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 / […]
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.
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 […]
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
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
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.
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 […]
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 […]
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.
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 […]