Historic Legislation Signed by President Obama To Help Patients with Rare Diseases

The Food and Drug Administration Safety and Innovation Act (FDASIA) provides major provisions that could help patients with rare diseases.

President Obama signed the Food and Drug Administration Safety and Innovation Act (FDASIA) yesterday.  This new law includes major provisions of importance to the rare disease patient community and creates several changes to FDA policy including a provision to empower the FDA to accelerate approval […]

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 […]

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 […]

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 […]

FAST Act – Speeding Rare Disease Therapies, Educating Capitol Hill

The Compassionate Allowance List to be announced on Thursday, December 6, 2012.

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 […]

Rare Disease Day: Together, We Can Do More

PMWC 2012

Today is the fifth annual observation of Rare Disease Day, an international event recognized in more than 50 countries.  (Learn about US activities here, including a day of lobbying on Capitol Hill for the ULTRA Act, which aims to stimulate the development of treatments for […]