ALS Association Priority Legislation Signed into Law

FDA Bill Will Speed Access to Breakthrough Treatments

The Advocacy Department is pleased to announce that yesterday, Thursday, September 27, 2007, President Bush signed into law one of The ALS Association’s top public policy priorities, the reauthorization of the Prescription Drug User Fee Act (PDUFA). The bill, which was included as part of a broader piece of legislation, the Food and Drug Administration Amendments Act of 2007 (H.R. 3580), will help to speed access to breakthrough treatments for ALS. Importantly, the legislation included critical provisions that will foster innovation, improve drug development and help us bring new treatments from the lab to the bedside as soon as possible.

As one of our top advocacy priorities, The Association played leading role in helping to pass the bill. In addition to testifying at public hearings convened by the FDA, which helped to create the initial legislation, The Association was the only patient organization invited to testify at a House Energy and Commerce Committee hearing held on PDUFA in April. At the hearing, Jim Thew, a person with ALS and advocate from The Association’s Greater Chicago Chapter, testified to Congress about the urgent need to act. Seated in a power wheelchair and with the assistance of his wife Kumiko, the 35 year-old Navy veteran and father of three educated the Committee on the true nature of ALS, its progression, its impact on families and the fact that currently there is no drug available to effectively treat the disease.

Jim’s testimony was a clear reminder to the Committee that the drug approval process must not limit innovation or delay access to drugs and biologics that could save or improve the lives of people with ALS or other serious and life-threatening diseases. This was a critical point of the debate, as a major focus of Congressional efforts has been to strengthen the drug safety system in the wake of controversies involving high profile drugs, such as Vioxx.

The bill enacted into law includes a number of key provisions supported by The ALS Association, including:

Reauthorizes the Prescription Drug User Fee Act, which helps to fund and expedite drug approvals. The bill will increase user fees to $393 million in Fiscal Year 2008, an $87 million increase over the current year.
Establishes the Reagan-Udall Foundation. The Foundation will advance the FDA’s Critical Path Initiative to explore innovative ways to improve the drug development process, including the pursuit of biomarkers and other strategies to more rapidly translate clinical advances into breakthrough treatments. The Foundation also will help to facilitate partnerships between industry, patient organizations, the public and the FDA to identify new ways to develop, manufacture and evaluate the safety and effectiveness of drugs and biologics.
Creates a Clinical Trials Registry, which will make Phase II, Phase III, and Phase IV clinical trials information available to the public. Clinical trial results also are required to be made available to the public.
Authorizes improved safety standards, such as Risk Evaluation and Mitigation Strategies (REMS) and enhanced post market surveillance activities that will not slow drug development or limit access to new drugs and biologics.
The ALS Association strongly believes that this new law will help us to continue to make progress in the fight for a treatment and cure for ALS.

If you would like additional information about this update, please contact the Advocacy Department at advocacy@alsa-national.org.