H.R. 3737, Unlocking Lifesaving Treatments for Rare diseases Act of 2012
Save The Date: Tuesday, February 28, 2012
U.S. Representatives Cliff Stearns (R-FL) and Ed Towns (D-NY), two senior members of the House Energy and Commerce Committee, introduced bipartisan legislation to help spur the development of treatments for very rare diseases.
The Unlocking Lifesaving Treatments for Rare diseases Act of 2012, or ULTRA, H.R. 3737, will improve access to the FDA's Accelerated Approval process for very rare diseases, provide a more predictable regulatory process, bring down development costs, and spur investment in the development of treatments. Additionally, ULTRA requires the FDA to use the best science available, ensuring treatments are safe and effective and reach patients sooner.
With the introduction of ULTRA, the EveryLife Foundation will begin to build Congressional support for this important legislation. Most importantly, we thank Representatives Stearns and Towns for their leadership and support of the rare disease community.
In order to empower you to be an expert on the bill and to answer any questions you may have, we have provided some background information on the bill:
- Feb. 8, 2102 Congress Member Towns speaks about ULTRA on the House Floor
- Feb. 7, 2012 Dr. Kakkis' Webinar explaining ULTRA
- Foundation President, Emil Kakkis' blog about why we decided to seek legislation
- Facts about what the bill does and does not do
- Legal explanation of H.R. 3737
- Take action links to write your Members of Congress that you can share with your parent advocates
- House sign-on letter with 140 organizations supporting ULTRA
- Track the bill's co-sponsors, actions, and Congressional Committee assignments
- Power point presentation on ULTRA
- Rep Stearns Press Release on the introduction of the ULTRA Act
- FDA Law Blog: ULTRA Bill Introduced in the House; Legislation Seeks to Permit Broader Use of Scientific Data to Support Surrogate Endpoints for “Ultra Orphan” Drug Approvals
- Dec. 16, 2011, InsideHealthPolicy.com's FDA Week reports Rep Stearns to Introduce the ULTRA Act
- Nov. 11, 2011 FDA Week: Patient Advocates Seek To Accelerate Approval of Ultra Orphan Drugs
- Oct. 14, 2011 Rep Stearns letter to FDA regarding ultra rare diseases
- Click here to read the final bill language
140 patient organizations have already signed on to the letters of support. If you would like to add your organization to the letters, please click here
If you have any additional questions, please don't hesitate to contact us at 415-884-0223 or patientadvocate@kakkis.org.
Watch testimonials on UTLRA from our parent advocates:
