December 2011

ACT-AD Holds Annual FDA/AD Allies Meeting on Phase II Trials

On December 9, 2011, ACT-AD hosted the fourth in a series of working meetings with senior representatives from the U.S. Food and Drug Administration, leading scientists, drug developers, and Alzheimer’s advocates to address critical issues affecting the development and review of new Alzheimer’s disease therapies.

The presentations, panels, and open dialog during the meeting focused on phase II experiences in current drug development programs and the potential application of scientific insights to support decision-making by drug developers and regulators in order to bring new treatments to market.

Topics raised at the meeting explored current areas of consensus for the use of biomarkers in Alzheimer’s disease; strategies for designing a clinical trial involving patients at different stages of dementia; and lessons learned from adaptive trials in cancer research that might be applied to Alzheimer’s drug development.

The meeting was organized by ACT-AD and co-hosted by two additional Alzheimer’s groups –The Cure Alzheimer’s Fund and Leaders Engaged on Alzheimer’s Disease (LEAD). More than 50 leading stakeholders in the Alzheimer’s community and representatives from the Food and Drug Administration and National Institutes of Health attended the meeting.

A transcript of the meeting will soon be available at www.act-ad.org.

Working Group of Experts Release Review on Biomarkers for Alzheimer’s Disease

A group of thought leaders in Alzheimer’s disease says the science is solid and the time has come for more widespread use of biomarkers in Alzheimer disease (AD) clinical trials, to speed the identification of effective disease-modifying treatments for this incurable disorder. Recommendations by the Alzheimer’s Disease Biomarker Expert Working Group, are published this month in a special issue of Neurobiology of Aging.

Co-chaired by Dr. John C. Morris of Washington University, St. Louis, and Dr. Dennis J. Selkoe of Harvard Medical Scholl, the Working Group systematically examined published research and determined the current status of biomarkers for AD, their potential for use in the design and conduct of clinical trials, and additional data that might be needed to fully establish their usefulness.

The Working Group advocates for the widespread use of biomarkers in AD clinical trials, noting some of the possible benefits: greater precision than clinical assessment tools in measuring dementia progression, the need for fewer participants, studied for shorter durations and reduced cost. The also can allow for selective enrollment of “relatively rapid decliners,” meaning fewer participants and less time to demonstrate treatment effect. They can also enhance confidence that patients are accurately diagnosed with AD and improve clinical trial design for disease-modifying therapies.

In addition to Drs. Morris and Selkoe, the following are members of the Working Group:

  • Paul S. Aisen, M.D. University of California San Diego
  • David M. Holtzman, M.D. Washington University School of Medicine
  • William E. Klunk, M.D., Ph.D. University of Pittsburgh
  • Eric M. Reiman, M.D. Banner Alzheimer’s Institute
  • Reisa Sperling, M.D. Harvard Medical School, Brigham and Women’s Hospital
  • John Q. Trojanowski, M.D., Ph.D. University of Pennsylvania
  • Clifford R. Jack, M.D. Mayo Clinic
  • Richard Mayeux, M.D. Columbia University Medical Center
  • Marilyn Albert, Ph.D., Johns Hopkins
  • Marc Walton, M.D., Ph.D., U.S. Food and Drug Administration * Non-voting member

The special supplement is available at http://www.neurobiologyofaging.org/.

ACT-AD Chair Speaks About Expedited Drug Pathways

On December 15, 2011, the Engelberg Center for Health Care Reform at the Brookings Institution hosted an expert workshop on Expedited Drug Development for Promising Therapies. This workshop brought together a diverse set of stakeholders who discussed existing tools for facilitating expedited market access, opportunities for improving and expanding upon these tools, therapeutic areas most in need of an expedited development pathway and how promising therapies can be defined. The second half of the workshop explored methodological approaches to enable a compressed drug development timeline and potential evidence gaps that may result in the post-market environment for products approved through an expedited pathway.

Discussion at the workshop helped to highlight some potential avenues for expediting patient access to promising therapies along with the practical implications of and challenges that may be associated with these approaches. ACT-AD’s Chairman, Dan Perry, was invited to speak about the Coalition’s experience in working with regulators on issues related to the development a review of Alzheimer’s disease therapies. He also highlighted in his remarks that the review of neurological products could be improved by additional clarity and transparency around the application of existing pathways like Fast Track and Accelerated Approval to new Alzheimer’s treatments.

The agenda and discussion guide from this workshop are now available. A workshop summary will be posted to this site in the weeks to come.