February 2012

PDUFA V Reauthorization Process Advances to Congress

On February 1, the House Energy and Commerce Subcommittee on Health held its first hearing on the fifth reauthorization of the Prescription Drug User Fee Act (PDUFA). The PDUFA agreement was submitted to congress in January. During the hearing, FDA Commissioner Margaret Hamburg discussed a number of topics affected by the legislation including new fees, initiatives, and conflicts of interest for experts on advisory committees.

In her testimony, Commissioner Hamburg reiterated the importance of PDUFA to both FDA and Industry by noting that 62% of FDA’s approval operations are funded by PDUFA fees, with the other 38% coming from congressional appropriations. And with President Obama’s Fiscal Year ‘13 Budget proposing no increase to $2.5 billion appropriated in Fiscal Year ‘12, the remainder of FDA’s $4.49 billion will need to come from significant increases in the user fees paid by various regulated industries.

The FDA’s responsibilities continue to grow in the global market, and in order to adequately protect public health, the agency’s capacity will need to grow with it. In its current form, PDUFA would reportedly generate more than $299 million in fees for FDA over a five-year period starting in 2013. Some of that increase will come from new fees for the review of generic drugs, biosimilar drug applications, and for food establishment registration.

User fees, under past PDUFA agreements, have enabled the FDA to significantly increase review staffing for drug applications and new drug safety initiatives. Median review times for priority drugs have been cut in half and standard applications have been cut by 37%; all without compromising safety. PDUFA V will play an integral role in making sure innovation and the American people are not put in jeopardy.

For more information on the February 1 PDUFA hearing, click here.

Two-Year Bolus of Funding for Alzheimer’s Research Announced

The Department of Health and Human Services (HHS) recently announced it would be reprogramming $50 million from its current budget to Alzheimer’s disease (AD) research, and pledging an additional $80 million in the President’s Budget for Fiscal Year ’13. The President’s Budget includes an additional $26 million specified for caregiver support, provider education, and other goals set forth by the National Alzheimer’s Disease Plan. Combined, the administration is committing a total of $156 million in new AD research investments over the next two years.

The additional funds will help boost projects with great promise for both basic research and therapeutic applications like that of induced Pluripotent Stem (iPS) cells. First harnessed three years ago, iPS cells could increase the efficiency of novel AD therapy discovery and approval by allowing developers and regulators to test new therapies on cells made to mimic neurons with AD pathologies. The use of simulated AD cells would dramatically cut the cost and time of currently burdensome AD trials, speeding new therapies to those affected by the disease.

Other areas of scientific explorations that could quickly produce meaningful results are expected to be highlighted by the Advisory Council on Alzheimer’s Research, Care, and Services created under the National Alzheimer’s Project Act (NAPA).

To read more on this announcement, click here.

To view HHS’s official announcement, click here.

For more information on NAPA and its Advisory Board, click here.

NIA to Hold Alzheimer’s Disease Research Summit

This May, the National Institute on Aging (NIA) will be hosting Alzheimer’s Disease Research Summit 2012: Path to Treatment and Prevention. The Summit has been identified by HHS and the Chair of the National Advisory Council for the National Alzheimer’s Project Act (NAPA) as a key objective to identify research strategies and priorities to prevent and treat Alzheimer’s disease (AD).

As part NAPA’s National Plan, the Summit is the first in a series of workshops and conferences on research into AD and related dementias. The goal of the summit is to create a new translational, inter-institutional agenda for the development of successful AD therapies within the next 10 years. The Summit will bring together private and international partners in the hope of finding new pathways of prevention and treatment for AD, create new research teams, and look at ways to expedite translation of findings to clinical practice and the community.

The program will be organized around 6 major themes presented in consecutive sessions:

  • Interdisciplinary Approach to Discovering and Validating the Next Generation of Therapeutic Targets for AD
  • Challenges in Preclinical Therapy Development
  • Who to Treat, When to Treat and What Outcomes to Measure?
  • Non-pharmacological Interventions
  • Drug Repurposing and Combinatorial Therapy
  • New Models of Public-Private Partnership

Each session will be comprised of experts in different disciplines tasked with the integration of research areas, research expertise, and research resources for the purpose of optimizing scientific discovery and its translation to efficacious therapies for AD.

The Summit will take place on Monday, May 14th from 8 AM to 6 PM, and Tuesday, May 15th from 8 AM to Noon. It will be located in the Natcher Auditorium of Building 45 at the National Institutes of Health in Bethesda, MD.

The ACT-AD Coalition is a supporter of the event and invites all ACT-AD members to attend, but space is limited. Sessions are open and free to the public.

For more information on the Summit, click here.