As we mentioned in August 2021, there were to be investigations into how it was Aduhelm was approved by the FDA and how Biogen justified its unprecedented price tag. Last week, the House Energy & Commerce and the Investigations & Reforms panels completed their investigation and issued their report. You probably anticipated correctly that it was quite critical of both the FDA and Biogen.
Recall a couple of things. Originally, Biogen (and its Japanese partner, Esai) said their clinical trials found the monoclonal antibody simply didn't work. Then they reevaluated one subset of their data to find that it may actually have worked some. So Biogen applied for approval, and the FDA put it on an unusual approval fast track. But when it seemed ready to go, the FDA's 11-member independent advisory panel reviewed the clinical trial data themselves and voted unanimously that it not be approved. In their view, the interpretation of the data didn't demonstrate that it worked. But the advisory panel's judgement was not binding, and the FDA gave the treatment a conditional approval. Three members of the FDA's advisory panel then quit in disgust.
This was (and is) a fraught time in the history of Alzheimer's research. The disease is far more complex than anyone dreamed, and proposed drug interventions have failed, one after the other. Yes, some drugs can give symptom relief, but none had been shown to modify the disease process, actually treating the disease.
With millions of people succumbing to this, the sixth leading cause of death in the US, incredible pressure has been on the pharmaceutical industry and their regulators to come up with something. I recall in 2017 seeing an article in a popular magazine saying that the FDA should simply approve an Alzheimer's drug even without completing clinical trials. The article didn't say, but it appeared to me that the treatment in question was aducanumab (later sold as Aduhelm). There were incredibly high hopes riding aducanumab -- and incredibly high pressure on those involved in developing any treatment that might help.
So when the FDA did provide conditional approval from questionable clinical trial results, and when Biogen started selling it at $56,000 per year (not counting the cost of regular MRIs), there was an uproar. At that price and with the anticipated population of patients, there was fear Aduhelm would simply bankrupt Medicare. Some in the congress vowed there would be an investigation.
Hence the investigation report released last week. Most of the criticism fell to the FDA for a process that was "rife with irregularities." It appeared to the investigators that the FDA was too cozy with Biogen. The FDA and Biogen claimed that closeness was necessary in the face of such a complex scenario. Here's what Biogen had to say.
So what will happen?
As we've discussed before, the FDA and Medicare have severely limited the availability of Aduhelm, essentially turning current commercial usage into an extended clinical trial. The FDA needs more evidence before giving the monoclonal antibody wider availability, and Medicare wants to know they're getting their money's worth, even after Biogen cut the price in half.
Where do we go from here? Hopefully the ongoing limited use of the Aduhelm will tell us how well it works. But that's going to take some time. As for consequences of the congressional report? Hopefully the FDA and Biogen will go forward in a way that builds public confidence in both the regulatory process and in this generation of Alzheimer's disease treatments.
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