By: Margaret A. Hamburg, M.D.
I had the opportunity yesterday to take part in the Alzheimer’s Association’s (AA) Advocacy Forum and engage in a public discussion with the organization’s President and CEO Harry Johns about the FDA’s role in helping advance Alzheimer’s disease research and the development of new treatment options and approaches.
This event recognizes the tireless efforts of a community determined to make strides in the fight against what is, in no uncertain terms, a devastating disease and world-wide crisis. Indeed, it is difficult to overstate the impact of Alzheimer’s disease on our society.
Only last month new research was published that suggests that deaths due to Alzheimer’s disease in the United States actually have been severely under-recognized. By these new estimates, Alzheimer’s disease may rank as the third leading cause of death, trailing only behind heart disease and cancer. What’s even more alarming is that it is the only cause among the top 10 without a way to meaningfully prevent, treat, or slow its progression.
Fortunately, in stark contrast to these disturbing figures, the commitment of the countless individuals entrenched in the fight against this epidemic remains resolute. The FDA shares in this determination and holds the need to facilitate the discovery of effective treatments for Alzheimer’s disease among its highest priorities.
In January 2011, President Obama signed into law the National Alzheimer’s Project Act (NAPA), which has the goal of creating a coordinated national plan to overcome the Alzheimer’s crisis. The FDA has been closely involved in the evolution of this plan and has taken several important steps toward realizing some of its stated goals.
In February 2013, the FDA published a draft guidance document responding to the shifting focus of the research community towards the earlier stages of Alzheimer’s disease. The hope is that intervening earlier in the disease process (before the onset of dementia) may provide a greater opportunity to alter the course of the condition. Given that these are uncharted waters, it is essential that we provide as much clarity as possible. Of particular note, the guidance discusses the possible use of the accelerated approval pathway as a means of approving drugs for patients in the earliest stages of the disease.
The FDA is also partnering closely with many public-private initiatives, advocacy groups and consortia in the Alzheimer’s community. As one example, the agency is a member of the recently announced NIH-led Accelerating Medicines Partnership (AMP), which is attempting to uncover biomarkers that may help predict clinical benefit in drug development. We are also working with the Coalition Against Major Disease (CAMD) to develop novel biomarkers to signal the onset of the disease and clinical-trial goals through the agency’s Drug Development Tool Qualification process, and have recently endorsed a clinical trial simulation tool they have developed. These represent just a few of our many collaborations within the Alzheimer’s disease field.
The Alzheimer’s Association used the occasion of their Advocacy Forum to honor the contributions of a number of individuals who have dedicated themselves to the vision of a world without the scourge of Alzheimer’s disease. We at FDA join in recognizing those who have made these important contributions and commitments. We also recognize and applaud the contributions of each and every member of the Alzheimer’s Association who have come to Washington this week to learn more, but also to educate policymakers through their experience, advocacy, and personal stories. Like all of them, we at FDA are committed to doing our part to meet the immense scientific and social challenges of this disease.
Margaret A. Hamburg, M.D. is the Commissioner of the Food and Drug Administration
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