JAMA Internal Medicine published three Viewpoints on the recent approval of aducanumab online, along with an editorial introduction.
In the first Viewpoint the three FDA officials that were responsible for the approval defended their action. The FDA officials argue that a delay in approval would have resulted in loss of brain function of patients who would benefit from early treatment. They felt this was justified due to the demonstrated reduction in amyloid-plaque, which was shown to decrease the decline in cognitive function. Because aducanumab received an accelerated approval, it was judged to meet criteria to likely provide a benefit for a disease without a current treatment. There was no discussion of the decision to approve the drug for all patients and not just those with mild disease, nor the long timeline for a confirmatory study.
The second Viewpoint, written by two JAMA Internal Medicine editors and an academic physician. This article examines the impact of the aducanumab’s cost on Medicare’s budget. The authors estimate patient out-of-pocket costs of up to $11,000 per year and the potential of increased premiums for Medicare Part B. Medicare expenditure for all drugs covered under Part B drugs was $35 billion in 2018. If just 17% of the 5.8 million Alzheimer patients on Medicare received aducanumab, the cost would be $57 billion per year.
Three academic researchers wrote the third Viewpoint. The group included one of the physicians that resigned from the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee because of the approval, after the committee overwhelmingly rejected the drug. The group proposed changes to the accelerated approval pathway, based on problems they identified with aducanumab and some oncology drugs whose approval was not rescinded after they failed confirmatory trials. They propose 1) a better definition of biomarkers and their use, 2) acceptance of a protocol for the confirmatory trial before accelerated approval, 3) Withdrawal should be automatic, when a confirmatory trial is negative, and 4) CMS and the VA should consider limiting use or expenditures for a drug with an accelerated approval. The fourth point is not under the responsibilities of the FDA but recommended due to potential large budgetary impact.
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