A rush to judgment on Alzheimer’s drug?
Imagine that your doctor has just told that you most likely have Alzheimer’s disease, an incurable type of dementia. And then you see on the news that the U.S. Food and Drug Administration (FDA) has approved a new drug for Alzheimer’s disease called aducanumab, made by a company called Biogen.
But, you also read, many doctors oppose the FDA decision.
Why would anyone oppose a drug for this terrible disease?
Over the past almost 40 years, as a doctor specializing in the care of older adults, I have cared for many patients with Alzheimer’s disease. I have done epidemiology research, but no drug research. I did serve, for about a year, on a “Patient Journey Working Group” for Biogen. This working group did not discuss the drug but rather how primary care doctors might recognize early dementia in their patients and refer them to specialists as needed. I had no inside knowledge about Biogen’s drug trials; I only know what I have read in the medical literature and the newspapers.
Like everyone in my profession, and every Alzheimer’s patient and family member, I really want there to be a safe and effective treatment for Alzheimer’s disease.
Like most others, I was hoping aducanumab might finally be that treatment. Aducanumab looked promising because it removes an abnormal protein called amyloid which is present in the brains of people with Alzheimer’s disease.
Sadly, it has not been shown to improve, or even restore, mental functioning.
Why not? There are many theories, but just because amyloid is present in the brain, removing it may not cure the disease.
Because the drug does not improve memory or other mental functions, the FDA’s advisory committee voted against its approval.
However, the FDA overruled its own advisory committee and approved the drug. They instructed Biogen to conduct a new study and report the results within nine years. But in the meantime, the drug will be available for doctors to prescribe to patients.
Understandably, the FDA’s decision has been hailed by drug manufacturers, and by some patient advocacy groups. There have been questions about potential conflicts of interest and an investigation is underway at the FDA. But that is not the only concern.
Patients and doctors understand FDA approval to mean that all evidence has been reviewed by independent experts, and that a drug is safe and effective for the conditions for which the FDA approves it, with the listed precautions.
Some feel that all possible treatments should be approved without bureaucratic interference.
Given how awful Alzheimer’s is, and how desperate patients and families are, isn’t any hope better than no hope? Why not let the market decide whether drugs are safe or effective, as it does with over-the-counter sleep aids and vitamins which the FDA does not regulate?
Let’s see how far we can stretch this argument.
Here is a drug which does not improve mental function, even in the manufacturer’s own studies.
The company re-analyzed its own data and found a group of patients who had received a high dose (not part of their original plan) went downhill a little more slowly than others. Many people questioned this re-analysis.
To participate in that study, patients had to meet several conditions. Their dementia had to be very mild, they should not have experienced a stroke or TIA (mini-stroke) in the past year, and have no poorly controlled diabetes, high blood pressure or psychiatric illness; no hepatitis or HIV infection; no alcohol or substance use disorders or use of marijuana or other cannabinoids; and no bleeding disorders or most cancers in the past five years. Only people free of all these health problems were allowed to be in the trial.
But the FDA did not restrict its approval of aducanumab only to such people.
To receive aducanumab, people will need to undergo a brain MRI scan, followed by a PET brain scan using an injected radioactive agent to look for amyloid.
Depending on those test results, patients can receive aducanumab intravenously once a month.
Because of a risk of brain swelling and small bleeds in the brain, people taking the drug have to undergo regular MRI brain scans to monitor for these side effects.
Biogen has estimated that taking aducanumab will cost $56,000 per person every year. Payment will presumably come from Medicare and commercial insurance into which we all pay our monthly premiums.
Meanwhile Biogen has begun its direct marketing campaign. Their ads warn the public that people who have been losing their train of thought, or misplacing their keys, might have Alzheimer’s disease and should ask their doctors about a new drug.
The public can also click on a link to find local experts in their areas, which often turn out to be specialists who run intravenous infusion centers.
Even before the FDA approved the drug, Biogen started sending doctors, including myself, unsolicited offers of “educational” programs to enhance our understanding of Alzheimer’s disease. Seems like a strategy which came out of that Patient Journey Working Group.
There are no bad guys here. Biogen invested a lot of money to develop aducanumab and expects to recoup its investment and turn a profit.
So, Biogen will do whatever it legally can to obtain FDA approval, and to get people to buy their product. Everybody wants a cure for Alzheimer’s disease. But we count on the FDA to put the brakes on runaway emotion and enthusiasm by objectively weighing risks and benefits.
What if the FDA gets caught up in the same wave of enthusiasm and feels pressured to approve a drug?
Next the Centers for Medicare and Medicaid Services must decide whether to approve payment for aducanumab, and if so, for whom.
Whether to ask for aducanumab will still be an individual decision for each Alzheimer’s patient and his or her family. To make that decision I hope they will consider all the information and ask their doctor the most important question: Will it make me better?