John Crowley Talks About the Fifth Prescription Drug User Fee Act


On the last day of the World Orphan Drug Congress, John Crowley, Chairman and CEO, Amicus Therapeutics provided an inspiring overview of how the Fifth Prescription Drug User Fee Act (PDUFA V) and new government policies may affect the orphan drug industry. From a historical […]

FDA User Fee Bill Is Landmark Legislation – Major Wins for the Rare Disease Patient Community Led by RDLA

RDLA is hosting an Advocates Luncheon & Conference Call on September 12, 2012.  RSVP is required for attendance.

Wednesday, June 27, 2012, Washington, DC — Rare Disease Legislative Advocates (RDLA) hails House and Senate passage of The Food and Drug Administration Safety and Innovation Act (FDASIA), S. 3187, landmark legislation that will encourage the development of new treatments for the 30 million Americans […]

HR 5651, the Food and Drug Administration Reform Act of 2012


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 […]

PDUFA Update and Accelerated Approval Program For Rare Diseases


We’re continuing to work on improving PDUFA to allow the FDA additional authority to include rare diseases in the Accelerated Approval Program. After a very successful response from the patient community last week, today we are mobilizing organizations to show their support for allowing the […]

RARE ACTION ALERT – Accelerated Approval to treatments for rare disease patients


The Global Genes Project and R.A.R.E. team have 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 […]

Legislative Advocates Conference Call: PDUFA Update

RDLA is hosting an Advocates Luncheon & Conference Call on September 12, 2012.  RSVP is required for attendance.

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 […]

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 community […]