Another controversial aspect of the drug is that Biogen, which developed the drug with Japanese pharmaceutical company Eisai, has set the price at $56,000 for a year's worth of treatment per patient, much higher than what industry experts were expecting. What's more, it's the kind of treatment that many people would need for years. Can you offer insight as to why Biogen set the price at that level?
Many experts were expecting a price around $10,000 based on current Alzheimer's treatments, but honestly the high price was not a shock to me. First, this is a biologic drug, and Biogen views it as a specialty drug, so given the prices of such specialty drugs in recent years, this high price was not atypical.
One thing to note is that the drug received a much broader approval label from the FDA than expected. Based on the trials and data, Biogen expected that the FDA would approve the drug for a subset of Alzheimer’s patients, specifically those in the early stages of the disease. Biogen was expecting a population of around 1 million to 2 million patients to be eligible for the drug. Instead, the FDA approved the drug under a much broader label, making all 6 million U.S. patients eligible. The original, smaller patient population was probably a consideration in the higher list price. Even with the new label, I don’t expect that the list price is going to be lowered unless Biogen faces significant backlash or if the drug faces hurdles with patient access.
Because the drug was OK'd as part of the FDA's “accelerated approval” process, Biogen must conduct a randomized controlled trial to verify aducanumab’s efficacy. Is that a routine procedure? And is it possible the FDA will rescind its approval if this subsequent trial shows poor results?
The Accelerated Approval pathway is an essential piece to this story. This pathway was initiated in 1992 specifically for conditions with a high unmet need. The idea is to approve the drug based on a surrogate marker to start helping patients and then have companies perform confirmatory trials. So, yes, this pathway is routine for many oncology drugs and others where, again, there is a high unmet need. If these confirmatory trials fail to demonstrate the clinical benefit, the drug is pulled from the market.
For example, Bristol-Myers Squibb withdrew its drug Opdivo for a particular subset of lung cancer patients in December 2020 after confirmatory trials did not meet the endpoint of overall survival. The drug was granted accelerated approval for this indication in 2018 based on a surrogate endpoint.
One major issue in the aducanumab decision is that the FDA has given Biogen until 2029 to complete these confirmatory trials, so this drug can potentially be on the market for at least until 2030, costing the health care system significantly before any decision might be made to withdraw. The FDA has come under scrutiny for failing to enforce these confirmatory trials. For example, confirmatory trials for Exondys51 have not been complete yet, although the completion date in their letter was November 2020.