The Grassroots Movement Behind ULTRA/FAST & How you can join the Movement!

By Julia Jenkins, EveryLife Foundation Director of Government Relations

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 ULTRA is the only mention of rare diseases in the entire 205-page bill. This is a huge accomplishment for the rare disease community and is a major step toward spurring the development of lifesaving treatments. While there is still work to be done, and it is too soon to pop open the champagne, I will take a few moments to tell the story of how a few parent activists ignited the grassroots patient community to influence the legislative process.

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Brownback/Brown Amendment for Rare & Neglected Diseases: FDA Report 2011

Report to Congress:  Improving the Prevention, Diagnosis, and Treatment of Rare and Neglected Diseases

In Response to the Brownback/Brown Amendment, Section 740
The Agriculture, Rural Development, Food and Drug Administration, and Related Agencies Appropriation Act, 2010

Department of Health & Human Services: Food and Drug Administration, March 2011

INTRODUCTION

Section 740 of the fiscal year (FY) 2010 Appropriation Act (Agriculture, Rural Development, Food and Drug Administration, and Related Agencies Appropriation Act, 2010, Public Law 111-80), dated October 21, 2009, required the Commissioner, Food and Drug Administration (FDA), to establish two review groups within FDA to make recommendations to the FDA Commissioner on appropriate preclinical, trial design, and regulatory paradigms and optimal solutions to prevent, diagnose, and treat (1) rare diseases and (2) neglected diseases of the developing world. Section 740 requires these groups to submit a report to Congress that describes their findings and recommendations. The language in section 740 of the Appropriation Act follows:

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NIH’s Cures Acceleration Network (CAN)

The Cures Acceleration Network (CAN) was authorized to advance the development of high need cures and reduce significant barriers between research discovery and clinical trials. To achieve these objectives, CAN provides the National Center for Advancing Translational Sciences (NCATS) at the National Institutes of Health (NIH) with new flexibilities in its funding authorities.

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Rare Disease Acts of 2002

On November 7, 2002, the White House announced that President Bush signed into law the Rare Diseases Act (H.R. 4013) and the Rare Diseases Orphan Product Development Act (H.R. 4014).

Originally introduced in August 2001 by Senators Edward Kennedy (D-MA) and Orrin Hatch (R-UT)  the Rare Diseases Act of 2001 (S. 1379) was later split into two separate bills: H.R. 4013, sponsored by Representative John Shimkus (R-IL), nearly doubles the budget for the Office of Rare Diseases (ORD) at the National Institutes of Health (NIH), and authorizes the office to enter into cooperative agreements and to award grants for clinical research into rare diseases. H.R. 4014, sponsored by Representative Mark Foley (R-FL), doubles the funding for the Food and Drug Administration’s Orphan Products Research Grant program. Congressman Henry Waxman (D-CA), a long-time supporter of the rare disease community and the chief sponsor of the Orphan Drug Act, co-sponsored both H.R. 4013 and H.R. 4014.

Prescription Drug User Fee Act (PDUFA) of 1992

In 1992, Congress first enacted the Prescription Drug User Fee Act (PDUFA) which collects fees from drug manufacturers. The FDA uses the additional revenue stream to hire more staff with the goals of:

  1. More efficient and timely review of license applications, and
  2. Increase their medical and scientific expertise.
The law was authorized for five years and has been reauthorized 4 times (PDUFA II, PDUFA III, PDUFA IV and PDUFA V in 2012) and is up for reauthorization again in 2017.

Orphan Drug Act of 1983

111TH CONGRESS

1ST SESSION

H. R. 5238

IN THE HOUSE OF REPRESENTATIVES

December 15, 1981

Mr. Waxman introduced the following bill; which was referred to the Committee on Energy and Commerce

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