Allysia Finley opines in The Wall Street Journal on June 24 that the FDA was correct in its overriding of its Advisory Board’s recommendation to reject the application of Biogen’s drug aducanumab. The agency approved it for treatment of early Alzheimer’s disease. The drug is reputed to reduce amyloid plaque in the brain and reduce cognitive decline. As Ms. Finlay points out, the clinical trials were not perfect in their conclusions but there was some signal of benefit at high drug doses.
There’s a “proof of concept” argument for approval in that this is the first drug that actually does reduce amyloid plaque, but at best the benefits are modest and the cost very high (estimated at $56,000 per year just for the drug. The associated testing will be additional costs.) If millions are prescribed the new drug, this would have a significant cost increase to the entire health care system with minimal benefit and much of the burden would fall on Medicare.
It is likely that there will be more and better drugs. Progress is incremental, any being welcome, but was this a wise decision by the FDA?
I think this is a typical scientific result with an equally typical political answer. Maybe the drug does some good. Whatever it does, it isn’t much and the ultimate defeat of degenerative neurological diseases will come with prevention strategies not treatments although progress in the treatment of multiple sclerosis has certainly improved since I was in medical school as one of the doctors quoted in the article says.
In the treatment of the remaining degenerative diseases of mankind—neurologic, cardiac or malignant—progress has always been slow with some incremental jumps like statins and combination chemotherapy. The FDA is in the difficult position of having to decide whether to unleash a new drug or device on the market based on clinical trials that are hard to do, very expensive and often somewhat ambiguous in their results.
The only logical thing to do is to allow the drugs and devices that show promise to be more widely employed while keeping close tabs on the progress being made by enforcing rigid standards of post-marketing study and data collection. Of course, toxicity signals that may be masked in the smaller initial trials may become manifest when a drug is used on a wider population. Thus, a very formal approach to data collection must follow early drug approval. Everyone that gets this new Alzheimer’s drug must have his or her data collected and reviewed by the FDA as the drug’s introduction rolls out.
This is a desperate group of patients looking for any progress. Nonetheless, the regulatory agencies must have rigid and rigorous standards with regard to approving new treatments. I am not at all sure that this new drug really met the threshold of benefit exceeding risk, but now that it’s out there, let’s make sure we get all the data and then hope that something better comes along. Soon.