Alzheimer’s Disease was supposed to get a new opponent after Biogen Inc. received unexpected approval from the U.S. Food & Drug Administration (FDA) for its Aduhelm in June 2021. You can read this article here. There was instant hope now that the first new drug to treat Alzheimer’s Disease would become widely available. Then something happened. As the pricing details became available, and as the reports around the actual approval process and study details came out, the fight was on over whether or not Medicare should spend tens of billions of dollars.
Now that the drug is approved, Inflammation Guide wanted to see exactly how available this drug is and take a 360-degree view of the gig picture. We also wanted to see if the unexpected FDA approval is playing a role in the drug’s acceptance and availability.
Aduhelm was approved by the FDA to treat patients with early-stage Alzheimer’s rather than the entire patient base. The drug acts by removing the sticky amyloid protein from the brain. One issue which has made effectiveness weak for so many cognitive drug studies is that many scientists and researchers do not even know about the actual cause and best practices to go after Alzheimer’s Disease. Of the companies still working toward those treatments, there are multiple approaches being studied and some of those vary greatly.
The first look at the launch of Aduhelm indicates that a small portion of patients who are eligible to receive the drug are actually getting access to the drug. One aspect that is holding the drug back is health insurance reimbursement. There is more to the story beyond dollars and cents.
It would be easy to take one side and just point the finger here. Instead, it is important to look at all of the topics from both sides even when this disease is in critical need of treatment. Doctors have been in serious debate over just how effective Aduhelm is as a treatment for Alzheimer’s. This drug experienced swings during its drug trials, and at one point the drug looked like it was a failure. Then a re-review of the data put the drug back on the map, and then an unexpected FDA approval was announced.
The FDA approval news was surprising enough that it briefly added 70% to Biogen’s stock value. That is not necessarily our focus, and that instant gain has by and large corrected itself for the most part. Still, a move of that magnitude is large enough to signal that the “financial bets” from the stock market were not expecting an automatic approval.
Biogen has thus far not had massive sales of Aduhelm. While the drug was originally expected to be made available to a wider patient population, its $56,000 annual list price has become a serious deterrent. It is now expected that the decision on Medicare payments will be decided by federal officials in 2022.
The Cleveland Clinic and Mount Sinai hospitals have announced that they will not use Aduhelm to treat Alzheimer’s over efficacy concerns and over concerns about relationships with the regulators who approved the drug.
One additional issue holding back the wider usage is widely reported placement on a waiting list for the drug rather than immediate use. And one sad issue about wait-lists for cognitive patients is that the longer they have to wait the worse their symptoms will get. That also translates to lower drug efficacy because Aduhelm was approved by the FDA to treat patients with early-stage Alzheimer’s.
Unlike oral or topical medications, Aduhelm is administered by doctors and nurses in a monthly infusion. This also eliminates some patients.
While the efficacy of Aduhelm has been a hot debate, the potential side effects of the infusion have also been a under controversy. One side-effect was bleeding in the brain and two studies showed conflicting results. One study showed that Aduhelm did slow the decline of cognitive functions. One group did not show the same result.
Also at issue is that Biogen’s marketing approval was based on a follow-on study (Phase 4) to prove its efficacy. The concern here, particularly with mixed results in prior studies, is that the high-priced drug might be administered to many patients only to find that the drug could suddenly be halted. That study was shown to include more minority participation and will enroll Alzheimer’s patients over four years who will then be monitored for up to five years.
According to a report in the Wall Street Journal, an electronic health records provider called Epic Systems Corporation sent an email that showed less than 10 patients since June 1, 2021 have been recorded as having been given an infusion of Aduhelm. What is troubling about that figure is that their database is represented as tracking some 116 million patients around the country. Even brokerage firm “channel checks” by analysts are showing very few neurologists prescribing the drug even though half of those surveyed expect to prescribe it over the coming year.
The Centers for Medicare and Medicaid Services (or CMS) began its formal payment review only in July 2021. Its formal proposed reimbursement decision is not expected to come until January of 2022, with a final CMS policy announcement due by April of 2022.
According to prior reports from Biogen, the company itself has said that Medicare is expected to cover around 80% of the Alzheimer’s patient which could benefit from Aduhelm. That number was put between 1 million and 2 million people, a far cry short of the full Alzheimer’s base of patients in America. Even 1 million patients and a $56,000 list price would translate to $56 billion annually that Medicare would have to come up with.
An additional concern is that $56,000 may be an understated annualized cost for Aduhelm. An article from Fierce Pharma suggested that the $56,000 is more likely to be $61,000 to $62,000 per year based on actual patient weight. The standard vial is efficiently dosed based on the “average” patient weight. Thus packaging waste and higher costs may result when more than one vial is used based on the patient’s actual weight. The excess drug left over in a vial after treatment is discarded. That would translate to $61 billion to $62 billion for CMS to come up with rather than $56 billion annually.
Receiving FDA approval for Alzheimer’s disease will be a major victory for the company that can treat the disease. Even a mild treatment rather than a cure would still be great news for millions of Americans and patients around the world. This review will hopefully show both sides of the coin about why this treatment’s launch has so far not become a typical standard of care after FDA approval.