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For all the explosive controversy over the approval of the first treatment for Alzheimer’s disease in nearly 20 years, hardly any patients have actually gotten it yet.

The drug’s eye-popping, $56,000 annual price and questionable benefit to patients have been a shock to the bureaucracy that makes the health care system run — and that’s having a clear effect on uptake. Some analysts estimated last month that fewer than 100 patients were dosed in the first weeks after the therapy was approved, though availability will likely ramp up over the coming months.

Though the Food and Drug Administration said in approving the therapy, Aduhelm, that the data indicate a likely benefit, hospital and insurer committees are conducting their own analyses, acting as another set of gatekeepers. They regularly review new treatments, but the lingering questions about the drug’s efficacy, as well as the logistical challenges of delivering an infused drug, are complicating and prolonging those discussions.

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“This is an added burden on health systems,” said Isha Rana, a pharmacy administrative specialist in formulary management at Houston Methodist. “You have a very contentious FDA approval, and now health systems are going to have to go back and do the same reviews that the FDA did and decide on whether we think the benefits outweigh the risks.”

The deliberations create a “holding pattern” for patients, as several experts described it, that could last months as doctors and insurers, including Medicare, sort out their policies. Usually when the FDA approves a drug, Medicare quickly covers it.

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“The FDA decision is just the first domino to fall,” said Anna Legreid Dopp, the senior director of clinical guidelines and quality improvement at the health-system pharmacists association ASHP.

Biogen, which makes Aduhelm, would not say how many people have been treated with the drug since it was approved.

On an earnings call last month, company executives argued there was an eagerness from both providers and patients for the therapy. Of the 900 sites that the company said would be ready to offer the drug after approval, one-third had already completed their reviews “with a positive outcome” or decided they didn’t need to go through such a process, CEO Michel Vounatsos said.

However, he acknowledged that with reimbursement decisions, “it is still the early days,” and that tests to confirm the presence of a particular protein in patients to ensure they’re eligible are “also taking time to schedule and coordinate.”

Chief Financial Officer Mike McDonnell said the company expected “modest” revenue this year from Aduhelm in part because of “the need for sites to prepare to diagnose and treat patients and the time that it will take to secure payer coverage.”

Health systems turn to what are called pharmacy and therapeutics committees when considering adding a drug to their formularies, weighing purported benefits, safety profiles, and costs. The committees — which can consist of pharmacists, doctors, nurses, and financial whizzes — set out policies for everything from which physicians will prescribe the therapy, to which patients could receive it, to how they’ll handle side effects, to how they’re going to store and administer the treatment. Physicians in private practice can often set their own policies, either with partners or by themselves.

Already, some health systems have said they’re not going to provide Aduhelm (though their physicians can still prescribe the treatment for patients to receive elsewhere), citing a desire for more data or concerns about the process by which FDA approved the treatment.

Critics have accused the agency of working too closely with Biogen on shepherding the drug through review and have raised questions about how the agency moved the drug to market under its accelerated approval process.

“It will be interesting to see how people deal with the controversy that FDA has dropped on everyone’s lap,” said Daniel Zlott, the senior vice president of education and business development at the American Pharmacists Association.

Health systems also have to decide whether to further limit the potential patient pool for Aduhelm. The FDA’s prescribing information for the treatment says it’s meant for people in the earliest stages of Alzheimer’s disease, diagnosed with either mild cognitive impairment or mild dementia. But health systems could create policies more in line with Biogen’s original clinical trial population and restrict Aduhelm to patients who also have confirmed amyloid protein plaques, which the drug is designed to clear.

The need to test for amyloid creates another hurdle for health systems. People are screened for amyloid either through PET scans or by obtaining a cerebrospinal fluid sample through a lumbar puncture, introducing another round of procedures and experts who need to assess the results. Not all clinics have access to PET scans, and it’s unclear whether Medicare will cover them. (The hope is that blood tests for amyloid, which are in development, could make the process easier and cheaper.)

“Without the target being present, there is no point in administering the drug,” neurologist Liana Apostolova of the Indiana Alzheimer’s Disease Research Center said on a panel at the Alzheimer’s Association International Conference last week. Apostolova has received consulting fees from Biogen.

Other physicians agree with the need to establish the presence of amyloid. Mia Yang, a geriatrician at Wake Forest Baptist Health, said a number of North Carolina hospitals have discussed setting common clinical guidelines, and that one easy point of agreement was that “we’re not going to offer the medication to people who do not fit those original clinical trial criteria.”

Even as health systems’ pharmacy and therapeutics committees are working on their strategies, many physicians say they will wait for insurance policies before they prescribe Aduhelm.

Most insurers, at least so far, aren’t saying much. Medicare officials are currently weighing who will be eligible for the drug, and they won’t come to a final decision until 2022.

It all leaves a black hole of uncertainty for patients over how they could pay for a treatment, even if their provider would prescribe it.

Regional Medicare contractors could issue statements about their coverage policies for Aduhelm before a national policy comes out, but they are holding off for now. Every contractor declined to comment on their plans in response to STAT inquiries.

“It’s all a little hazy with this drug,” Rana said, referring to how coverage is going to work until there’s a national decision.

Medicare has an appeals process if patients want to petition a coverage denial, but there’s not much additional real-world evidence yet for patients to make a case for coverage, said James Chambers, an associate professor of medicine at Tufts Medical Center.

Most commercial insurance plans have been similarly cautious. The largest health insurance lobby AHIP asked Medicare officials to make a uniform national policy for how to cover Aduhelm, and several large insurers including UnitedHealthcare, Humana, CVS Health, Cigna, and Anthem didn’t answer inquiries from STAT about their plans.

Ceci Connolly, president and CEO of the Alliance of Community Health Plans, said an expert advisory panel’s decision not to recommend Aduhelm’s approval and the public announcements that major medical centers, including Cleveland Clinic and Mount Sinai Health System, would not administer the drug contributed to insurers’ hesitance to cover it.

“It gives us all pause, and at a minimum suggests we’ve got to keep digging,” Connolly said.

While many plans are waiting, some are moving forward. An apparent draft Centene policy has been published online, which would provide the treatment to patients, but only if they test positive for amyloid plaques and are not taking blood thinners. Centene did not respond to a request for comment on the draft policy.

The combined Harvard Pilgrim Health Care and Tufts Health Plan has threatened not to cover Aduhelm unless Biogen lowers its price tag. The plan has more resources and expertise to review the drug faster than some plans with less institutional support, and Connolly said smaller plans will likely defer to the federal government’s decision.

Some affiliates of Blue Cross Blue Shield have decided not to cover Aduhelm, saying the drug is still investigational and experimental, though Biogen has contended the classification is a mischaracterization.

Even though most patients eligible for Aduhelm will be in the Medicare program, Chambers said the drug’s price and population still makes it likely that commercial plans will eventually have an explicit policy on how to cover the drug, though he expects them to join Medicare contractors in waiting until after Medicare’s decision.

Medicare [decisions like this] are usually reserved for big-ticket drugs where there is a chance of real inconsistency,” Chambers said. “With a drug as controversial as Aduhelm, I would be surprised if anyone wanted to get out in front of that.”

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