The U.S. health care system lacks the capacity to rapidly move a treatment for Alzheimer's disease from approval into wide clinical use, a shortcoming that could leave millions of people without access to transformative care if such a breakthrough occurs, according to a new RAND Corporation study.
The primary problem is that there are too few medical specialists to diagnose patients who may have early signs of Alzheimer's and confirm that they would be eligible for therapy to prevent the progression of the disease to full-blown dementia. Other shortcomings include a relatively low number of specialized diagnostic scanners and too few infusion centers to deliver treatments to patients.
Researchers estimate that as many as 2.1 million patients with mild cognitive impairment could develop Alzheimer's dementia over a two-decade period while waiting for evaluation and treatment resources after approval of an Alzheimer's disease-modifying therapy by the federal Food and Drug Administration.
"While significant effort is being put into developing treatments to slow or block the progression of Alzheimer's dementia, little work has been done to get the medical system ready for such an advancement," said Jodi Liu, lead author of the study and a policy researcher at RAND, a nonprofit research organization. "While there is no certainty an Alzheimer's therapy will be approved soon, our work suggests that health care leaders should begin thinking about how to respond to such a breakthrough."
An estimated 5.5 million Americans live with Alzheimer's dementia today, with the number projected to increase to 11.6 million by 2040.
Advanced clinical trials are underway for at least 10 investigational therapies that have shown promise in slowing or blocking development of Alzheimer's disease. Researchers say the progress provides guarded optimism that a disease-modifying therapy could become available for routine use within a few years.
Liu and her team examined the pathway patients would likely take to receive an Alzheimer's therapy and created a model to simulate the pressures that such an approved therapy would put on the health care system.
The analysis assumes that a therapy is approved for use beginning in 2020 and screening would begin in 2019, although researchers stress that the date was chosen only as a scenario for the model, not as a prediction of when a therapy may be approved.
Under such a scenario, about 71 million Americans aged 55 and older would have to be screened for signs of mild cognitive impairment. After follow-up examinations and imaging to confirm evidence of Alzheimer's, the RAND analysis estimates 2.4 million people ultimately could be recommended for treatment.
Researchers then analyzed how the activity might challenge the current health care system's supply of dementia specialists, scanners used to identify Alzheimer's abnormalities in the brain, and access to infusion centers that would deliver the treatment.
They found shortcomings in the capacity of all three areas of the health care system to cope with a surge in demand triggered by an initial Alzheimer's therapy. Wait times to complete all three stages of the diagnostic and treatment phases would be 18.6 months in 2020, shrinking to 1.3 months in 2030 as the system develops more capacity and the surge in demand declines.
The analysis suggests several measures that could be taken to prepare for advent of an Alzheimer's therapy.
To account for a shortage of neurologists and geriatricians, researchers suggest primary care physicians or mid-level providers such as nurse practitioners be trained to conduct initial screening for mild cognitive impairment. In addition, some primary care physicians and specialists such as general psychiatrists could become certified in dementia care to allow them to provide advanced testing and treatment. Use of telehealth also may play a role, researchers say.
Researchers suggest intensifying efforts to develop alternatives for PET scans, which are now the only FDA-approved diagnostic to confirm brain changes caused by Alzheimer's. Options include further refining experimental tests that diagnose the illness using cerebrospinal fluid, as well as using mobile PET clinics to reach more patients.
The third potential bottleneck is a shortage of infusion centers where patients could receive medications. Most of the treatments under development for Alzheimer's are biologic drugs that must either be injected or infused. While infusion centers may be able to ramp-up capacity quickly, a better option may be a combination of some clinic expansion, treatment in physician offices and home infusion. Such an approach might be able to meet the needs more quickly and at a lower cost, according to the analysis.
"Addressing the capacity constraints may turn out to be as challenging as developing an effective treatment for Alzheimer's," said Dr. Soeren Mattke, the study's senior author and managing director of RAND Health Advisory Services. "But it is important to begin the discussion among stakeholders about how to start addressing the obstacles in a timely manner."
The research was sponsored by Biogen, a biotechnology company that is working to develop a treatment for Alzheimer's disease.
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