5

• Garret Graves LA-6

• Raul Grijalva AZ-3

• Glenn Grothman WI-6

• Brett Guthrie KY-2

• Kevin Hern OK-1

• Jim Himes CT-4

• Richard Hudson NC-8

• Jared Huffman CA-2

• Hank Johnson GA-4

• Dusty Johnson SD-AL

• David P. Joyce OH-14

• Marcy Kaptur OH-9

• Tom Kean NJ-7

• Ro Khanna CA-17

• Derek Kilmer WA-6

• Andy Kim NJ-3

• Raja Krishnamoorthi IL-8

• Darin LaHood IL-18

• John Larson CT-1

• Bob Latta OH-5

• Susie Lee NV-3

• Teresa Leger Fernandez NM-3

• Debbie Lesko AZ-8

• Mike Levin CA-49

• Ted Lieu CA-33

• Zoe Lofgren CA-19

• Blaine Luetkemeyer MO-3

• Stephen Lynch MA-8

• Nancy Mace SC-1

• Nicole Malliotakis NY-11

• Kathy Manning NC-6

• Brian Mast FL-18

• Doris Matsui* CA-6

• Michael McCaul TX-10

• Jim McGovern MA-2

• Cathy McMorris Rodgers WA-5

• Grace Meng NY-6

• Mariannette Miller-Meeks IA-2

• Barry Moore AL-2

• Seth Moulton MA-6

• Kevin Mullin CA-15

• Richard Neal MA-1

• Joe Neguse CO-2

• Donald Norcross NJ-1

• Ralph Norman SC-5

• Eleanor Holmes Norton DC

• 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

• Dean Phillips MN-3

• Chellie Pingree ME-1

• Katie Porter CA-45

• Bill Posey FL-8

• Mike Quigley IL-5

• Jamie Raskin MD-8

• Deborah Ross NC-2

• David Rouzer NC-7

• C.A. Dutch Ruppersberger MD-2

• John Rutherford FL-4

• Maria Elvira Salazar FL-27

• Mary Gay Scanlon PA-5

• Jan Schakowsky IL-9

• Brad Schneider IL-10

• David Scott GA13

• Mikie Sherrill NJ-11

• Mike Simpson ID-2

• Elissa Slotkin MI-8

• Adam Smith WA-9

• Chris Smith NJ-4

• Jason Smith MO-8

• Lloyd Smucker PA-16

• Darren Soto FL-9

• Abigail Spanberger VA-7

• Melanie Stansbury NM-1

• Haley Stevens MI-11

• Marilyn Strickland WA-10

• Eric Swalwell CA-15

• Glenn Thompson PA-5

• Rashida Tlaib MI-13

• Paul Tonko NY-20

• Lori Trahan MA-3

• Jeff Van Drew NJ-2

• Juan Vargas CA-51

Caucus: 171 Members

• Nydia Velazquez NY-7

• Ann Wagner MO-2

• Debbie WassermanSchultz 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

Senate

• 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

• Charles Grassley IA

• 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

• Markwayne Mullin OK

• Alex Padilla CA

• Gary Peters MI

• James Risch ID

• Kyrsten Sinema AZ

• Jeanne Shaheen NH

• Tina Smith MN

• Debbie Stabenow MI

• Chris Van Hollen MD

• Raphael Warnock GA

• Roger Wicker* MS

House: 144 Members Senate: 27 Members

Caucus Co-Chairs*: Sen. Amy Klobuchar (MN), Sen. Roger Wicker (MS), Rep. Gus Bilirakis (FL), and Rep. Doris Matsui (CA)

Sen. Amy Klobuchar (MN) Sen. Roger Wicker (MS)

Rep. Gus Bilirakis (FL) Rep. Doris Matsui (CA)

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.

• 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 partisan 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.

RDLA Congressional Scorecard

Tip: Encourage your legislator to join the Rare Disease Caucus and cosponsor rare disease legislation to improve their score. Scan to view your state specific scorecard. 

Social Media Advocacy Tips

• How does a “#” work?

On Facebook, Instagram, LinkedIn, and X (formerly Twitter) 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 #RareDC2024 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 them in your post about #RareDC2024 using the “@” symbol before the name. If you don’t know your legislator’s social media handle, check his or her official website.

Tip: Look for a blue checkmark on the Instagram or X account to make sure the account’s identity has been verified.

• Before your meeting:

Create a post tagging the Member’s office and the issue you will be talking about. Example post shown at right.  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:

Ask to take a photo, preferably towards the end of the meeting. Write down any notes that might make for good tweets or quotes for your social post.

• After the meeting:

Post your picture with a thank you note on Facebook, Instagram, LinkedIn, or X to re-emphasize the ask or any key points you discussed during the meeting. Example post shown at right. 

Share Your #RareDC2024 Experience

Follow us and share your Rare Disease Week experience for a chance to win a $100 Gift Card of your choice.

Tip: Be Creative!

@RareAdvocates #RareDC2024

“We are excited to meet with @RepGusBilirakis for #RareDC2024 to talk about ways to bring more treatments to #RareDisease patients.”

Thank you @RepGusBilirakis for joining the Rare Disease Congressional Caucus and supporting #RareDisease legislation! #RareDC2024.

Glossary Congressional Terms

Scan to access full glossary

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

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

• Bill Sponsor: A Representative or Senator who introduces a bill.

• Bill Cosponsor: 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.

• 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 Congressional Caucus).

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

• Legislator: An elected official of a legislative body.

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

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

• Nonpartisan: Not associated with a single political party or caucus.

• Partisan: Associated with a single political party or caucus.

Glossary Government Agencies

• 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).

• Centers for Disease Control and Prevention (CDC)

Tasked with protecting the nation from health, safety and security threats, both foreign and domestic. Monitors reported disease and maintains information databases on prevalence, region, and other factors.

• Centers for Medicare and Medicaid Services (CMS)

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

• Food and Drug Administration (FDA)

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)

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.

U.S. Legislative Process

Accessibility Resources

General Travel Links

• Visit the Capitol

www.visitthecapitol.gov/plan-visit/visitors-disabilities

• Wheelchair Travel

www.wheelchairtravel.org/washington-dc/

• Washington.org

www.washington.org/dc-information/washington-dcdisability-information

Capitol Shuttle

For your convenience, the Capitol Visitor Center provides an on-demand shuttle service for individuals who use wheelchairs or who need mobility assistance. The shuttles run from the bus drop-off and pick-up areas on the West side of the Capitol to the Capitol Visitor Center entrance at the center of the Capitol’s East Plaza. Shuttles operate continuously, as needed, from 8:30am – 4:30pm, Monday – Saturday.

Transportation Options in DC

• Metro (Subway)

https://www.wmata.com/service/accessibility/ #main-content

A list of out-of-service elevators are available at the information booth of every metro station. Check the WMATA website or call 202-637-7000 for outages before leaving.

• UberWAV

https://www.uber.com/us/en/ride/uberwav/

• Lyft Access

https://help.lyft.com/hc/en-us/articles/ 115013081668-Accessible-vehicle-dispatch

Medications

• Cannabis on Federal Property

https://mpdc.dc.gov/marijuana

https://www.acludc.org/en/know-your-rights/know-yourrights-marijuana-laws-district-columbia

Wheelchair Accessible Van Rentals and Sales

• Mobility Works – Service (877) 275-4912 Rentals (877) 275-4915

• Accessible Vehicles – (301) 838-9700 119 Taft Street, Rockville, MD

For easy access to links scan here

Scooters and Wheelchair Rentals

• Scootaround – (888) 441-7575

Scooter and wheelchair rentals are available daily, weekly, or for longer periods of time. Take a tour of DC and the National Mall on a mobility scooter.

• Bike and Roll – (202) 842-BIKE

Electric scooters and manual wheelchairs 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 residents

Rare Disease Week Event Buildings, Entrances and Services on Capitol Hill

To enter the Capitol and the office buildings, all visitors are screened by a magnetometer and all personal items are screened by x-ray. Some items are prohibited from entering the Capitol such as liquid, including water. Capitol Police can make exceptions if a prohibited item is determined to be necessary for childcare, medical, or other special needs.

• For more information, go to:

https://www.visitthecapitol.gov/visit/know-before-yougo/prohibited-items

• For questions on accessibility contact the Office of Congressional Accessibility Services (OCAS) on Capitol Hill – (202) 224-4048

https://www.aoc.gov/accessibility-services

House Office Buildings Accessible Entrances:

• Cannon House Office Building: Entrance on New Jersey Avenue, SE, south of the terrace at the intersection with Independence Avenue.

• Rayburn House Office Building: Main entrance, horseshoe drive off South Capitol Street.

• Longworth House Office Building: Main entrance, Independence, and New Jersey Avenues.

Senate Office Buildings Accessible Entrances

• Dirksen Senate Office Building: First Street and C Street entrance.

• Russell Senate Office Building: Delaware entrance on ground level closest to Constitution Avenue.

• Hart Senate Office Building: Second Street entrance.

Resources Section

Presented by:

Capitol Hill Resources

Food Service Options on Capitol Hill buildings

• Longworth Cafeteria - B223

Basement level of Longworth House Office Bldg.

• Rayburn Cafeteria - 2063

Ground level of Rayburn House Office Bldg.

• Dirksen Cafeteria - SD-G26

Ground level of Dirksen Senate Office Bldg.

Understanding Room Numbers in the House and Senate Office

• Rooms with three 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 four digits:

The first digit indicates the building. Rayburn and Longworth are the only building with four-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 Room is located on the 3rd floor

Example: 2145 Rayburn House Office Room is located on the 1st floor

Resources Section

Presented by:

• The Coffee Shop - SD-BR8

Basement level of Dirksen Senate Office Bldg.

• Southside Buffet - SD-BR8

Basement level of Dirksen Senate Office Bldg.

• Dirksen North Cafe - SD-BR7

Basement level of Dirksen Senate Office Bldg.

House of Representatives

Office Buildings

Network Name: HousePublic

Password: HousePublic

Senate Office Buildings:

Network Name: Senate_Guest

Password: 118congress

Notes

Our Mission

We empower 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.

We Must Act Now

More than 30 million Americans live with one or more rare diseases.

The economic impact of 379 rare diseases reached nearly $1 trillion in the U.S. in 2019.

95% of the more than 10,000 known rare diseases have no FDA-approved treatment.

Rare disease patients wait an average of 6.3 years and 17 healthcare encounters before receiving a rare disease diagnosis.

It takes an average of 15 years for a drug to be developed and approved by the FDA.

Delays in receiving a diagnosis result in an average of $220,000 of avoidable costs per person.

We are an innovative global healthcare company, driven by one purpose: we chase the miracles of science to improve people’s lives.

In Specialty Care, our mission is to help people with debilitating and complex conditions in rare diseases, rare blood disorders, neurology, oncology, and immunology. These conditions are often difficult to diagnose and treat. But we aren’t afraid of challenges. They just push us to work harder, to chase new potential therapies that help patients to live their lives.

MAT-US-2204557-v.1.0-06/2022

Sanofi speciality Care_RareDisease - US _8x4.93.indd 1

1/25/2024

10:29:53 AM

At Travere

Therapeutics,

we are in rare for life.

Transforming Lives. Every Day.

At Alexion, our mission is to transform the lives of people  affected by rare diseases and devastating conditions  through the development and delivery of innovative medicines, as well as through supportive technologies and healthcare services. alexion.com

We come together every day to help patients, families, and caregivers of all backgrounds as they navigate life with a rare disease. On this path, 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.com

ALL NEW UNTOLD STORIES!

The compelling podcast series, Untold Stories: Life with a Severe Autoimmune Condition, is back with a second season! Previously highlighting the experiences of real people living with MG, this season is expanding to share the stories of people living with other rare autoimmune conditions, such as CIDP. Host Martine Hackett continues to speak with a variety of guests, as they share their trials, tribulations, and triumphs.

MG=myasthenia gravis

CIDP=chronic inflammatory demyelinating polyneuropathy LISTEN NOW

Host Martine Hackett Associate Professor, Director of Public Health Programs Hofstra University

Tip: Be Creative! @RareAdvocates #RareDC2024