PDUFA V: Accelerated Approval Expansion May Outshine Rare Disease Improvements

Rare disease advocate says the focus should be on ensuring the accelerated approval expansion and surrogate endpoint guidances become FDA priorities as the rare disease drug program is set to grow under PDUFA V.

By Derrick Gingery / Email the Author / “The Pink Sheet” Sep. 17, 2012, Vol. 74, No. 38

Regulatory Update / Article # 00120917013

Congress mandated specific upgrades to FDA’s rare disease program in the new user fee legislation, but the largest impact may come through the expected changes to the accelerated approval pathway.

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September Sequester Surprise: FDA User Fees on the Chopping Block

BY ELIZABETH M. WROE & PHILIP S. BONFORTE

The threat of a delayed or deterred (re-)authorization of key Food and Drug Administration (FDA) user fee programs (hereinafter referred to as the ‘‘FDA user fees’’) caused significant angst among biomedical product stakeholders over the course of the last year and a half.

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FDA Law Blog: Congressional Interest in FDA Remains High, Even After the Enactment of FDASIA

By Kurt R. Karst 

Just before Congress recessed for the month of August, and less than a month after the July 9th enactment of the FDA Safety and Innovation Act, several FDA-related bills were placed in the hoppers in the U.S. Senate and U.S. House of Representatives.  With an election on the horizon and several other non-FDA-related issues to handle, it seems highly unlikely that Congress will be poised to tackle the FDA bills this year; however, they may provide some insight into some of the issues that will be debated in the 113th Congress come 2013.

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The ULTRA/FAST Legislation included in FDASIA

From the EveryLife Foundation for Rare Diseases: FOR IMMEDIATE RELEASE

 Obama Signs FDA User Fee Legislation Bringing Hope to Rare Disease Patients
EveryLife Foundation for Rare Diseases Applauds Congress for Including Provision to Empower the FDA to Accelerate Approval of Lifesaving Treatments

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National Pediatric Research Network Act (H.R. 6163/S. 3461)

SENS. SHERROD BROWN, ROGER WICKER INTRODUCE BIPARTISAN LEGISLATION to Strengthen and Expand Pediatric Research

Despite Children Making Up 20 Percent of the U.S. Population, Just Five Percent of NIH’s Research is Dedicated to Pediatric Research; National Pediatric Research Network Act Expands NIH’s Investments into Pediatric Research, Including Rare Diseases

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Obama signing a bill

President Signs Rare Disease Legislation into Law


FDA User Fee Bill Promises Real Hope for Rare Disease Patients

Major Wins for the Grassroots Patient Community

July 9, 2012, Washington, DC —– Rare Disease Legislative Advocates (RDLA) hails the President’s singing 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 suffering from rare diseases. There are currently fewer than 400 treatments approved by the U.S. Food and Drug Administration (FDA) for the nearly 7000 rare diseases which have been identified.

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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.