When the drug Aduhelm (aducanumab) was first approved by the FDA in June 2021, it was touted as a groundbreaking development. It was the first new Alzheimer’s treatment in nearly two decades, and the first to target the presumed root cause of the disease: amyloid-beta plaques in the brain.
Supporters and some patient advocacy groups made bold claims about Aduhelm. The Alzheimer's Association called the approval a historic moment and a "turning point in the fight against Alzheimer’s.”
Aduhelm was presented as a beacon of hope for the more than 6 million Americans living with Alzheimer’s.
The FDA used its accelerated approval pathway, based not on clear clinical benefits, but on Aduhelm’s ability to reduce amyloid plaques—a surrogate marker. This move was seen by supporters as innovative and compassionate, allowing early access while further data was gathered.
However, its approval was contentious due to mixed clinical trial results. For example, one study showed slight benefits, while another showed none—and significant safety concerns, including risks of brain swelling and bleeding. The FDA's decision, made against the recommendation of its advisory committee, led to resignations from panel members and sparked widespread criticism.
Despite an initial annual price tag of $56,000, Aduhelm faced limited adoption. Insurers, including Medicare, restricted coverage, requiring patients to enroll in clinical trials to qualify. By early 2024, Biogen discontinued Aduhelm due to poor sales and terminated the confirmatory trial necessary for full FDA approval.
The Aduhelm saga underscores several critical lessons: the importance of robust evidence before drug approval, the risks of prioritizing surrogate endpoints over clinical outcomes, and the need for transparency in regulatory processes. It also highlights the challenges of balancing hope for patients with scientific rigor, especially in diseases with limited treatment options.
Dr. Jerry Avorn calls the FDA’s approval of Aduhelm “disastrous” in his new book “Rethinking Medications: Truth, Power and the Drugs you take.”
Avorn, who is a professor of medicine at Harvard Medical School and chief of the Division of Pharmacoepidemiology and Pharmacoeconomics at Brigham and Women's Hospital in Boston, explains there are many drugs that make big promises, rack up big price tags yet deliver little, if any benefits to patients. Some drugs and supplements even cause physical harm.
Avorn also praises many pharmaceutical breakthroughs that can treat, cure or prevent illnesses with a power that was impossible a generation ago. Yet he points out that many American doctors and patients feel a profound unease about the effectiveness, safety and affordability of the drugs they take.
Guest:
Dr. Jerry Avorn is a professor of medicine at Harvard Medical School and chief of the Division of Pharmacoepidemiology and Pharmacoeconomics at Brigham and Women's Hospital in Boston.
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This discussion will be recorded on Tuesday, April 22, 2025.
