H.R. 460 – Patients’ Access to Treatments Act (PATA)

The bipartisan Patients’ Access to Treatments Act (H.R. 460), introduced by Rep. David McKinley (R-WV) and Rep. Lois Capps (D-CA), limits cost-sharing requirements applicable to medications in a specialty drug tier (typically Tier IV or higher) to the dollar amount applicable to drugs in a non-preferred brand drug tier (typically Tier III). It will enable patient access to treatments, reduce disability and constrain health care costs.

Read more

Senator Brown wants more pediatric-research funding: Bill would shift more federal money to kids’ illnesses

THE COLUMBUS DISPATCH Tuesday January 29, 2013 8:24 AM
By  Joshua Jamerson 

When Madison Reed was 7 months old, doctors told her parents she wouldn’t live past 2. “No parents should have to hear those words,” Annette Reed said. But unconventional methods helped Madison, who had type-1 spinal muscular atrophy, a rare disease impairing her motor functions. Now the Dublin resident is 15 years old.

The Reeds stood behind Sen. Sherrod Brown yesterday at Nationwide Children’s Hospital as the Ohio Democrat used their story to illustrate the need to increase funding for pediatric research.

Read more

National Neurological Disease Data Collection – H.R. 2595/S.425

The legislation will establish a national data surveillance system that will track and collect data on the epidemiology, incidence, prevalence, and other factors of neurological diseases, including Parkinson’s and Multiple Sclerosis (MS).  The surveillance system will be developed and maintained by the Centers for Disease Control and Prevention (CDC).  This system will provide researchers and epidemiologists with a coordinated system of health data on neurological diseases and should fuel further research.

Read more

Life-Threatening Diseases Compassion through Combination Therapy Act of 2012, HR 6502

New Legislation Would Provide Exclusivity Add-on for Significant Drug Combinations

By Kurt R. Karst, FDA Law Blog

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 encourge the development of so-called “significant drug combinations” by offering the carrot of an extension of marketing exclusivity.

Read more

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

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.

Read more