Politics. It isn’t always for the best but political leaders usually get their seat because, according to their constituents, they know best. Best, of course, is relative, as in better than the other guy, which can be far from perfect.
What is worse, however, is many of the elected are infected with a certain laissez faire cum libertarianism that suggests the people that got the most votes on that day in early November don’t know much at all.
Take for example things like “right-to-try” or even the FDA’s accelerated approval pathway. Both suggest that high quality research is not required, at least not immediately. The idea is that this disease or that is so bad, so in need of a cure that – if we are being direct – it justifies experiments on humans.
Those humans might be all in, unlike the beagles that Congress has fought hard to protect from laboratory science, but there also exists the possibility they may not understand FDA’s imprimatur for these unmet medical needs ain’t worth much at all.
They may also not fully appreciate that once you relieve the FDA of their historical mission to require rigorous trials and independently review clinical data, narrative and politics slip into place and you get:
Aduhelm.
And a whole lot of other bad stuff especially in the oncology field, like Selinexor.
Without irony, the House Oversight and Reform Committee issued a report on Friday that details just how far the FDA, drug manufacturers and patient advocates can wander when a whole bunch of money can be made, medical evidence not required.
Policy. The House Oversight report is not exactly news for anyone paying attention. To our mind, the most interesting sections are the ones detailing the FDA’s pivot from a traditional new drug application to the accelerated pathway and the broad label indication.
It is standard government practice to try and find a way to “yes” on virtually everything as long as it is reasonable and meets the requirements of the law. In this case, however, Aduhelm had failed to meet its primary endpoint in a traditional path to approval so instead of “Better luck next time!” FDA’s Dr. Billy Dunn brought up the accelerated approval pathway.
To BIIB’s credit, they at least acknowledged internally, the downside of a broad label. The FDA did issue a clarification, but the episode makes one wonder if the agency was purposefully trying to drive the drug off a cliff.
As things turned out, with CMS stepping in to control Aduhelm’s use, the outcome is closer to what it should have been had the regulatory system been working properly.
The price for all this is more deterioration of the FDA’s reputation.
Power. Congress has come to understand the temptation of Accelerated Approval and included some changes in the FY 2023 Omnibus like the initiation of trials prior to applicant submission.
What is not yet factored in is how the group think in scientific research made it nearly impossible to reject anything that sounds like it might one day undermine the Amyloid-Beta theory.
The NIH loves them some Amyloid Beta. So much so they were willing to fund only that research that confirmed it. A few months ago, I interviewed Aditi Bhargava at UCSF and she also described the ostracism that funding organizations use to discourage requests to investigate alternative theories.
The FY 2023 omnibus included a little-less-than-status quo budget increase of $2.5B for NIH. That is good and bad. Good, because it sends a message to the NIH that it probably needs little reform. Bad because it makes funding even more competitive which means research will continue to crowd into the same questions as it has done with Alzheimer’s research.
Perhaps the changing of the guard will help. Although faithful lieutenants of the unquestionably powerful Drs. Anthony Fauci and Francis Collins have taken over, history suggests the sycophancy that made them faithful second bananas will probably make them less than ideal leaders.
Have a great long weekend and Happy New Year.
Emily Evans
Managing Director – Health Policy
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