Biogen’s Alzheimer’s drug Aduhelm has recently secured FDA approval to much fanfare. But do the benefits of the treatment justify the weighty $56,000 per year price tag?
When it comes to the treatment of Alzheimer’s disease, up until now the options have been severely limited. At least 50 million people worldwide are believed to be living with this fatal neurodegenerative brain disease or other dementias, with an overall economic impact of over US$1 trillion.
The condition is characterised by progressive accumulation of beta-amyloid protein plaques and neurofibrillary tangles which damage neurons, leading to the decline of cognitive and physical functioning. Symptoms include impairment of memory, language and visuospatial function which eventually lead to the need for around-the-clock care. The average life expectancy of Alzheimer’s patients is four to eight years while the emotional and financial strain on caregivers – in most cases unpaid family members and friends – accumulates rapidly over this period.
So the US Food and Drug Administration’s approval of Biogen and Eisai’s Aduhelm (aducanumab) on June 7th was on the face of it fantastic news for the Alzheimer’s community. This human monoclonal antibody, which is administered via an intravenous (IV) infusion every four weeks, stimulates the immune system to target and eliminate beta-amyloid plaques in the brain.
It should be noted that the decision was made not without controversy; three members of the FDA’s advisory panel immediately resigned after the decision was made.
“We believe this first-in-class medicine will transform the treatment of people living with Alzheimer’s disease and spark continuous innovation in the years to come,” Biogen chief executive Michel Vounatsos proclaimed after the approval was sealed.
The biotech had spent years developing the treatment, which looked at one stage in late 2019 like joining the industry’s long list of investigational drug failures after disappointing and conflicting results in two Phase 3 clinical trials (ENGAGE and EMERGE).
The FDA approval sent Biogen stocks soaring as investors bought into the potential for it to become a blockbuster generating billions of dollars in annual sales. This was likely in no small part to Aduhelm being approved not just to treat patients afflicted with early onset of the disease but all Alzheimer’s patients.
Based on patients with mild dementia symptoms, Biogen has estimated the target population for Aduhelm at around 1-2 million people, but the FDA decision could technically increase it to a huge 5.8 million, the number of adults aged 65 and over in the US with Alzheimer’s.
The drug does not come cheap. Biogen has set the price at US$56,000 per year, which came way above analysts’ expectations. In a research note, analyst Brian Abrahams at RBC Capital Markets described Biogen’s price tag as “far above where we believe most investors had speculated,” adding that this type of pricing is more in line with specialty medications for diseases that affect hundreds of thousands of patients per year, rather than common diseases like Alzheimer’s.
Cost-benefit analysis
The high price has stimulated further close examination of where Aduhelm stands on the cost-benefit axis.
In its revised evidence report for Aduhelm, the independent non-profit research group, the Institute for Clinical and Economic Review (ICER), confirmed that in both the trials and at all doses, the drug effectively removed beta-amyloid.
According to the ICER report, while the EMERGE study did reveal a possible positive treatment effect, “a minimal clinically important difference in Clinical Dementia Rating Scale has not been clearly defined.”
“Furthermore, there is disagreement about whether the degree of improvement seen in EMERGE is clinically important, and the relationship between clearance of beta-amyloid in the brain...
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