The CMS Aduhelm Decision: Why It Was Dead On
By
Leonard Zwelling
I adore The Wall Street Journal and often agree with its politics, but the editorial that led the Journal’s editorial page, Review & Outlook, on Monday January 24 misses the mark completely.
The Journal calls the Centers for Medicare and Medicaid Services (CMS) a “death panel” for being unwilling to pay for a new class of Alzheimer’s drug that was given accelerated approval by the FDA recently despite the advisory board’s recommendation not to do so. While it may be true that these new agents like Aduhelm decrease the amyloid in the brains of people with Alzheimer’s, they have not been conclusively shown to alter the course of the disease or the decline in cognitive function of its victims. Thus, CMS will pay for the drug IF the recipient is on a clinical trial—one that is randomized between drug and placebo. This is exactly as should be the case with a drug given accelerated approval. That approval basically says we need more data to know if this really works, but since it might and the target disease is one desperately needing treatment, a randomized, placebo-controlled trial is the best way to do it.
I agree.
The Journal’s stance about “right-to-try,” another foolish concept, is well known. The conservative right that the Journal represents believes that the individual ought to have the final say when it comes to experimental drugs. I don’t.
The clinical trial and human subjects research protection system of the United States has its flaws, but it was the one that kept Thalidomide out of the medicine cabinets of many pregnant American women thus saving their babies from birth defects.
I’m not a huge fan of the FDA, but the advisory boards made up of experts from academia and industry provide the best chance that only safe and effective drugs and devices find their way to patients who have no way to know what will benefit them or harm them when a new treatment option is offered. There have been more than enough examples of unethical experimentation and medical care associated with novel treatments. The system may be flawed, but it’s better than most others and surely better than allowing every sick person to have to decide for him or herself whether a new treatment actually will benefit them without clear rules.
And then there’s the cost. The drug’s initial cost estimated was $56,000 per patient. It has since been reduced. There are hundreds of thousands who might qualify and most are over 65. Thus, Medicare would be on the hook for millions for an unproven therapy even at the new discounted rate. Unacceptable.
CMS made the right call on this even if my friends at the WSJ disagree. In essence, federal law says there is no “right-to-try.” Everyone cannot do anything he or she wishes to do when it comes to novel treatments, especially if the American people have to bear the cost. The FDA has grown and developed as an agency to protect the public. Is it perfect? No. Is it better than nothing? Much.
The WSJ is just plain wrong here. If they wrote about the price tag and the amount it would raise Medicare deductions from paychecks, perhaps they would be doing a better job of reporting than what they printed on January 24.
4 thoughts on “The CMS Aduhelm Decision: Why It Was Dead On”
Perhaps a better way to deal with new, unproven drugs/treatments would be to allow anyone the “right to try”. In return, all those who received the drug/treatment would be automatically enrolled as a participant in a well designed a randomized, placebo-controlled trial to evaluate the efficacy of the drug/treatment. This is of course contingent upon the safety of the drug having already been established. This is the price the individual pays for the opportunity to get the unproven drug. In turn, the pharmaceutical company would have to provide the drug for free for all participants until such point as the drug is deemed safe and effective and has FDA approval. This is the price they pay to speed up the clinical trials and the approval process. This also eliminates insurance payer denials for “unproven” treatments. This system would hopefully facilitate obtaining and aggregating larger amounts of clinical data in a shorter period of time
That’s pretty much the way this is for the new Alzheimer drugs. You can try, but only in a trial and you may not get the drug. You may get placebo.
In fact, that’s how it is for most new drugs. The new drugs are of no use to pharma without the good trials because the FDA won’t approve them for general use without the trial.
Keep reading, Burt!
LZ
There is little chance that the millions of Americans who eventually suffer Alzheimer’s Disease and other dementias will ever receive theses extraordinarily expensive drugs. The rich may, but the uninsured and poor will not.
We need more education and money invested in what we already know about decreasing the risk of all forms of dementia with diet, environment, and avoidance of factors that accelerate dementia, e.g. alcohol, lack of exercise, etc. And, we need better resources to help families that must care for the demented or that struggle to afford a place for them to be cared for with compassion.
Amen