Rare Disease Legislative Advocates in coordination with Rare Disease Congressional Caucus

Co-Chairs Representatives Leonard Lance (R-NJ) and G. K. Butterfield (D-NC) and

Senators Orrin Hatch (R-UT) and Amy Klobuchar (D-MN) invite you to a briefing on:

The Rare Disease Lifecycle: Diagnosis to Treatment

Wednesday, February 28th, 2018

Ask your Members of Congress to Join the Caucus

It will only take 2 minutes: RDLA will automatically find your legislators for you. All you need to do is enter your address and add a personal note about why it’s important to you. If your legislator is already a member of the Caucus then you can send a thank you letter to your legislator instead.

Caucus Briefings

February 28th, 2018 – The Rare Disease Lifecycle: Diagnosis to Treatment

November 15th, 2017 – Diagnostic Challenges for Rare Disease Patients

September 13th, 2017 – Curing Rare Disease: Policy and Regulation Needed for Emerging Technology

May 18th, 2017 – Incentivizing Innovation for Rare Disease Treatment Development

March 2nd, 2017 – Advancing Rare Disease Treatments in the Era of Cures and Health Care Reform

November 15th, 2016 – Driving Innovation for Lifesaving Therapies through PDUFA Reauthorization in 2017

September 14th, 2016 – Strengthening Medical Innovation in America for Rare Disease Patients

May 18th, 2016 – The NIH and FDA: Vital Agencies in the Fight Against Rare Diseases

March 3rd, 2016 – The Rare Disease Ecosystem: Fostering Patient Engagement & Driving Biomedical Innovation

Nov. 5th, 2015 – Precision Medicine: New Frontiers for Rare Diseases

May 21st, 2015 – 21st Century Cures Initiative: Priorities for the Rare Disease Community

Feb. 26th, 2015 – Urgent Healthcare Policy Needs of the Rare Disease Community

Nov 13th, 2014 – Creating Economic Incentives to Spur the Development of Treatments for Ebola and Other Life-Threatening Rare Diseases

Sept. 17th, 2014 – Implementation of Rare Disease Provisions in FDASIA

May 7th, 2014 – Access to Care & Therapies in the New Healthcare System: A Rare Disease Perspective

Feb. 28th, 2014 –  Science Behind Rare Disease Policy, Feat. Special Guest Jonny Lee Miller


The Congressional Rare Disease Caucus is a forum for Members of Congress to voice constituent concerns, collaborate on ideas, facilitate conversations between the medical and patient community and build support for legislation that will improve the lives of people with rare diseases.

The bipartisan (comprising both Democrats and Republicans) Rare Disease Caucus was established in 2009 by original GOP Co-Chair Congressman Fred Upton (R-MI), who went on to become Chairman of the House Energy and Commerce Committee, arguably the most powerful House committee. Jurisdictional issues within the E & C committee include Food and Drug Administration oversight, as well as a myriad of issues related to healthcare reform and committee rules required Rep. Upton remove himself from leadership of all Caucuses. Upton recommended that Leonard Lance (R-NJ) take over as the Republican Co-Chair of the Caucus, where he now serves with Rep. Joe Crowley (D-NY).

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.

Congressional Caucus

A congressional caucus is a group of members of the United States Congress that meets to pursue common legislative objectives. Formally, caucuses are formed as congressional member organizations (CMOs) through the United States House of Representatives and governed under the rules of that chamber. There are hundreds of Caucuses. The most common caucuses consist of members united as an interest group. A Caucus can hold briefings to raise awareness on an issue. However, briefings are not actionable, ie: no bills can be introduced or voted on. A Caucus may join Members together in a voting block to support or oppose legislation, however most interest group caucuses are used to gain media attention and raise public awareness. Congressional Caucuses must be re-filed in the House at the start of each new Congress. The filing papers must be submitted by the majority party, which is currently the Republicans.