Washington D.C.—Recognizing that many patients either have no treatments available for their rare diseases or are using older therapies not designed for their disease and the need to update the Food and Drug Administrations decades-old Accelerated Approval regulation, Congressmen Cliff Stearns and Edolphus Towns on March 5, 2012, introduced House Resolution (H.R.) 4132, the bi-partisan bill known as the Faster Access to Specialized Treatments (FAST) Act. Passage of this legislation would modernize and expand the FDA’s Accelerated Approval regulation to encompass a broader range of diseases, speeding the approval of new and novel therapies for rare diseases, while ensuring that the FDA’s high standard for approval is maintained, thereby protecting patients while allowing them access to new and novel treatments for serious or life-threatening diseases. The bill currently is under review by the House Committee on Energy and Commerce.
We encourage you to write to your representative in Congress, especially if he/she is a member of the House Committee on Energy and Commerce and ask him/her to support HR 4132. You can find a sample letter below. If you aren’t sure who is your representative in Congress go here, enter your zip code, and you will be taken to a page with information about and the link to your representative’s official page. This legislation is important to speed the process of making new and innovative treatments available to patients within the LGDA community. The more members of Congress who support the bill, the more likely it will be passed. So, please write today.
The LGDA thanks Congressman Ted Deutch of Florida for joining as a co-sponsor of the bill and encouraging the whole Florida delegation to do the same. Congressman Deutch represents Florida’s 19th Congressional District wherein the Lymphangiomatosis and Gorham’s Disease Alliance is incorporated.
The Honorable ____________________
U.S. House of Representatives
Washington, DC 20515
As a voting constituent in your area dealing with a rare disease for which there currently is no effective treatment and no cure, I am writing to ask you to support H.R. 4132, the FAST Act. This bill modernizes the FDA’s Accelerated Approval rule and provides for speeding the approval of new and novel therapies, while ensuring that the FDA’s high standard for approval is maintained, thereby protecting patients while allowing them access to new and novel treatments for serious or life-threatening diseases. The bill currently is under review by the House Committee on Energy and Commerce.
The course and prognosis of Lymphangiomatosis and Gorham’s Disease are very difficult to predict because there have never been any studies done to analyze these mysterious conditions. Cases vary from person to person and there is no recognized standard diagnostic measure or treatment for physicians to use. In severe cases, the fatality rate is high and in others the quality of life can be greatly affected by a life-time of doctor’s appointments, chronic pain, and even deformity. Our children are those hardest hit by this disease. It happens that the most severe form generally begins at a very young age giving children little hope for chance at a good life and even survival. Passage of the FAST Act will help in the search for treatments and a cure for these diseases and save lives. Again, I ask you to support the FAST Act.
Thank you for your time.