Total direct health care costs for skilled nursing care, home health care, and hospice care were estimated at $305 billion in 2020. Yet indirect costs — borne mainly by families in the form of lost wages and other costs — are thought to be even higher. By the year 2050, the affected population is projected to reach nearly 14 million individuals, with direct care costs over $1 trillion.7
The history of how aducanumab came to this point is somewhat confusing. On March 21, 2019, the manufacturer (Biogen) announced it had ended two Phase 3 clinical trials of aducanumab for Alzheimer’s. The drug had failed a “futility analysis,” which means a clinical trial is stopped when the interim results suggest that it is unlikely to achieve statistical significance.8
But then just five months later, Biogen announced that they were indeed applying for FDA marketing approval. They explained that data gathered in the three months between the start of the futility analysis and the decision to end the trial hadn’t been used in the original analysis. After adding the additional three months of data, they realized that a subset of patients who had been given higher doses saw significant benefits on measures of cognition and function, including memory, orientation and language. Those patients also saw benefits in daily living activities.9
The OptumRx Pipeline Surveillance team observes that despite the positive reported outcomes, aducanumab has only demonstrated a statistically significant improvement in one of the two pivotal trials. Further, in the one positive trial, the reported improvements were small, and the true clinical relevance of the findings remain uncertain.1
On November 6, 2020, an FDA advisory committee convened to discuss the safety and efficacy of aducanumab voted not to endorse its approval.10
What happens next?
The FDA usually follows the recommendations of its advisory committees, but that’s not required.1
Industry analysts appear split; some believe the FDA has no choice but to follow the panel’s recommendations, given the contentious recent history of certain COVID-19 drug approvals. But others are not so sure. An analyst at SVB Leerink writes that, despite the negative vote, “…we still believe there is a good chance that the FDA will approve this product anyway.”10
For detailed trial results, please refer here: Q4 2020 Report, p. 16.