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 [...]
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 [...]
FAST Act – Speeding RARE Disease Therapies
Last week over 80 RARE Disease advocates lobbied on Capitol Hill to enable better access to Accelerated Approval of therapies. We discussed H.R. 3737 “Unlocking Lifesaving Treatments for Rare-Diseases Act (ULTRA)” during our day on the hill. Today, an additional piece of bipartisan legislation, the FAST Act (HR 4132), was introduced in the House. Incorporating [...]
RARE Disease Drug Development … it’s complicated!
The development of any drug is a very complicated process … for RARE diseases the task is especially difficult due to the limited patient populations. Typically the disease is less well understood and there are fewer patients to access for testing to prove the drug works. We’re hoping this simplified overview of the process will [...]
RARE DISEASE LOBBY DAY – ADVOCATES WELCOME
Are you an advocate for Rare Disease? Are you interested in furthering the development of treatment options for Rare Disease? Are you wanting to attend but just not sure how to get there? JOIN US AND MAKE SURE CONGRESS HEARS YOUR VOICE! RDLA has $500 travel scholarships available to advocates who wish to attend and [...]