Please Join the Rare Disease Congressional Caucus

The bipartisan and bicameral Rare Disease Congressional Caucus is led by Representatives G. K. Butterfield (D-NC) and (R-FL), and Senators Roger Wicker (R-MS) and (D-MN) to promote awareness of rare disease issues.

Background: There are more than 7,000 known rare disorders that together affect more than 30 million Americans and their families. One in 10 Americans has a rare disease. 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 affect fewer than 100 people. 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 Orphan Drug Act was enacted in 1983 to encourage pharmaceutical companies to develop drugs for diseases that have relatively small patient populations. Despite the success of the Orphan Drug Act, there have been fewer than 700 treatments for less than 550 diseases approved for marketing by the Food and Drug Administration (FDA) in the last 30 years. The science exists for many of these diseases to be treated; however, treatments may never be developed because of roadblocks in the development process, such as a lack of investment and a challenging regulatory environment. Additionally, while a relatively few treatments have become available, patients struggle with insurance companies and government programs to afford these lifesaving treatments. Solution: 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 roadblocks to the development of and access to crucial treatments. The Caucus gives a permanent voice to the rare disease community on Capitol Hill. Working together, we can find solutions that turn hope into therapies and cures.

Contact: RDLA: [email protected]; Senator Wicker’s office: [email protected] or Rep. Butterfield’s office: [email protected]

Rare Disease Legislative Advocates (RDLA) is a program of the EveryLife Foundation for Rare Diseases designed to support the advocacy of all rare disease patients and organizations. RDLA is committed to growing the patient advocacy community and working collaboratively, thereby amplifying the patient voice to be heard by local, state, and federal policy makers.

RARE DISEASE LEGISLATIVE ADVOCATES (A PROGRAM OF THE EVERYLIFE FOUNDATION) 1012 14TH STREET NW, SUITE 500, WASHINGTON DC 20005 OFFICE: 202-697-RARE (7273) WWW.RAREADVOCATES.ORG @RAREADVOCATES

Senate Co-Chairs: Senators Roger Wicker (MS) and Amy Klobuchar (MN) House Co-Chairs: Representatives G. K. Butterfield (NC-1) and Gus Bilirakis (FL-12)

HOUSE NE-1 OK-2 MI-6 NV-2 AZ-7 MA-6 CA-51 IA-3 CA-3 MA-1 Nydia Velazquez NY-7 Andy Barr KY-6 NJ-5 NJ-1 Peter Visclosky IN-1 OH-3 LA-6 SC-5 MO-2 CA-7 Danny Heck WA-10 DC OR-2 TX-30 Jaime Herrera-Beutler WA-3 MS-4 Debbie Wasserman-Schultz Donald Beyer Jr. VA-8 Kevin Herns OK-1 NJ-6 FL-23 Gus Bilirakis* FL-12 CT-4 CA-20 NJ- , Jr. GA-2 Kendra Horn OK-5 Chris Pappas NH-1 12 DE CA-2 NJ-9 Joe Wilson SC-2 OR-1 GA-4 Donald Payne, Jr. NJ-10 Robert Wittman VA-1 AL-5 SD-AL Scott Peters CA-52 KY-3 CA-26 David P. Joyce OH-14 MN-7 NY-1 FL-16 OH-9 ME-1 SENATE Michael Burgess TX-26 Joseph Kennedy, III MA-4 FL-8 John Barrasso WY IL-17 CA-17 David Price NC-04 John Boozman AR G.K. Butterfield* NC-1 WA-6 Mike Quigley IL-5 Maria Cantwell WA CA-24 NJ-3 MD-8 Shelley Moore Capito WY Andre Carson IN-7 WI-3 NY-4 Christopher Coons DE John Carter TX-31 Peter King NY-2 Martha Roby AL-2 AR IL-6 IL-8 Max Rose NY-11 Charles Grassley IA OH-1 Darin LaHood IL-18 NC-7 John Hoeven ND CA-27 Jim Langevin RI-2 C.A. Cindy Hyde-Smith MS RI-1 OH-5 MD-2 James Inhofe OK Lacy Clay MO-1 NV-3 John Rutherford FL-4 Doug Jones AL , II MO-5 CA-49 IL-9 John Kennedy LA TN-9 CA-33 IL-10 Angus King ME KY-1 IL-3 David Scott GA-13 Amy Klobuchar* MN Gerald Connolly VA-11 Dave Loebsack IA-2 NJ-11 Edward Markey MA TN-5 CA-19 ID-2 Jeff Merkley OR CO-6 CA-47 NJ-8 Gary Peters MI Susan Davis CA-53 MO-3 MI-8 James Risch ID Rodney Davis IL-13 NW-7 WA-9 Kyrsten Sinema AZ Peter DeFazio OR-4 NY-12 Chris Smith NJ-4 Jeanne Shaheen NH Diana DeGette CO-1 NY-18 MO-8 Tina Smith MN Rosa DeLauro CT-3 TX-24 PA-16 Debbie Stabenow MI Suzan DelBene WA-1 FL-18 FL-9 MD Antonio R. Delgado NY-19 CA-6 CA-14 Roger Wicker* MS FL-21 Ben McAdams UT-4 Chris Stewart UT-2 MI-12 Michael McCaul TX-10 OH-15 Mike Doyle PA-14 Jim McGovern MA-2 CA-15 NY-16 David McKinley WV-1 Glenn Thompson PA-5 CA-18 Cathy McMorris Rodgers MI-13 Abby Finkenauer IA-1 WA-5 NY-20 Brian Fitzpatrick PA-8 NY-6 MA-3