The Rare Disease Congressional Caucus

Established in 2009, the Rare Disease Congressional Caucus is bipartisan (comprising both Democrats and Republicans) and bicameral (comprising both Representatives and Senators). The original GOP Co-Chair Congressman Fred Upton (R-MI) has become Chairman of the House Energy and Commerce Committee, arguably the most powerful House committee. Jurisdictional issues within the committee include Food and Drug Administration oversight as well as a myriad of issues related to healthcare reform. Committee rules require that Rep. Upton remove himself from all Caucuses. While Upton is not able to serve as the Rare Disease Caucus Co-Chair, he remains passionate about helping patients with rare diseases and helping us seek legislative solutions. He will continue to be a trusted ally to the rare disease community. Upton recommended that Leonard Lance (R-NJ) take over as the Republican Co-Chair of the Caucus.

Full House Member List listed at: Rareadvocates.org/rarediseasecaucus

Rare Disease Congressional Caucus Briefing Videos Now Available

The Rare Disease Congressional Caucus hosted a briefing during Rare Disease Week on Capitol Hill which focused on Advancing Rare Disease Treatments in the Era of Cures and Health Care Reform.

After welcoming remarks from Caucus Co-Chair Senator Amy Klobuchar (D-MN), the panelists addressed implementation of the 21st Century Cures Act, reauthorization of the Prescription Drug User Fee Act, efforts to repeal and replace the Affordable Care Act, new models for rare disease drug development such as repurposing, and the role of incentives to encourage the development of orphan therapies.

Videos from the briefing are available here. A full list of Caucus members and videos from previous briefings are available on the Caucus webpage.

Video from Rare Disease Congressional Caucus Briefing on PDUFA Now Available

The final Rare Disease Congressional Caucus briefing of 2016 focused on the Prescription Drug User Fee Act (PDUFA), the agreement between the biopharmaceutical industry and Food and Drug Administration (FDA) regarding user fees paid by industry to supplement federal funding for human drug review.

Congress will need to reauthorize PDUFA before the current agreement expires at the end of September 2017, which will be an opportunity to include provisions to encourage the development and streamline the review of rare disease treatments.

Watch the videos to learn more from representatives from debra of America, National Health Council, Biotechnology Innovation Organization, Genentech and office of U.S. Representative Leonard Lance (NJ).

http://rareadvocates.org/driving-innovation-for-lifesaving-therapies-through-pdufa-reauthorization-in-2017/

Video from Rare Disease Congressional Caucus Briefing on Strengthening Medical Innovation Now Available

On September 14, 2016, the Rare Disease Congressional Caucus gathered a contingent of thought-leaders in the rare disease space to discuss Strengthening Medical Innovation in America for Rare Disease Patients.

The NIH and FDA: Vital Agencies in the Fight Against Rare Diseases

Rare Disease Legislative Advocates in coordination with Rare Disease Congressional Caucus Co-Chairs: Representative Leonard Lance (R-NJ), Representative Joe Crowley (D-NY), Senator Orrin Hatch (R-UT), and Senator Amy Klobuchar (D-MN); and the Office of Senator Mark Kirk (R-IL) will host a briefing on: The NIH and FDA: Vital Agencies in the Fight Against Rare Diseases

Video: March 3rd Rare Disease Caucus Briefing on The Rare Disease Ecosystem

Earlier this month, the Rare Disease Congressional Caucus hosted a briefing on The Rare Disease Ecosystem: Fostering Patient Engagement & Driving Biomedical Innovation. This briefing was featured as part of the 2016 Rare Disease Week on Capitol Hill. In addition to speeches by caucus co-chairs Senators Orrin Hatch and Amy Klobuchar, Representatives Leonard Lance and Joe Crowley, Representative Gus Bilirakis made a surprise appearance.

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Rare Disease Congressional Caucus Adds Nine Members

Thanks to their diligent work educating Members of Congress, rare disease advocates were successful in expanding the ranks of the Rare Disease Congressional Caucus. In March, nine legislators agreed to join the Rare Disease Congressional Caucus:

Senator James Risch (Idaho)
Senator David Vitter (Louisiana)RDCC-Horizontal-CMYK
Representative Brad Ashford (NE-2)
Representative David Cicilline (RI-1)
Representative John Garamendi (CA-3)
Representative Joe Kennedy, III (MA-4)
Representative Donald Norcross (NJ-1)
Representative David Rouzer (NC-7)
Representative Juan Vargas (CA-51)

We applaud these legislators for taking leadership on the issues that are important to the rare disease community. You can find the complete Rare Disease Congressional Caucus membership at http://rareadvocates.org/rarecaucus/.

EveryLife Foundation for Rare Diseases Announces Formation of Bicameral Rare Disease Congressional Caucus

12-16-15 – Washington, D.C. – In a show of bipartisan support for the rare disease community, members of the House and Senate have come together to form the first bicameral Rare Disease Congressional Caucus.

The Caucus will be critical in raising awareness of the challenges faced by rare disease patients and serve as a platform for giving those individuals a voice in both the House and Senate. In addition, the Caucus will convene briefings throughout the year to educate Congressional staff on topics of importance to the rare disease community.

The Caucus will be co-chaired by Representatives Lance (R-NJ) and Crowley (D-NY)  and Senators Hatch (R-UT) and Klobuchar (D-MN).

“Throughout my time in the Senate, I have worked hard to help the 30 million Americans with rare diseases,” Sen. Hatch said. “While we have previously taken important steps, like passing the Orphan Drug Act, the fact that 95 percent of rare diseases currently have no treatment shows that much more needs to be done. Senator Klobuchar has been a terrific partner on legislation to get vital treatments to patients in need, and I am honored to join her as Senate Co-Chairs of the Rare Disease Congressional Caucus.”

“While there are about 7,000 known rare diseases, there are fewer than 500 drugs approved to treat any of those conditions,” Sen. Klobuchar said. “It is critical that we work together to increase the number of safe, effective, and affordable treatments that are available for people with rare diseases. I have long worked with Senator Hatch on this issue and I look forward to continuing this work as a Senate Co-Chair of the Rare Disease Congressional Caucus.”

The Rare Disease Congressional Caucus, originally formed in 2009 by Rep. Upton (R-MI), is currently comprised of 88 Members of the House and will be expanded to include the Senate.

“We are grateful to our House champions and to Senators Hatch and Klobuchar for stepping-up to lead this Caucus on behalf of the rare disease community. We are looking forward to raising awareness about the importance of public policy to spur the development of new therapies for rare disease patients,” said Julia Jenkins, Executive Director of the EveryLife Foundation for Rare Diseases.

Rare Disease Legislative Advocates (RDLA), a program of the EveryLife Foundation for Rare Diseases, will continue to coordinate regular Hill briefings and act as a legislative clearinghouse for the patient community, with the goal of mobilizing patients to be effective legislative advocates (www.rareadvocates.org).

Precision Medicine: New Frontiers for Rare Diseases

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Rare Disease Legislative Advocates in coordination with Rare Disease Congressional Caucus Co-Chairs:

Representatives Leonard Lance (R-NJ) and Joe Crowley (D-NY), will host a briefing on:
Precision Medicine: New Frontiers for Rare Diseases

Thursday, November 5th, 2015
12:00 pm – 1:00 pm (lunch provided)
Rayburn House Office Building
Room: B318 

speakers for precision Medicine briefing

Moderator: John Crowley, Chairman & CEO, Amicus Therapeutics

  • Matt Might, President, NGLY1.org & Associate Professor, University of Utah
  • Erynn Gordon, Director of Clinical Development, 23andMe
  • John Stone, Counsel, Committee on Energy and Commerce – U.S. House of Representatives
  • Dr. Christopher Austin, Director, National Center for Advancing Translational Sciences, NIH
  • Sean Sigmon, Business Development Director, Oracle Health Sciences

Limited Seating, RSVP to attend

Can’t attend? Click here to contact your congressional office to ask them to send a representative!

Hosted by:

Precision Medicine Logo Mashup

Congressman Eric Swalwell Joins the Congressional Rare Disease Caucus

077In July, Rep. Eric Swalwell (D-CA) became the 89th member of the Congressional Rare Disease Caucus. Congressmen Swalwell has consistently urged Congress to increase funding to the National Institutes of Health (NIH) in order to fund the research necessary to battle the rare diseases that affect 30 million Americans.

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Rare Disease Congressional Caucus Briefing – 21st Century Cures Initiative: Priorities for the Rare Disease Community