The FDA and Aduhelm: WTF?


  • Jefferies analysts found doctors have shown strong interest in prescribing Aduhelm for about 35% of early-stage Alzheimer’s patients with mild cognitive impairment after surveying 50 U.S. neurologists or psychiatrists currently treating about 12,000 Alzheimer’s patients. Source: Fierce Pharma
  • If that enthusiasm pans out in the long run, it could mean at least 300,000 patients taking the drug, or $10 billion to $15 billion in peak Aduhelm from U.S. sales alone, which is above consensus estimates of around $7 billion to $8 billion, the team said.
  •  One agency FDA adviser, resigning from his committee post in protest, called it “probably the worst drug approval decision in recent U.S. history.” Source: STAT News
  • Our healthcare system can’t continue to support drugs that don’t have good clinical outcomes behind them.

In my career in healthcare marketing, I have done my best to understand the “science” behind certain health conditions along with how some medications can help. When I didn’t understand something, I went to the medical people on the team to gain insights. I always thought if there were questions about efficacy or treatment, we should be overly cautious and inform our customers (patients). Therefore, I find it hard to understand how so many physicians could show “strong interest” in prescribing a drug with so many questions vs. answers.

The story of how a drug that’s never even been proven to work is now poised to rake in billions of dollars for its manufacturer, impose needless suffering on millions of families, and destabilize Medicare and Medicaid in the process, goes well beyond the failures of what should be the toughest regulatory body of the U.S. government. Aduhelm is far from the first $55,000 flop put forth by the toxic American combination of a highly commodified pharmaceutical industry and disjointed financing stymied by private interests. Without a total overhaul of this dysfunctional status quo, it won’t be the last.

Natalie Shure, The New Republic

Biogen’s Aduhelm won FDA approval after discovering that some patients on higher dosages had displayed improvement of .39 points on an 18-point scale that measures cognitive abilityThe finding was far lower than the one to two points generally considered to be statistically significant improvement. What’s more, 40 percent of those subjects suffered side effects, including severe brain swelling.

That might be tolerable if the drug actually worked. Alzheimer’s really is a devastating diagnosis, and if Biogen had actually produced a game-changing product to slow its worst symptoms, it would have an immeasurable impact on millions of lives. While there’s no indication that this drug delivers anything close to this standard, it will still be immensely difficult for desperate patients, along with their families and physicians, not to roll the dice on Aduhelm—even if it means that the patients involved will spend the bulk of the lives they have left undergoing what’s likely to be a futile and risky intervention on behalf of a drug company that couldn’t get the result it sought at trial.

Aduhelm joins a group of drugs that potentially leave patients worse off and much poorer for it. Recall that with this drug, about 30–40 percent of study participants experienced either brain swelling or bleeding. Not to mention the $56,000 annual price tag.

According to STAT News “Patrizia Cavazzoni, director of the FDA’s drug division, in an interview with STAT on Tuesday. “We think the decision is on very solid ground, that we are on very solid ground when it comes to the data and the rationale for utilizing accelerated approval to greenlight this drug.”

While it’s not clear that Aduhelm actually slows down Alzheimer’s, there was “convincing evidence” that it had a “robust” effect on amyloid plaques. OK the amyloid hypothesis has never been universally accepted or proven. A common counter-argument to the amyloid hypothesis is that plaques are found in the brains of many elderly people with normal cognition. So far, no method of breaking down/removing amyloid plaques has been shown to prevent symptomatic Alzheimer’s disease in humans.

I, therefore, find the FDA’s decision to be puzzling. Why didn’t they listen to their advisory panel? Why were the people on the advisory panel so convinced this drug should not have been approved?

According to JAMA, there are a LOT of drugs that have been approved by the FDA that doesn’t show a statistical improvement in outcomes. The FDA needs an overhaul from top to bottom to protect patients.