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  • D. Maurice Kreis

FDA Unleashed a "Gusher of Profits" on June 7

It's not my habit to trash the Food and Drug Administration (FDA), our national gatekeeper when it comes to the safety and efficacy of the medications on which we all depend. Indeed, as a Cystic Fibrosis parent I have had occasion to dance in the street as the FDA has swiftly reviewed and efficiently approved breakthrough medication after breakthrough medication.

But when the FDA approved a new drug called Aduhelm yesterday -- presumably raising hopes for six million Americans -- the experts I respect were floored. Thus, so was I.


It's the World Series of drug reviews. Aduhelm, manufactured by the Big Pharma firm Biogen, is now approved for the treatment of Alzheimer's disease.


The CF drugs about which I obsess are able to help, at most, 30,000 people in the U.S. If Aduhelm (whose generic name is aducanumab) is truly effective in treating Alzheimer's it won't just be a boon to the 6,000,000 people in the U.S. who have this heartbreaking illness. It will be a balm to -- oh, gosh -- maybe a hundred million Americans, when you consider everyone who loves an Alzheimer's patient and/or who worries about getting the disease themselves some day.


The FDA "has failed in its responsibility to protect patients and families from unproven treatments with known harms," declared the Institute for Clinical and Economic Review (ICER) yesterday. ICER is a respected nonprofit which does what in a better world the government would do -- evaluate the cost-effectiveness of expensive medications. That, of course, requires ICER to know a lot about the efficacy of the emerging drugs it studies.


[Full disclosure: I recently joined the New England Comparative Effectiveness Public Advisory Council (NE-CEPAC), one of three public bodies that ICER convenes to evaluate its findings.]


"Alzheimer’s disease has a tremendous impact on patients and loved ones, and no one can be insensitive to the hopes and the fears that are part of their daily lives," ICER stated in reaction to the FDA decision. "But no one should assume that approving a drug with such conflicting and uncertain evidence will necessarily help patients and families. On them now falls the decision of whether to use a treatment that may not work, that has modest effects at best, and that causes brain swelling and potential bleeding in approximately 30% of patients. This is the type of situation in which the FDA should play a supremely important role: to protect patients and families by holding firm to a standard that evidence reasonably establish that a therapy benefits patients."


At first I was somewhat puzzled by ICER's statement. I know it's been studying Aduhelm/aducanumab and that one of the other ICER public advisory councils (not the NE-CEPAC) will be deliberating on this subject next month. But I wasn't used to seeing ICER critique the FDA's drug approval process.


The account of these developments in the June 8 New York Times helped me understand. ICER always takes the high road but the Times came right out and said it.

Adulhem "is all but certain to unleash a gusher of profits for Biogen," reported Rebecca Robbins and Pam Belluck of the Times. "[T]he new drug is poised to be a blockbuster. For just about everyone else, it is likely to further inflate high U.S. healthcare costs. And that is despite the fact that there is not much evidence the drug actually works."


Just what we need in the struggle to save and strengthen the Affordable Health Care Act, to assure that treatments and drugs are available to all who need them, and to avoid breaking the back of the national economy in the process.


For the salient details, it's interesting to flip back to the ICER statement.

"Our review of the evidence was concordant with that of many independent experts: current evidence is insufficient to demonstrate that aducanumab benefits patients. The avenue forward has seemed clear: another study would be needed to reduce the substantial uncertainty about the drug’s effectiveness, a requirement of even greater priority because of the drug’s common and potentially serious side effects."


Common and potentially serious side effects, on top of everything else? Good grief. "However," ICER continued, "instead of waiting for such a trial, the FDA chose to move the goalposts and approve aducanumab based on the surrogate outcome of removing amyloid from the brain rather than the patient-centered outcome of clinical benefit, which has been required of all previous emerging treatments for Alzheimer’s disease."


Amyloid, as I was only dimly aware before yesterday, is a protein that accumulates into plaque in the brains of people with Alzheimer's. Thus Aduhelm ostensibly treats the underlying cause of the disease as distinct from its symptoms. That's exactly where we're at when it comes to cutting-edge drugs for CF, so I can relate to how joyous that could feel for people affected by Alzheimer's.


"Many other drugs have been shown to remove amyloid from the brain, yet have failed to help patients, making this decision all the more puzzling," declared ICER. "Additionally, the FDA granted approval for treatment of all patients with Alzheimer’s disease despite the fact that the drug has been studied only in patients with mild cognitive impairment (MCI) and mild dementia. Trials of other amyloid therapies in later Alzheimer’s disease have all failed. Thus, there appears to be no evidentiary basis to justify the FDA’s decision to extend the label of aducanumab beyond the study populations."


"No evidentiary basis to justify the FDA's decision" is a pretty damning indictment from the high-minded physicians and economists who are the key officials at ICER. And the price, $56,000 a year per patient, is yet another bloated sum floated by Big Pharma as it continues to savor the benefits of its unregulated monopoly in new medications.


"A therapy of this cost is going to have enormous implications for everyone," Dr. Joseph Ross, a Yale University expert on drug policy, told the Times. "And by everyone, I literally mean you too. There's going to be some 60- and 70-year olds on your plan. If they start getting this treatment, you will see your premiums will go up."


ICER has estimated that a fair price -- one that reflects the actual value of the drug in comparison to other available therapies -- would be something like $8,300 a year. Something is deeply wrong with a system in which a product that has an objective value of $8,300 is able to squeeze an additional $47,700 a year out of every patient who could theoretically seek the drug and will have little interest in shopping around for something cheaper.


That's about $2.9 billion a year in free money to Biogen -- what I learned in law school to call "monopoly rents." No wonder the price of the company's stock leapt 38 percent yesterday. It was a bad day for healthcare.

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