In a highly anticipated decision, the Food and Drug Administration (FDA) last week cleared Alzheimer’s disease drug aducanumab on an accelerated, conditional approval program. This is the first approved medication to slow progression of the disease and the first new medicine for the disease in nearly two decades. Simply put, this decision is a big deal. For patients and their families who live with Alzheimer’s disease, this is a groundbreaking moment. But this decision’s implications should be carefully considered.
Consequential decision
There are questions about the efficacy of the drug (which will be sold as Aduhelm):
- One positive trial: Biogen stopped both their clinical trials in March 2019 after preliminary data from two phase III trials suggested it would not show benefit; later, one was determined to show benefit based on a surrogate endpoint of beta-amyloid plaque reduction.
- Departure from FDA advisory panel: In November 2020, the 11-person FDA advisory panel (independent panel of experts) voted nearly unanimously that the data did not warrant approval and chided the FDA for even accepting the filing. It is exceedingly rare for the FDA to overrule a unanimous panel vote.
- Staying power: The FDA has asked Biogen to conduct a confirmatory trial; however, Biogen has 9 years to deliver the final results of this study. In other words, Aduhelm is here to stay.
Subsequent value of and access to Aduhelm will be closely watched
Biogen priced Aduhelm higher than analysts expected, a list price of about $56,000 a year. The company puts forth that the investment in treatment is justified, in part, by the $600 billion per year of direct and indirect costs associated with Alzheimer’s disease and other forms of dementia. Biogen has pledged to not increase the price for at least four years.
Payers, notably Medicare, will likely grapple with coverage policy for Aduhelm given its price and broad label: the drug has been studied only in patients with mild cognitive impairment (MCT) and mild dementia, but approved for treatment of all patients with Alzheimer’s disease
Implications
- Overall, this is good news for life science companies advancing therapies for devastating diseases – this provides additional precedent for FDA to approve drugs for devastating diseases based on surrogate endpoints but unproven clinical benefits.
- This shows the power of patient advocacy groups – The Alzheimer’s Association is thrilled. Their advocacy efforts have yielded results.
- Provider uptake is uncertain. An opinion leader wrote last week that he would not prescribe aducanumab to his patients even with FDA approval, due to risks of small bleeds in the brain, financial cost and unclear benefits.
What life science companies can do
- Evaluate the data and announcement milestones for pipeline drug candidates and appropriately work in explanation on data, outcomes, safety and value. Expect heightened awareness and scrutiny.
- Harness relationships with key patient advocacy groups and influential stakeholders early, prior to approval—and be prepared to show through the patient lens how companies are realizing value.
