Why was the FDA approval controversial?
The drug was approved through the Accelerated Approval pathway. The approval was based on evidence that Aducunumab (Aduhelm) appeared to reduce clumps of brain amyloid on special brain scans. However, the medicine did not improve memory or thinking. The official FDA advisory committee (physician experts) who reviewed all the data recommended against approval. This was because Aducunumab (Aduhelm) did not clearly improve memory or thinking. You can read some of this information here:
6 ways the FDA’s approval of Aduhelm does more harm than good
What is FDA accelerated (“conditional”) approval?
As explained in the FDA’s press release, accelerated (sometimes called “conditional”) approval is based on a “surrogate” or substitute marker. In this case, the FDA felt that the medication decreased the amyloid protein in the brain. However, it is unknown if this corresponds to change that is important to people in real life. For approvals based on these substitute markers instead of real-life change in memory and thinking, drug companies are required to conduct more research after the conditional approval to make sure the drug helps people’s life (memory and function). If the follow up studies don’t show that the drug helps daily life, it is removed from the market. This follow up study for Aducunumab (Aduhelm) is expected to take another 3 to 5 years.
Who should consider this medication?
The studies were done on people who had either early Alzheimer’s dementia or mild cognitive impairment (MCI) due to Alzheimer’s (with a positive special amyloid brain scan). MCI is when people have a decline in memory and thinking but it’s not bad enough to impact most parts of daily life. Alzheimer’s is only one cause of MCI and dementia. Thus, people interested in the treatment will probably need extra testing to show that they have too much amyloid. Also, since the studies of
Aducunumab (Aduhelm) were only in people with MCI and mild AD, it is expected that Medicare and insurance companies may only pay for the drug for people in these mild stages. People with worse memory problems may not improve with the medication or may have a higher risk of side effects.
What testing may be required?
The FDA requires an MRI brain scan to look for other causes of the memory problems and to serve as a baseline. Your insurance or physician may require a special brain scan to look at amyloid levels or a lumbar puncture (spinal tap) to look at amyloid levels. The medication will probably only help people with abnormal amyloid levels. Currently, amyloid scans are not covered by insurance companies, including Medicare.
How is this medication taken?
The medication is a one-hour intravenous (IV) infusion every 4 weeks.
What is the cost?
The manufacturer, Biogen, estimates the cost of the medicine to be $40,000 to $54,000/year. There will be other costs related to seeing physicians and going to the special centers where a person can get an IV infusion. Currently we do not know if Medicare or insurance companies will pay for the medication.
Insurance companies sometimes have stricter guidelines than FDA approval. Information about insurance coverage is available through Biogen Patient Support Services (they will calculate the out of pocket cost based on a person’s insurance (call: 1-833-425-9360).
Can there be side-effects?
Aduhelm may have serious side effects. About 4 in 10 patients receiving Aducunumab (Aduhelm) developed inflammation in the brain. Some people also had bleeding in the brain. Because of this, the FDA requires that patients receiving Aducunumab (Aduhelm) have an MRI scan before starting the medicine and after the 7th and 12th infusions. Emergency brain scans may be needed if a person suddenly develops new symptoms that could suggest a brain problem. The potential risks and benefits of Aducunumab (Aduhelm) must be weighed carefully for each person.
Does it work?
There are not clear benefits for memory and thinking based on the research we have right now.
Per the Alzheimer’s Association –
“More research will be needed to better understand the benefits, side effects and best application.”