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July 19, 2021

Will CMS Find Aducanumab Reasonable and Necessary for Alzheimer Disease After FDA Approval?

Author Affiliations
  • 1Clinical Excellence Research Center, Department of Medicine, Stanford University, Stanford, California
  • 2Graduate School of Business, Stanford University, Stanford, California
  • 3Department of Neurology and Neurological Sciences, Stanford University, Stanford, California
  • 4School of Law, Duke University, Durham, North Carolina
JAMA. 2021;326(5):383-384. doi:10.1001/jama.2021.11768

The recent US Food and Drug Administration (FDA) approval of aducanumab for the treatment of Alzheimer disease triggered both praise and criticism from various advocacy groups, but a more important decision regarding the drug—whether to pay for it—will now need to be undertaken by the federal government. This next decision offers a window into both the challenging politics of value-based health care and the difficult administration of pharmaceutical policy in the US.

Alzheimer disease is a devastating illness, with significant unmet clinical need, and effective therapies for the prevention or treatment of the disease would be welcome. There is significant controversy around the assessment of whether aducanumab (a monoclonal antibody that targets amyloid-β) met the FDA’s legislative standard of “safe and effective”1 required for approval. The 2 primary clinical trials (EMERGE and ENGAGE) were stopped for futility according to a prespecified pooled interim analysis, but the final analysis of the individual trials found a statistically significant yet modest slowing of decline in cognition compared with placebo at 78 weeks in the high-dose treatment group of one trial (in the FDA analysis, a decline of 1.35 in the treatment group vs 1.74 in the placebo group on the 18-point Clinical Dementia Rating Scale Sum of Boxes score2). Differences in rates of cognitive decline in the placebo groups in the 2 trials, among other factors, could have accounted for the observed benefit in the trial with positive results.3

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