I’ve talked before about the U.S. Food and Drug Administration’s (FDA) approval of a drug that slows the advance of Alzheimer’s for those in the early stages of the disease. The controversy that followed this approval was the decision by the Centers for Medicare and Medicaid Services (CMS) to make this approved drug available only to those who qualify and join a clinical trial (you can click here to read that earlier blog). My problem with that whole process was the fact that a civilian agency, with limited scientific expertise in drug development and manufacturing, was limiting access to this new drug. In the past the word rationing, when discussing healthcare policy, has been criticized as being inflammatory, but when I look at the definition, I think it absolutely applies in this situation. Rationing is defined as “the controlled distribution of scarce resources, goods, services, or an artificial restriction of demand”. When a drug is approved for use by the FDA and then the demand is artificially restricted, I would call that rationing. That’s what happened with Aduhelm, and it continues to experience restricted access. Well, I’m glad to report that a new Alzheimer’s drug has just been approved by the FDA, and (maybe because of the uproar you and I and others made about Aduhelm) CMS has not restricted this drug’s access to those in a clinical trial. They did, however, require the use of a registry to collect data.
This new drug is lecanemab (brand name Leqembi), and while it is one of the first FDA-approved treatments to favorably affect the underlying biology of Alzheimer’s disease, it is not a cure. It has shown to help the body to reduce the plaque that has accumulated in the brains of Alzheimer’s suffers. According to the FDA, the reduction in this plaque is reasonably likely to lead to a slowing in the clinical decline due to the disease. The clinical decline was slowed by 27% relative to the placebo group, over 18 months of treatment.
It should be pointed out lecanemab has only been studied in people living with early Alzheimer’s disease and mild cognitive impairment due to Alzheimer’s who showed evidence of a buildup of amyloid plaques. The therapy has not yet been tested on people with moderate or more advanced cases of Alzheimer’s or on individuals with other forms of dementia.
The drug does have some side effects which cause a small percentage of the trial participants to have some bleeding in small blood vessels in the brain. Most of this small percentage had no symptoms, and the situation resolved itself. There were, however, some who had longer term symptoms. This medicine should not be used on people that were prone to this type of problem, like people who were taking blood thinners.
While I’m always leery of any CMS restrictions or obstacles to access, the idea of using a registry to document results, if used in an efficient manner, may show that there is a possibility of expanded use of lecanemab to include those with moderate to advanced Alzheimer’s or those with other forms of dementia.
I can’t over emphasize the importance of these new drugs. This disease has never had any drugs approved that have slowed the onslaught of this terrible disease, and there is now some hope for those that suffer from Alzheimer’s and those that care for them. If the disease continues on with no cure or slowing of symptoms, it would cost our country’s healthcare system an estimated 1 trillion dollars by 2050. Which begs the question, “why are we passing legislation that will restrict innovation?”
If in 2050 we will spend 1 trillion dollars a year on Alzheimer’s caregivers and treatments, and in 2022 we spent 340 billion dollars, then we are talking at least 14 trillion dollars will be spent by 2050. It seems from just a financial point of view that we should be passing legislation that expands innovation, not restricts it. The impact on those with the disease and the American caregiver families from an emotional and physical point of view is the greatest reason for expanding research to find a cure or a treatment for Alzheimer’s.
It seems we are finally unraveling some of the secrets of this debilitating disease. As people begin taking this medicine, I think their results will help advance the understanding of what causes this disease and will spur more resources dedicated to finding a cure or a treatment for Alzheimer’s. I only hope that our lawmakers don’t continue to restrict investment in the research and development that will be required to make these life changing discoveries.
I’m very thankful for the hope these new drugs have given to those that suffer from Alzheimer’s and to those that care for them.