An 80-year-old successful and active businessman came to see me because of mild memory problems, and was afraid he had early Alzheimer's disease. Before he stepped into my office he'd already been considering major life-altering questions such as "Do I now need to quit the lifelong work that I love?," "how soon will I be disabled and demented?," and "how will my family take care of me if I have Alzheimer's?"
People like my patient are said to have "mild cognitive impairment," or MCI. About 50 percent of people with MCI have Alzheimer's disease and will progressively get worse, ultimately dying from the disease. But 50 percent of people with MCI don't have Alzheimer's, and some may get better with proper assessment and care. So accurate diagnosis is critical to prognosis, treatment and planning for people with MCI.
Imagine if I could do a test that would tell me whether he actually had Alzheimer's disease in his brain! Up until 2012, the only way to do this was to wait for the autopsy, or do a brain biopsy -- not very practical solutions.
However, in 2012, the US Food and Drug Administration (FDA) approved a diagnostic brain imaging test called Amyvid™ for the detection of amyloid in the brain, the hallmark of Alzheimer's. It enables doctors to much more accurately diagnose Alzheimer's without the need for a brain biopsy or autopsy. Most experts believe the test has significant clinical value.
Without it, a misdiagnosis of Alzheimer's is possible, perhaps up to 30 percent of the time, and especially in people with MCI. If positive, the test has important prognostic value (like many other FDA approved tests) and helps physicians counsel people with MCI while they're still able to prepare for challenges that lie ahead, and identify appropriate and FDA-approved treatments for their symptoms once they progress to dementia (usually within 3-5 years). If negative, doctors can seek other, potentially reversible causes of the MCI and recommend measures to manage risk factors that may improve cognition.
Fortunately, my patient could afford the Amyvid scan, which is not currently approved for payment by Medicare or insurance. His scan was negative, so prognosis was probably much better! He and his family were extremely grateful for the news. The patient continued his work, remained engaged in life, and his condition actually improved with the lifestyle and medical recommendations that I made.
But for most people, Amyvid is simply too expensive.
Unlike the FDA, which approved Amyvid as "safe and effective" in identifying beta-amyloid in the brain, the characteristic sign of Alzheimer's disease, Medicare approves payment for tests like Amyvid. In considering payment, Medicare seeks to determine the "value" of the test to individuals and society. Unlike FDA, Medicare is not asking the question, "is Amyvid safe, and does Amyvid do what it is reported to do, that is identify amyloid in the brain?" In contrast, Medicare is asking, "does the Amyvid scan change health care outcomes?" And while Medicare cannot decide to approve or disapprove payment for a test solely on the basis of the cost, in considering the "value" of a test, they can consider whether it is "cost-effective" from a societal perspective. No doubt Medicare is concerned that many people over the age of 55 with memory issues will want the test, and that appropriate use of the test (such as in people with MCI) will be difficult to control in clinical practice.
So, Medicare has not approved payment for the test in clinical practice. This means most people won't have access to it. Most experts, including those testifying to Medicare, responded that when used appropriately according to the guidelines suggested by experts for use in individual patients, Amyvid clearly has benefit for certain patients in accurate diagnosis, prognosis, and clinical care (such as the appropriate use of Alzheimer's medications), and that a negative test can have considerable value as well (as it was for my patient and his family).
Medicare has proposed to only approve payment in research studies through a mechanism called "coverage with evidence development" or CED. This means that Amyvid will need to tested in large, lengthy and expensive research studies to prove its "clinical value." No doubt this will take years and millions of dollars, the studies will be difficult to conduct and demonstrate effects on populations of patients, and ultimately if the value of the test is not shown, payment will be denied. In the meantime, the vast majority of people will not be able to get the test.
The potential for actual use of new diagnostic tests in improving the quality of healthcare, and reducing overall costs is important. The question, the problem, is when should these data be obtained? Prior to FDA regulatory approval? Prior to Medicare approval for payment? Or after approval, so that individual patients can have access while the societal cost-effectiveness of the test is being studied (which can take many years)?
The case of Amyvid also has repercussions for the drug discovery and development process. With no treatments available to prevent or slow Alzheimer's, the only way to halt the epidemic is to invest in new research for new drugs and diagnostics. This requires 12-15 years of research and billions of dollars. The high research and development costs create tremendous risk and the reality is that most drug research programs fail.
From a research and development perspective, there is little incentive for researchers, investors, or a pharmaceutical company, to spend billions of dollars on the risk of development of a new therapy or test that may never be paid for, even if approved by the FDA. The possibility of great risk in payment, after decades of research and billions of dollars, will clearly hinder innovation in the development of new drugs and diagnostic tests.
These conflicts in perspectives between Medicare, healthcare providers, patients, industry and researchers that develop tests like Amyvid is a harbinger of our new world under healthcare reform, and will increase dramatically in the coming years. And as the largest payer in the United States, the insurance industry generally follows Medicare policy for the elderly, so the implications are for everyone.
We have to find a way to make the criteria and the process for approving payment more effective, efficient, and collaborative, while effectively balancing the needs of individual patients and society. Current quality of care and future research on the development of new diagnostic tests and drugs depend on it.
This can be done by approving tests for payment so patients and physicians have access under specific conditions with clear guidelines, in conjunction with procedures for monitoring the appropriate, approved utilization of the test or drug. For patients with dementia, this is already being done with a test like Amyvid called FDG-PET to distinguish those with fronto-temporal dementia from people with Alzheimer's disease.
We urge Medicare to work collaboratively with patients, doctors, and the research industry to make decisions that are beneficial to both individuals and society, as we transition from the individual driven, fee-for-service system to what is essentially managed care and population-based health under healthcare reform.