Two major U.S. health systems announced in mid-July that they will not administer an expensive, newly approved drug to treat Alzheimer's disease.
That action, by the Cleveland Clinic in Ohio and Mount Sinai Health System in New York, once again focuses attention on a fundamental question: Most of us already know that Americans pay the world's highest prices for prescription medications, but are they worth the sometimes-exorbitant prices drug companies charge for them?
The drug in question, Aduhelm, was granted accelerated approval on June 7. It was approved even though 10 of 11 members on a neurology advisory panel said there was not enough evidence that the drug could slow cognitive decline. The drug is expected to be priced at $41,000 for the first-year of therapy and $56,000 for subsequent years.
In the wake of Aduhelm's approval last month, three of the advisory panel members resigned. Among them was Dr. Joel Perlmutter of Washington University, a leading Alzheimer's researcher.
Aduhelm is the first new Alzheimer's drug approved since 2003. It has been shown to reduce a brain protein called amyloid. People with Alzheimer's disease have a buildup of amyloid plaque in their brains. The hope for decades was that reducing the amyloid plaque would improve cognitive function. Unfortunately, that hope hasn't been realized.
Aduhelm isn't the first experimental drug to reduce amyloid plaque. But those earlier drugs have not been shown to improve cognitive function. Two clinical trials of Aduhelm were stopped early because independent reviewers determined that they did not appear to be helping patients taking the drug.
"Based on the current data regarding its safety and efficacy, we have decided not to carry Aduhelm at this time," officials at the Cleveland Clinic said in a statement.
For a widely feared disease like Alzheimer's or cancer, the lure of a newly approved drug can be irresistible. Desperate patients and their families can abandon therapy with older drugs that might work simply because of the hype surrounding a newer drug. Hope is important for people with cancer and Alzheimer's disease, but not more important than effective clinical therapy.
The Affordable Care Act set aside funding for what's known as comparative-effectiveness research. Such research is used around the world to assess the value of new treatments, drugs and devices and to set prices based on that value. Unfortunately, opposition to the bill has limited comparative-effectiveness research.
The approval of Aduhelm is already the subject of an investigation by the Food and Drug Administration's independent inspector general. Since approving the drug for widespread use, the FDA has since dialed back, restricting the drug to people with mild cognitive impairment. Further studies of the drug are continuing.
It's clear that patients don't benefit from expensive new medications that produce no clinical improvement, especially when those medications cost as much as a new Mercedes-Benz. It's also clear that the FDA's approval of Aduhelm was hasty and deeply flawed.
FROM AN EDITORIAL IN THE ST. LOUIS POST-DISPATCH