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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Rare disease spurs Utah County woman to be Democratic activist

Politics » Utahn Jamie Hartley says nobody was more surprised than she that her politics leaned left.
by: By Matt Canham| The Salt Lake Tribune

Washington • Of all the Utahns elected as delegates to the Democratic National Convention, Jamie Hartley is the outlier. She isn’t a party insider or union leader, a politician or a campaign strategist.

Hartley has no real political experience at all.

But as a Utah County resident struggling with a painful genetic disorder she’s very familiar with the nation’s medical system, and that’s something she wants to bring to the big political gathering in Charlotte, N.C.

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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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