FOR IMMEDIATE RELEASE – HR 5651, the Food and Drug Administration Reform Act of 2012
FOR IMMEDIATE RELEASE House FDA User Fee Bill Will Unlock Lifesaving Treatments for Rare Diseases Rare Disease Community Applauds House Energy & Commerce Committee for User Fee Reauthorization Legislation that Spurs the Development of Treatments Thursday, May 10, 2012, WASHINGTON, DC—– Today the House Energy & Commerce Committee finished mark-up of HR 5651, the “Food and [...]
Michelle Obama’s Video Challenge: Vote for Prader-Willi Syndrome and Rare Disease
Taking Rare Disease To the White House The Foundation for Prader-Willi Research (FPWR) and the Prader-Willi Syndrome Association (PWSAUSA) have entered a video into Michelle Obama’s video challenge to fight childhood obesity. You can view their video submission and help to spread the word about rare disease by casting your VOTE (everyday) at “Take One SMALL [...]
Policy Update – PDUFA
We’re continuing to work on improving PDUFA to allow the FDA additional authority to include RARE disease in the Accelerated Approval Program. You can learn more here from last week’s public call for support and our other public policy blogs. After a very successful response from the patient community last week, today we are mobilizing organizations [...]
ACTION ALERT – Accelerated Approval to treatments for rare disease patients!
The RARE Project has been working with Rare Disease Legislative Advocates (RDLA) on legislative improvements that would help bring more drugs to rare disease patients more quickly. This legislation promotes the use a variety of scientific data to qualify a surrogate endpoint for use in a clinical trial, when the required historical clinical information on [...]
Legislative Advocates Conference Call: PDUFA Update
Please join us for a Conference Call on Wednesday, March 28, 2012 at 1:00 p.m. Eastern Draft Agenda: Update the Prescription Drug User Fee Act (PDUFA) reauthorization process Accelerated Approval Language: ULTRA/FAST/TREAT Acts – EveryLife Foundation EXPERRT Act - Cystic Fibrosis Foundation - invited Expediting New Treatments to Patients: FDA Approval Mechanisms - Friends of Cancer Research – invited Accelerated Approval Challenges [...]
CALL TO ACTION !!
Rare Disease Language included in House draft PDUFA Bill RARE Project would like you to consider taking action in support of the new language (FAST Act) related to the Prescription Drug User Fee Act (PDUFA) in the following ways; The more support there is for the FAST Act, the better positioned the rare [...]
The Grassroots Movement Behind Ultra and Fast: Join the Movement!
A Guest Blog by Julia Jenkins: Director of Government Relations, EveryLife Foundation The Grassroots Movement Behind ULTRA-FAST & How you can join the Movement! We are very excited to share the news that rare disease Accelerated Approval language made it into the House draft PDUFA bill. The language that incorporates the goals of [...]
USA Today Spotlights FDA Approval
Advocating for your children can and does make a difference! On March 6, USA Today published an article spotlighting a brave family with twin girls suffering a rare and fatal genetic disease. Niemann-Pick Type C disease, often called childhood Alzheimer’s disease, has brought the Hempel family through rigorous challenges and constant battles in an effort to keep [...]
Change is Needed at the FDA
“Breakthrough technologies deserve a breakthrough in the way the FDA evaluates them.” - Andrew Von Eschenbach Mr. Von Eschenbach couldn’t have stated it any clearer. On February 14, the Wall Street Journal posted an on-line article by Mr. Eschenbach titled: Medical Innovation: How the U.S. Can Retain Its Lead, The FDA should approve drugs based on [...]