Aduhelm is the first Alzheimer's medication approved by the FDA since 2003.
According to the influential Institute for Clinical and Economic Review (ICER), the FDA "has failed in its responsibility to protect patients and families from unproven treatments with known harms" in approving Aduhelm.
The FDA, which announced the approval on June 7, said that the drug was the first to treat the underlying mechanism in Alzheimer's. But ICER didn't see it that way.
ICER argued that Aduhelm was effectively approved on the strength of a biomarker – removing amyloid from the brain – rather than clinical outcomes, which is puzzling as other drugs have been shown to remove amyloid from the brain yet failed to help patients.
In a statement, ICER said the view of many experts was that current evidence is not enough to support the use of Aduhelm in Alzheimer's patients, and the FDA had "moved the goalposts" by not holding Biogen's drug to the standards of other therapies.
"The avenue forward has seemed clear. Another study would be needed to reduce the substantial uncertainty about the drug's effectiveness, a requirement of even greater priority because of the drug's common and potentially serious side effects," said the ICER, which deliberates on the cost-effectiveness of new medicines.
ICER pointed to brain swelling and potential bleeding seen in approximately 30 percent of patients treated with Aduhelm. (Related: Alzheimer's breakthrough: scientists discover omega-3s override the bad gene causing Alzheimer's.)
Experts also questioned the FDA's decision to approve the drug for all Alzheimer's patients, rather than just the patients with mild cognitive impairment (MCI) and mild dementia – which are included in Biogen's studies of the drug.
Dementia specialist Robert Howard of University College London (UCL) said that the FDA has "sidestepped available clinical trial outcomes data that indicate the drug probably doesn't work."
"FDA approval closes the door on further placebo-controlled trials of aducanumab that might have helped to resolve disputes about efficacy quickly and cleanly," Howard said. "Now, we'll wait a decade before it becomes obvious to everyone that there are no benefits – only high healthcare costs – associated with the treatment."
Meanwhile, Biogen has been taking flak over its decision to price Aduhelm at $56,000 per year. ICER said a fair price would be in the $2,500 to $8,300 range while analysts at Evercore ISI expected it to be set at around $10,000.
Michel Vounatsos, Biogen's chief executive, defended Aduhelm in an open letter on the company's website.
The company has "engaged extensively with health economists, public health experts and payers about Aduhelm – and we have examined other recent biologic drug innovations," Vounatsos said. "Consistent with our pricing principles, we have established a price for Aduhelm that reflects the overall value this treatment brings to patients, caregivers and society – and one that will enable continuous innovation."
Vounatsos recently told CNBC that the drug's price will allow the company to further invest in its pipeline of medicines for other diseases. Vounatsos stressed that it was time to "invest" in Alzheimer's treatment when asked if he expected pushback from patients for the high price of Aduhelm.
Company executives said the total price figure for the new treatment is "substantiated" by the value it is expected to bring to patients, caregivers and society. They insisted the price is "responsible," noting the disease costs the U.S. billions each year.
More than 6 million Americans are living with the disease, according to estimates by the Alzheimer's Association. The company said it currently has the capacity to provide 1 million patients with the drug annually, with more than 900 sites in the U.S. ready to implement the new medicine. (Related: Coming wave of Alzheimer's patients threatens to bankrupt U.S. health care system, says leading Alzheimer's doctor.)
Biogen has committed not to raise the price of Aduhelm over the next four years and said it has agreed to access deals with some payers – including the Veterans Health Administration, CVS Health and the National Association of Free and Charitable Clinics (NAFC) – while negotiations are ongoing with Cigna and other groups.
Analysts at GlobalData think that Biogen will have challenges rolling out Aduhelm in the U.S., but are still predicting sales of $5.6 billion in 2027. Shares of Biogen surged as high as 60 percent following FDA's announcement.
Aside from the high price, another issue is its dosing by monthly infusion, as clinics that can provide this are not available in all areas. Side effects such as amyloid-related imaging abnormalities (ARIAs) could also be a barrier to uptake, according to Alessio Brunello, managing analyst at GlobalData.
"Physicians will also be obliged to do regular MRIs on patients to see if they have ARIA reactions, which could be a limitation, as repeated MRIs represent an additional economic burden," Brunello said.
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