May 2012

User Fee Reauthorization Update

On May 24, the Senate passed its omnibus Food and Drug Administration (FDA) user fee legislation, the FDA Safety and Innovations Act (S. 3187) by a vote of 96-1. The Senate’s final version of the bill, introduced the week prior by Sens. Enzi and Harkin, replaces the version the Senate Health, Education, Labor and Pensions Committee readily approved last month. The new bill has a number of additions, including the authorization of foreign studies for drug and device approvals and a mobile health monitoring study group.

On May 30, the House of Representatives passed the Food and Drug Administration Reform Act of 2012 (H.R. 5651), its own version of the omnibus user fee legislation, by a vote of 387-5. Like the Senate bill, the House version includes measures intended to address prescription drug shortages, antibiotic incentives, improved access to accelerated approval pathways, expedited reviews for “breakthrough” therapies, modified Conflict of Interest rules, and an extension of Critical Path partnerships.

A number of issues will need to be agreed upon in conference, including compliance deadlines for the FDA and discrepancies over the review of new medical devices. It appears all parties are interested in passing the bill before the August recess, and the previous user fee agreements expiration in September.

To view the Senate bill, click here.

To view House bill, click here.

ACT-AD Represented at First-Ever FDA Patient Network Meeting on Risk-Benefit Analysis

On May 18, the Food and Drug Administration’s (FDA) Office of Special Health Issues (OSHI) in collaboration with the Center for Drug Evaluation and Research (CDER), Center for Biologic Evaluation and Research (CBER) and the Center for Devices and Radiological Health (CDRH), held the Inaugural Patient Network Annual Meeting – Patient Input into FDA Benefit-Risk Decision-Making: Opportunities and Challenges. The meeting was convened to discuss opportunities for FDA to enhance its regulatory decision-making, through the inclusion of patients and patient advocate groups during benefit-risk assessments of drugs and devices.

In order to reach a greater understanding of how patients define and perceive benefits and risks related to medical products, and integrate understanding into the regulatory framework, the FDA Patient Network framed the one day meeting around three major topics:

  • Review the drug and medical device regulatory processes
  • Discuss where patient input is practical and most valuable
  • Explore practical approaches to collecting meaningful patient input

The meeting included a series of presentations, exercises, and panel discussions to facilitate a conversation with the patient community about these topics. One such panel included ACT-AD Coordinator Cynthia Bens, who also serves as Director of Public Policy for the Alliance for Aging Research. Her remarks stressed the importance of considering disease severity and unmet need in FDA’s risk-benefit decision-making on early stage treatments for Alzheimer’s. Ms. Bens also highlighted that for chronic, progressive diseases like Alzheimer’s the views of caregivers on benefit and risk must be taken into account along with patient reports at every phase of the regulatory process.

At this meeting FDA announced that it will publish and seek comment on a list of 20 disease areas identified as priorities for more in-depth discussion on how to incorporate a full range of patient views into their work to develop tools weighing risks and benefits of medical products. The meeting was hosted in conjunction with the launch of the overarching FDA Patient Network program. The FDA Patient Network is a resource aimed at patients, caregivers, and advocates, that seeks to educate and convene stakeholders for the purposes of increased clarity and transparency on Agency actions. In addition to the annual meeting, the Patient Network will create a website, biweekly e-mail newsletters, and informational session for the sake of increased communication between FDA and advocates.

For more information on the meeting, click here.

For more information on the FDA Patient Network and their bi-weekly e-mails, click here.

NIA Holds Alzheimer’s Disease Research Summit

On May 14-15, the National Institute on Aging (NIA) hosted Alzheimer’s Disease Research Summit 2012: Path to Treatment and Prevention. The summit was convened as an initiative under the National Alzheimer’s Project Act (NAPA) to formulate an integrated multidisciplinary research agenda that will accelerate the development of successful therapies for AD across the disease continuum, and identify what types of resources/infrastructure and new public private partnerships are necessary to successfully implement this research agenda.

The Summit, supported by the ACT-AD Coalition, was attended by over 500 international researchers, clinicians and advocates. The topics that were discussed included the current understanding of AD, the need for more basic research into its pathobiology, existing models and approaches to drug development, and new ideas to speed the development of effective interventions.

The program’s six major sessions each concluded in a set of specific recommendations pertaining to their topic, but the Summit as a whole produced a set of recommendations that were recurring themes during the two days of discussions:

  • Recognize the heterogeneity and the multifactorial nature of the disease
  • Employ new research paradigms such as systems biology and network pharmacology
  • Enable rapid and extensive sharing of data, disease models, and biological specimens
  • Build new multidisciplinary translational teams and create virtual and real spaces where these teams can operate
  • Develop strategies to overcome intellectual property barriers to Alzheimer’s disease drug development
  • Develop new public-private partnerships
  • Establish a National Institutional Review Board (IRB) for Alzheimer’s disease clinical research

Also during the Summit, Health and Human Services (HHS) Secretary Kathleen Sebelius released the NAPA-mandated National Plan to Address Alzheimer’s Disease. The plan has five ambitious goals including the development of effective preventions and treatments for AD by 2025. Within the plan were a number of exciting initiatives to further research, caregiver training, public education, and awareness.

To view the National Plan to Address Alzheimer’s Disease, click here.

To view the Summit’s detailed initial recommendations, click here.

To view Day 1 of the Summit, click here, and here for Day 2.