FDA puts the brakes on use of new Alzheimer’s drug and asks for outside investigation of its own hasty OK

aduhelm-300x250The  Food and Drug Administration has back-tracked on a major part of its  accelerated approval of Aduhelm, a prescription medication targeted at Alzheimer’s patients.

The  FDA green light for the drug also has created such consternation among medical specialists, insurers, policy experts, and politicians — including with news reports of hidden, cozy dealings between a top regulator and the medication’s maker —  that the acting agency chief has asked the independent inspector general to investigate what happened.

The fury over Aduhelm is occurring even as another drug maker is pushing legal action that authorities argue also could saddle taxpayers with other soaring costs for other expensive drugs.

Label restrictions

Just weeks after overruling its own elite, independent advisers, and other prominent experts in the field and green-lighting Aduhelm, the FDA suddenly sought to tap its persuasive powers, after the fact, to slash the group of patients the agency gave its official OK to use the drug. The FDA initially had approved it for all Alzheimer’s patients, even though clinical trials for the medication had been run only on those in early stages of the condition.

But in agreement with the drug maker, the agency-approved label for Aduhelm now will say it is recommended for treatment only for those with early-stage Alzheimer’s, slashing to 1.5 million from 6 million the individuals advised for the drug, the New York Times reported.  The label change won’t necessarily affect how doctors use the drug, because once any drug is approved for any purpose, physicians are free to use their medical judgment to prescribe it beyond the labeling recommendations.  And of course there will be massive pressure on prescribing doctors from desperate families to try out the drug on family members with more advanced Alzheimer’s despite what the label says. So the rollback in the label comes after the horse has left the barn.

Before doctors can prescribe Aduhelm, they also are supposed to ensure their patients’ difficult, early Alzheimer’s diagnosis through extensive exams, including pricey brain scans. Because Biogen, Aduhelm’s maker, has found that the drug can have significant side effects, including brain swelling and bleeding, those taking the drug must undergo heavy-duty monitoring, again, replete with those sometimes-risky scans. Aduhelm also must be administered as an infusion, meaning it can only be given in doctor’s offices, clinics, or hospitals — adding further to costs associated with the drug.

Critics ripped the FDA for not only expediting approval for Aduhelm, but accepting what they said were dubious, after-the-fact interpretations by Biogen of data from two clinical trials of the drug. Those trials provided scant evidence of the safety and effectiveness of Aduhelm, critics said.

Still, with FDA approval in hand, Biogen flabbergasted critics and supporters by announcing it planned to charge $56,000 annually for Aduhelm alone. A respected, independent expert group, citing the drug’s minimal effects, had reported the medication should be priced at $2,500-$8,000 annually.

Because the FDA not only permitted the drug on the market but said initially it could be used widely, taxpayers were confronting whopping new costs for patients taking it under the already financially challenged federal Medicare program. New coverage for Aduhelm, critics said, could add up to $29 billion annually — more than what taxpayers spend for the space agency NASA.

Now, with the FDA suddenly wavering in its sweeping endorsement of the drug, the New York Times reported:

“The new guidance does not prevent doctors from prescribing Aduhelm to patients with moderate or severe Alzheimer’s. But the about-face sends a strong message to doctors and insurers about who should receive the drug. It also substantially increases the odds that Medicare and private insurers will restrict coverage of the drug, which is given as a monthly intravenous infusion. That would mean that patients with moderate or severe Alzheimer’s would have to pay the five-figure annual costs out of their own pockets, which experts regard as unlikely to happen frequently.”

Doubts about FDA oversight

Dr. Jason Karlawish, a co-director of the University of Pennsylvania’s Penn Memory Center, told the newspaper the FDA approval of Aduhelm raised major questions about the watchdog agency:

“The revision of this label is yet another piece of evidence that should cause the American public to be concerned about how FDA is practicing its regulatory science.”

Several different House panels have plans to investigate the Aduhelm approval, which also has been assailed by senators, demanding probes by committees, too, on that side of Congress.

Janet Woodcock, the acting head of FDA, took note of the furor and asked for a complete investigation of the agency’s dealings on Aduhelm, as Stat, the medical and scientific news site reported:

“In a letter posted [on July 9], acting Commissioner Janet Woodcock asked the independent Office of Inspector General to investigate how agency staff interacted with Biogen in the run-up to the June 7 approval of Aduhelm. The agency cited STAT’s reporting that FDA officials worked hand in hand with Biogen executives to get the drug on the market, including an off-the-books meeting and an unprecedented decision to approve Aduhelm through a regulatory shortcut.

“’There continue to be concerns raised … regarding contacts between representatives from Biogen and FDA during the review process, including some that may have occurred outside of the formal correspondence process,’ Woodcock wrote in a letter to acting Inspector General Christi Grimm. ‘To the extent these concerns could undermine the public’s confidence in FDA’s decision, I believe it is critical that the events at issue be reviewed by an independent body such as the Office of Inspector General in order to determine whether any interactions that occurred between Biogen and FDA review staff were inconsistent with FDA policies and procedures.’”

Big Pharma challenging rules for pricey drugs

Even as the fury over Aduhelm shows few signs of diminishing, the FDA is grappling with another Big Pharma maneuver to challenge oversight rules, and, effectively, target taxpayers with big costs.

The news site Axios reported that Pfizer has asked a federal judge to declare unconstitutional the FDA rules “prohibiting pharmaceutical companies from directly or indirectly funding patients’ drug copays.”

Critics say these programs, purporting to benefit patients struggling with a prescription medication’s sky-high costs, constitute shift-and-shaft, bribery. Th schemes not only persuade doctors to prescribe drugs with minimal benefit and bankrupting costs, they also convert seriously ill patients into powerful lobbyists for medications that may have little or no effect on improving the quality of their lives or extending them.

Once patients start taking any medication, especially if their prognoses previously were poor and they feel any better at all, they can be noisy advocates for a drug and its maker, the FDA has argued. The agency says that Big Pharma exercises total control over co-pay programs, effectively bribing patients to start a medication, then yanking the financial support out from under them once they start to be persuaded about a costly drug.

Pfizer is contesting the co-pay ban, Axios reported, as the maker of “Vyndaqel and Vyndamax, which treat a potentially fatal type of heart failure …  [T]hose drugs are priced at $225,000 … Medicare patients face out-of-pocket costs of around $13,000 annually [for them].”

In my practice, I see not only the harms that patients suffer while seeking medical services, but also their struggles to access and afford safe, efficient, and excellent health care. This has become an ordeal due to the skyrocketing cost, complexity, and uncertainty of treatments and prescription medications, too many of which turn out to be dangerous drugs.

The FDA, long an important check on Big Pharma and taxpayers’ leading means to ensure the safety, effectiveness, and cost-value of prescription drugs, has been collapsing in its crucial functions. The agency’s useful oversight has been undercut by pro-business, anti-regulation advocates who have made the FDA fee-reliant on the industry it is supposed to regulate, while also failing to stop the spinning door for experts between commercial enterprises and oversight responsibilities.

President Biden can’t wait any longer and clearly can’t rely on an interim chief of the FDA. He needs to appoint a new commissioner to head the agency.

In the meantime, Big Pharma has in relentless fashion also spread its extensive cash in sketchy fashion, whether pouring big money into lobbying Congress and federal regulators, in undercutting the reputation and independence of purported patient advocacy groups, blurring drug finances for patients and doctors with co-pay programs, or even in funding phony fronts, “AstroTurf” groups with alluring names that fail to show their real advocacy in health care issues.

We’ve got a lot of work to do to get new and improved leadership aboard at the FDA, and to restore public trust that the agency is protecting the interests of real people, not wealthy corporations and other political interests eager to suck taxpayers dry.

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