H.R. 6342 – The Compassionate Freedom of Choice Act of 2012

Statement on Introducing the Compassionate Freedom of Choice Act, by Rep. Ron Paul (R-TX)

Mr. Speaker, I rise to introduce the “Compassionate Freedom of Choice Act.” This legislation allows terminally ill patients to use drugs, treatments and devices that have not yet been approved by the Food and Drug Administration (FDA) if their physicians certify: (i) such patients have no other treatment options; and (Ii) the patient executes written, informed consent that they are aware of any potential risks from the drug, device, or treatment.

Read more

H.R. 6288 – The Patient Choice Act of 2012

Congressman Brian Bilbray introduces bill to provide patients fighting life threatening diseases greater access to treatment

Washington, D.C. – Congressman Brian Bilbray (CA-50) today introduced H.R. 6288, the Patient Choice Act of 2012, a bill to speed up the approval process of drugs used in therapies and treatments of patients fighting life threatening diseases.

Read more

The Food and Drug Administration Safety and Innovation Act (FDASIA), S. 3187

One Hundred Twelfth Congress of the United States of America

An Act  To amend the Federal Food, Drug, and Cosmetic Act to revise and extend the user-fee programs for prescription drugs and medical devices, to establish user-fee programs for generic drugs and biosimilars, and for other purposes.

Signed into law on July, 9, 2012

Read more

Life-Threatening Diseases Compassion through Combination Therapy Act of 2012 (H.R. 6502)

Legislation co-sponsored by Representatives Brian Bilbray (R-CA), Carolyn Maloney (D-NY) and Rosa DeLauro (D-CT), and supported by the Melanoma Research Foundation, seeks to encourage the development of so-called “significant drug combinations” by offering the carrot of an extension of marketing exclusivity.

Read more

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.

Read more

September Sequester Surprise: FDA User Fees on the Chopping Block


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.

Read more

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.

Read more

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

Read more

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


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

Read more

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.

Read more