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Stakeholders meeting in Washington, D.C., discussed funding, costs, challenges, and directions.
A dozen promising trials of Alzheimer’s disease could be launched today if funds were available, said Paul Aisen, MD, director of the Alzheimer’s Disease Cooperative Study at the University of California, San Diego. “We know how to pick the drugs. We even know what designs we would use for the trials. But these are expensive and we don’t have the money.”
With a tsunami of future Alzheimer’s cases facing the nation and a number of recent drug trials proving unsuccessful, an October meeting in Washington, D.C., sponsored by the Pharmaceutical Research and Manufacturers of America (PhRMA) and others, focused on how to better fund and accelerate research.
The clinical trials he referred to, said Aisen, would focus on presymptomatic patients and emphasize “anti-amyloid drugs in combinations, but extending to unrelated therapeutic approaches.” The nation would be able to fund all that with $2 billion, he said.
According to the Alzheimer’s Association, spending on Alzheimer’s disease by the National Institutes of Health totaled about $484 million in fiscal year 2013, the equivalent of $100 “for every $29,000 Medicare and Medicaid spends, caring for individuals with Alzheimer’s.”
Data from the Kaiser Family Foundation indicate that as of 2011, long-term care, much of it related to patients with Alzheimer’s disease, already accounted for approximately 30% of Medicaid expenditures.
The Alzheimer’s Association estimates that costs associated with the disease for Medicare and Medicaid beneficiaries will climb from $122 billion in 2010 to $344 billion in 2030.
At present, according to PhRMA, only three new medicines for Alzheimer’s disease have been approved since 1998, resulting in a 34-to-1 ratio of “failures” to successes, although researchers stress that failures teach researchers a great deal.
Aisen also told meeting attendees that although the members of the Alzheimer’s disease research community have developed good communications with each other, there still is no “czar” or other infrastructure for coordinating the effort. This is another example, he said, of recommendations from the 2012 National Plan to Address Alzheimer’s Disease that have not been addressed.
Reed Tuckson, MD, consultant and former chief of medical affairs for UnitedHealth Group, warned that, days into the October government shutdown, the nation was already facing the next battleground for federal dollars. In a budget-cutting process that will be hard, fast, and ugly, he said, Alzheimer’s disease research advocates need to unite behind a strategically designed set of a few investigations, in spite of the fact that there is so much research to be done.
A key component for research success would be finding a connection between a clinical biomarker and a clinical outcome, said Nicholas Kozauer, MD, clinical team leader in the FDA division of neurology products. He emphasized that FDA is not in a position to approve a drug solely on the basis of a biomarker such as amyloid as a surrogate.
“If anything, we have evidence, at least from the dementia stage of the disease, that affecting amyloid doesn’t necessarily correlate with clinical outcomes,” he said.
But once there is reassurance a biomarker is clinically meaningful, “that is going to accelerate Phase II development significantly,” he said.
In Alzheimer’s disease, he said, where researchers look for small changes caused by treatment over time, being able to screen drugs more quickly will open up many avenues.
FDA released an Alzheimer’s disease draft guidance for developing drugs for early stage Alzheimer’s in February.
Neill Graff-Radford, MD, neurologist at the Mayo Clinic, urged that research into the effects of lifestyle factors should not be forgotten. There is powerful evidence, for example, that aerobic exercise and other factors may be helpful for the brain, actually increasing the hippocampus volume. What researchers don’t know is whether aerobic exercise will work in the setting of Alzheimer’s pathology, he said.
But a critical factor about Alzheimer’s disease, he said, is the fact that people are afraid of the disease, even more than they are of cancer. “If we knew that exercise could prevent Alzheimer’s disease, genuinely,” he said, maybe people at risk would actually do the exercise.
Robert Egge, the Alzheimer’s Association’s vice president for policy, said that advocacy for the disease now has a movement and a path forward. Positive indicators include President Obama’s mention of Alzheimer’s disease in the State of the Union address, the Department of Health and Human Services’ emphasis on Alzheimer’s in talking points about its proposed budget, and the resources NIH has directed to it from its discretionary funds.
Egge also noted there have been indications in Congressional budget statements that Alzheimer’s disease research has bipartisan support, a rare commodity these days, and there has been increased grassroots lobbying for it on Capitol Hill.
Kathryn Foxhall is a healthcare journalist based in the Washington, D.C., area.