The aducanumab shock waves will radiate beyond Alzheimer's treatments

There's been a lot in the news lately, and the controversy over the FDA's approval of aducanumab/Aduhelm has receded.  But that doesn't mean that the controversy has gone away.  In fact, both the price and the way in which it was approved have invited several investigations.  These will likely impact both how the FDA manages accelerated approvals and how new drugs are priced.

When the FDA approved aducanumab, they did so without the evidence of effectiveness normally required for approval of a new drug, and their advisory committee had given it a thumbs down.  When Biogen announced their pricing of $56,000 per year for the treatment (but varying depending on how much the patient weighs), there was quite an uproar.  For one thing, there was anticipation that this could drive Medicare into an even deeper hole than what already appears to be on the horizon.  So now many players want to get into the act of critiquing how we got here.

While three members of the FDA's advisory committee quit over the controversy, the committee members together wrote an article for the New England Journal of Medicine criticizing how they were informed (or misinformed) of the FDA's strategy.  They also cast doubt on the utility of using beta amyloid plaque loading as a surrogate for effectiveness -- a substitute for showing that the treatment actually improves cognition.  At the same time, the FDA's inspector general is being tasked to investigate the appropriateness of the FDA's relationship with Biogen.  This may extend into an evaluation of the whole accelerated approval program.  The investigation was prompted by an inquiry from Rep. Katie Porter (D-CA). 

Is that enough?  No.  Two House committees, Oversight and Reform as well as Energy and Commerce, have begun collecting documents from Biogen and other entities preparatory to hearings.  And Senator Ron Wyden, chair of the Senate Finance Committee, already has an interest in how new drugs are priced at launch.  He is making noises suggesting an investigation by him will fuel interest in drug price controls.  However, the Senate has not yet responded to the letter from Elizabeth Warren and Bill Cassidy highlighting the challenges that Aduhelm presents to Medicare.

What does this mean for the career of Dr. Janet Woodcock, acting commissioner of the FDA?  In a letter to President Biden, Senator Joe Manchin (D-WVa) outlines the Aduhelm controversy, stating that it's time to put in a permanent commissioner -- someone other than Dr. Woodcock.

Fun times.

Now, some FDA officials have responded to the attention on the accelerated approval process.  In an article in JAMA (formerly the Journal of the American Medical Association) they make the claim that the program has been instrumental in getting some new cancer treatments to market.  So there's something to be said for it.  Presumably, this required judicious and thoughtful application of the process -- and not influenced by political pressure.