As the nomination of Dr. Robert Califf to head the federal Food and Drug Administration advances, he and the agency already are confronting a major regulatory crisis over Aduhelm, a prescription drug targeted for Alzheimer’s treatment and approved on the thinnest of evidence.
An FDA sister agency, the Centers for Medicare and Medicaid Services (CMS), has joined the Department of Veterans Affairs in sharply restricting Aduhelm’s use and coverage for payment.
Quickly after the FDA approved the drug made by Biogen and the maker priced it at $56,000 annually for patients, the VA said it would consider Aduhelm for use in one of the nation’s largest health systems only on a case-by-case basis.
After months of study, CMS — which had announced one of the largest Medicare premium increases in recent memory, in part to cover Aduhelm prescribing — announced that it will cover the drug only for patients participating in rigorous clinical trials.
This may provide the only glimmer of good news is this sorry saga: the Biden Administration’s top health official has ordered CMS to reconsider its planned Medicare premium hike and pressure may be increasing for this to occur.
With the new CMS plan, two federal agencies have lined up with major hospitals, academic medical centers, insurers, and prominent Alzheimer’s specialists in blasting the FDA’s expedited approval of Aduhelm — over the counsel of the agency’s own elite, independent advisors.
Congress already is investigating whether FDA regulators got too cozy with Biogen, for example, publishing research studies on Aduhelm with company staff, and if U.S. officials incorrectly green-lighted the drug in the belief that some hopeful step needed to be taken because Alzheimer’s is so devastating for patients and has no current therapies that work.
Critics, however, point out that Aduhelm has major side-effects, including its ties to brain swelling and brain bleeding, requiring patients to undergo regular and pricey brain scans. The drug is an infusion that can only be administered in doctors’ offices, clinics, and hospitals.
The most optimistic reading of the results from two clinical trials shows that the drug has only the slightest benefits to early-stage Alzheimer’s patients. Biogen halted trials of the drug in a technical step that ascribes its prospects as “futile,” only to reanalyze its data and declare the drug helpful and worthy of an accelerated regulatory approval.
For Americans accustomed in discussing the coronavirus pandemic, it may be helpful to know that Aduhelm is a monoclonal antibody. It has been shown to reduce amyloid plaque, a protein buildup in the brain implicated in the cognitive decline due to Alzheimer’s.
But as critics have emphasized, this is a theory, not a proven way to treat a debilitating, progressive illness. With decades of research behind them, medical scientists have not shown in definitive fashion that reducing amyloids successfully treats Alzheimer’s.
Giving maximum benefit of the doubt to the FDA regulators who wanted to throw open the doors for Aduhelm to prove or disprove the amyloid theory, critics cringe. This is not how medical science works, they say, pointing out that can amount to cruel human experimentation that may raise false hopes.
Further, with all the turmoil surrounding Aduhlem, even Big Pharma is upset. Drug makers long have relied on the FDA’s once-high credibility in its work to ensure that others would not intervene in the ways that prescription medications get to market. CMS and the VA, typically, would approve coverage of drugs that were rigorously tested and analyzed by FDA experts.
Biogen’s competitors have expressed major worry that their drugs targeted at amyloids and Alzheimer’s, no matter if they present evidence that they are better, cheaper, and more effective, now will hit harsh scrutiny — not only at the FDA but also with other federal agencies that might be involved.
Patient advocacy groups that had put their reputations on the line to endorse Aduhlem now find themselves in the lurch, too, especially with tough questions surfacing about just whose interests the organizations may represent. Key advocacy organizations have said they will fight for the drug.
Critics have pointed out that the hurry to approve Aduhelm meant that many kinds of patients, particularly from communities of color, were under-represented in the drug maker’s clinical trials. Further, because Biogen defied independent experts and their assessments in setting a nose-bleed price for Aduhelm, the drug, critics say, was headed to being a therapy only for wealthy patients who lived near expensive hospitals and had fancy doctors who might have gotten access to it. This became truer with the VA and CMS curtailing coverage.
Doctors, of course, may prescribe the drug for any patients, now that it is FDA approved, and particularly because the agency bungled the way it let the medication on the market. Biogen’s clinical trials targeted only early-stage patients (who can be tough to diagnose, by the way) but the FDA approved Aduhelm for all Alzheimer’s patients, though the agency tried to walk this back in labeling.
As for Biogen, it halved its announced price for Aduhelm after the drug’s revenues came in, in relatively paltry fashion for Big Pharma, at a few hundred thousand dollars, not the tens of millions expected. The company declared that it would speed up FDA-required, further clinical testing of the effectiveness and safety of Aduhelm. The company also has announced a major retrenchment that will cost a substantial number of the company’s workers their jobs. Biogen is looking to acquire other makers with promising drugs that might boost its own sagging fortunes.
In my practice, I see not only the harms that patients suffer while seeking medical services but also the damage that can be inflicted on them and their loved ones by dangerous drugs. They also struggle to access and afford safe, efficient, and excellent medical care due to the skyrocketing cost, complexity, and uncertainty of therapies and prescription medications.
It is vital, if taxpayers are spending billions of dollars on federal health agencies, for them to be the people’s advocates — not cronies of makers of prescription drugs or medical devices or pals with patient advocacy groups that are sometimes in the pocket of Big Pharma. We need tough oversight of Big Pharma, such as the tens of millions of dollars in regulatory claw-backs and lifetime banishment from the business handed to an awful executive like Martin Shkreli for trying to gouge patients ruthlessly. We must insist that our drug regulators remain independent, rigorous, unbiased evaluators of products and that they not go out on rogue adventures that leave patients, taxpayers, and their federal colleagues in untenable circumstance. We have much work to do with Dr. Califf, President Biden, and Xavier Becerra, the head of the sprawling Health and Human Services agency to make the FDA a much better functioning watchdog.