April 2010

ACT-AD Chair Makes Remarks on Prescription Drug User Fee Act Reauthorization

On April 12th, ACT-AD Chair Daniel Perry presented remarks at a public meeting on the reauthorization process for the Prescription Drug User Fee Act (PDUFA).  PDUFA IV, currently set to expire in September of 2012, authorizes the Food and Drug Administration (FDA) to collect user fees from companies wishing to submit their products for agency review. The intention is that the fees would be used to provide an additional revenue stream for FDA to hire more staff, improve systems, and establish a better managed drug review process to speed up the delivery of needed therapies to the public.

In his remarks, Mr. Perry stressed the importance of FDA increasing stakeholder involvement in the regulatory process to address any barriers that slow patient access to new therapies. He also emphasized the need for FDA to have the ability to play a more significant role in encouraging the development of new therapies for diseases like Alzheimer’s disease by providing guidance on streamlining clinical trial designs, qualifying biomarker for use in development, and assessing the benefits and risks of new therapies post-approval so as not to delay treatments for patients who may benefit from them. Lastly, Mr. Perry highlighted the fact that user fees cannot be the sole source of support for the FDA; they must be balanced with Congressional appropriations.

The PDUFA reauthorization process has historically been conducted through negotiations between the Agency and regulated industries, however the current round of negotiations are open to broader participation and transparency. The FDA will be seeking feedback from patient and consumer groups as well as the general public before entering into formal negotiations with industry and also before submitting its recommendations to Congress. If your organization would like to submit comments to the docket, they will be accepted until May 12, 2010. Information can be found in the Federal Register notice below.

 

Patient Groups Seek Funding for New Cures Acceleration Network

One component of the healthcare reform legislation signed into law by President Obama on March 23rd is the authorization of a Cures Acceleration Network (CAN) at the National Institutes of Health (NIH).  Championed by Senator Arlen Specter (D-PA), CAN’s aim is to reduce the delay that exists between scientific discovery and the translation of discoveries into life saving therapies.  Once established, CAN will be administered out of the Office of the Director at the NIH and create a new grant mechanism targeted at projects that are expected to more quickly move bench science into new treatments and potential cures for high need diseases and conditions.  CAN will also strengthen the coordination between all parties involved in the drug development process, including the patient community and the FDA.

On April 16th, a letter signed by patient advocacy groups, including members of ACT-AD, was sent to Congressional appropriators urging them to provide start-up funding for CAN.  The letter calls for $500 million in fiscal year 2011.  The requested level of funding will provide CAN with the resources necessary to establish itself as part of the NIH and begin the process of awarding grants that could enhance medical innovation and speed the development of life-saving therapies.

To read the letter on CAN appropriations, click here.