As the Food and Drug Administration moves closer to a decision about whether to approve a promising new Alzheimer’s treatment, the collaboration between scientists, regulators, and business leaders that produced this encouraging drug has come under fire as a “black eye” for the FDA that “dangerously compromised” its objectivity. This unfortunate charge misconstrues the drug development process and the indispensable partnerships that are needed to deliver new treatments.
The drug in question, aducanumab, takes aim at Alzheimer’s disease, the sixth leading cause of death in the U.S. and the only disease in the top 10 that has no cure and no treatment to slow its devastating impact. About 5.7 million Americans already live with Alzheimer’s, a figure projected to triple by 2050. Managing the disease cost the U.S. $305 billion in 2020, and is estimated to rise to $1.1 trillion per year by 2050.
Where is the indignation about Biogen doing poor clinical trials? They rushed into phase 3 before they had all their data from phase 2. Then when the phase 2 data was more mature, they only changed part of their phase 3 trials, leaving intact a futility rule that they apparently did not fully understand. They were then left to advocate for analyses that are neither scientifically nor statistically rigorous. Unfortunately, there is no law against a Sponsor advocating for such poor scientific practices as removing patients from a statistical analysis precisely because they make their treatment look bad. There are laws, however, that the FDA should use good scientific practices. So while a collaborative effort between Sponsor and FDA can only improve public health, that collaborative effort should not be driven by a Sponsor who advocates so strongly for poor scientific practices. There have been many failed clinical trials that looked at whether modifications to amyloid deposition might translate into a benefit on cognition and function. That one such trial is positive is pretty much in line with a type 1 error. This is especially true when an identical trial was negative. Thus approving the drug without adequate evidence may just change the high cost of Alzheimer’s Disease by adding more than $50,000 per year per patient for a drug that does not change the main symptoms of the disease.
The authors doth protest too much, methinks.
So is the first commenter against the approval of ANY drug that doesn’t have 2 phase 3 positive trials, including oncology? Doubtful.
For quite some time, the FDA guidance for unmet needs is one phase 3 and supporting evidence, such as the PRIME study AND the bio marker changes. If you looked at the failed phase 3, it could not be more obvious that the high dose arm is the outlier, especially when you include prime results AND those that were treated to completion.
It’s pretty clear that multiple adcom members didn’t even understand a large chunk of the briefing, and the loudest mouth couldn’t even support his argument when asked questions by other panelists because he didn’t have the documents in front of him. Really? Look up how many on that panel were AD experts? Did it ever occur to you that these academics don’t want drugs approved with slightly less data because it affects their paychecks?
Hydroxychloroquine for Covid anyone? How about some eteplirsen for DMD? The problem isn’t with FDA “collaborating” with industry; its with the agency CONSPIRING to circumvent well-accepted scientific standards, hampering innovation and providing false hope.
Throwing a leaden life vest to a drowning man isn’t compassion. Its cruelty.