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 over 7,000 rare disorders that together affect more than 30 million Americans and their families. Rare or orphan diseases are defined as diseases affecting fewer than 200,000 people in the . Many rare diseases are considered ultra-rare; 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 incentivize pharmaceutical companies to develop therapies for diseases that have relatively small patient populations. Despite the success of the Orphan Drug Act, 93% of rare diseases still do not have a treatment approved by the Food and Drug Administration. 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 relatively few treatments have been approved, patients struggle with health insurance reimbursement and other coverage barriers that prohibit access to potentially 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 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.

For more information and to cosponsor contact: RDLA: [email protected]; Sen. Klobuchar’s office: [email protected]; Sen. Wicker’s office: [email protected]; Rep. Bilirakis’ 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 NV-2 NE-1 SC-5 CA-51 IA-3 AZ-7 DC Nydia Velazquez NY-7 Andy Barr KY-6 CA-3 MS-4 MO-2 OH-3 NJ-5 NJ-6 Debbie Wasserman-Schultz CA-7 LA-6 CA-20 FL-23 TX-30 Jaime Herrera-Beutler WA-3 Chris Pappas NH-1 NJ- Donald Beyer Jr. VA-8 Kevin Herns OK-1 NJ-9 12 Gus Bilirakis* FL-12 CT-4 Donald Payne, Jr. NJ-10 Joe Wilson SC-2 , Jr. GA-2 CA-2 Scott Peters CA-52 Robert Wittman VA-1 DE GA-4 MN-03 KY-3 OR-1 SD-AL ME-1 NY-1 AL-5 NY-17 CA-45 SENATE CA-26 David P. Joyce OH-14 FL-8 John Barrasso WY FL-16 OH-9 David Price NC-04 John Boozman AR Michael Burgess TX-26 CA-17 Mike Quigley IL-5 Maria Cantwell WA IL-17 WA-6 MD-8 Shelley Moore Capito WY CA-42 NJ-3 NY-4 Christopher Coons DE G.K. Butterfield* NC-1 WI-3 Martha Roby AL-2 AR CA-24 IL-8 NC-7 Charles Grassley IA Andre Carson IN-7 Darin LaHood IL-18 C.A. John Hoeven ND John Carter TX-31 Jim Langevin RI-2 MD-2 Cindy Hyde-Smith MS IL-6 OH-5 John Rutherford FL-4 James Inhofe OK OH-1 NV-3 PA-5 John Kennedy LA CA-27 AZ-08 IL-9 Angus King ME RI-1 MI-09 IL-10 Amy Klobuchar* MN , II MO-5 CA-49 David Scott GA-13 Edward Markey MA TN-9 CA-33 NJ-11 Jeff Merkley OR Jenniffer Gonzalez-Colon PR- CA-19 ID-2 Jerry Moran KS AL CA-47 NJ-8 Gary Peters MI KY-1 MO-3 MI-8 James Risch ID Gerald Connolly VA-11 NJ-7 WA-9 AZ TN-5 NY-12 Chris Smith NJ-4 Jeanne Shaheen NH CO-6 NY-18 MO-8 Tina Smith MN Rodney Davis IL-13 FL-18 PA-16 Debbie Stabenow MI KS-03 CA-6 FL-9 MD Peter DeFazio OR-4 Michael McCaul TX-10 CA-14 Roger Wicker* MS Diana DeGette CO-1 Jim McGovern MA-2 Chris Stewart UT-2 Rosa DeLauro CT-3 David McKinley WV-1 OH-15 Suzan DelBene WA-1 Cathy McMorris Rodgers NY-03 Antonio R. Delgado NY-19 WA-5 CA-15 FL-21 NY-6 Glenn Thompson PA-5 MI-12 OK-2 MI-13 Mike Doyle PA-14 MA-6 NY-20 CA-18 MA-1 MA-3 Brian Fitzpatrick PA-8 NJ-1 MI-6