For all the explosive controversy over approval of the first treatment for Alzheimer’s disease in almost 20 years, almost no patient has contracted it yet.
The drugs dazzling, $ 56,000 annual price And the questionable benefit to patients has been a shock to the bureaucracy that runs the health care system, and that has a clear effect on acceptance. Some analysts estimated last month that fewer than 100 patients were dosed in the first few weeks after the therapy was approved, although availability is likely to increase in the coming months.
Although the Food and Drug Administration said in approving the therapy, Aduhelm, that the data indicates a possible benefit, hospital and insurance committees are conducting their own analyzes, acting as another group of watchdogs. They regularly review new treatments, but lingering questions about drug efficacy, as well as the logistical challenges of administering an infused drug, are complicating and prolonging those discussions.
“This is an additional burden on healthcare systems,” said Isha Rana, pharmacy management specialist in forms management at Houston Methodist. “It has a very controversial FDA approval, and now health systems will have to go back and do the same reviews that the FDA did and decide if we think the benefits outweigh the risks.”
The deliberations create a “waiting pattern” for patients, as described by various experts, that could last for months as doctors and insurers, including Medicare, solve your policies. Usually, when the FDA approves a drug, Medicare covers it quickly.
“The FDA’s decision is just the first domino to fall,” said Anna Legreid Dopp, senior director of clinical guidelines and quality improvement for the ASHP Association of Healthcare Pharmacists.
Biogen, which makes Aduhelm, did not say how many people have been treated with the drug since it was approved.
In an earnings call last month, company executives argued that both providers and patients were eager for the therapy. Of the 900 sites the company said would be ready to offer the drug after approval, a third had already completed their reviews “with a positive result” or decided they didn’t need to go through that 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 are eligible “also take time to schedule and coordinate.”
Chief Financial Officer Mike McDonnell said the company expected “modest” revenue this year from Aduhelm, in part due to “the need for sites to prepare to diagnose and treat patients and the time it will take to secure payer coverage.” .
Health systems turn to so-called pharmacy and therapy committees when considering adding a drug to their forms, weighing the supposed benefits, safety profiles, and costs. The committees, which may be made up of pharmacists, doctors, nurses, and financial experts, establish our policies for everything from which doctors will prescribe the therapy, to which patients could receive it, how they will manage side effects, how they will store and administer the treatment. Doctors in private practice can often set their own policies, either with their partners or on their own.
Already some health systems have said they are not going to provide Aduhelm (although their doctors may still prescribe the treatment for patients to receive elsewhere), citing a desire for more data or concerns about the process by which the FDA approved the treatment.
Critics have accused the agency of labor too close to Biogen about pasturing the drug through review and they have raised questions on how the agency brought the drug to market under its expedited approval process.
“It will be interesting to see how people deal with the controversy that the FDA has dropped on everyone’s lap,” said Daniel Zlott, senior vice president for education and business development for the American Pharmacists Association.
Health systems must also decide whether to further limit the potential pool of patients for Aduhelm. FDA Prescribing Information because the treatment says it is intended for people in the early stages of Alzheimer’s disease, diagnosed with mild cognitive impairment or mild dementia. But health systems could create policies more in line with the population of Biogen’s original clinical trial and restrict Aduhelm to patients who also have confirmed amyloid protein plaques, which the drug is designed to remove.
The need to test for amyloid creates another hurdle for healthcare systems. People are screened for amyloid using positron emission tomography scans or by obtaining a sample of cerebrospinal fluid through a lumbar puncture, which presents another round of procedures and experts who need to evaluate the results. Not all clinics have access to PET scans and it is unclear if Medicare will cover them. (The hope is that amyloid blood tests, which are in development, can make the process easier and cheaper.)
“Without the goal present, there is no point in administering the drug,” said neurologist Liana Apostolova of the Indiana Alzheimer’s Disease Research Center in a panel at the International Conference of the Alzheimer’s Association last week. Apostolova has received consulting fees from Biogen.
Other doctors agree on the need to establish the presence of amyloid. Mia Yang, a geriatrician at Wake Forest Baptist Health, said that several North Carolina hospitals have discussed establishing common clinical guidelines, and that one point of easy agreement was that “we are not going to offer the drug to people who don’t fit those criteria. originals of the clinical trial “.
Even as health systems pharmacy and therapeutic committees are working on their strategies, many doctors say they will wait for insurance policies before prescribing Aduhelm.
Most insurers, so far at least, aren’t saying much. Medicare officials are currently weighing who will be eligible for the drug and won’t reach a final decision until 2022.
It all leaves a black hole of uncertainty for patients about how they could afford a treatment, even if their provider prescribed it.
Regional Medicare contractors could issue statements about their coverage policies for Aduhelm before a national policy comes out, but they are waiting for it for now. All contractors declined to comment on their plans in response to STAT inquiries.
“Everything is a bit confusing with this drug,” Rana said, referring to how coverage will work until there is a national decision.
Medicare has an appeals process if patients want to request a denial of coverage, but there isn’t much additional real-world evidence yet for patients to file a coverage case, said James Chambers, associate professor of medicine at Tufts Medical Center.
Most commercial insurance plans have been equally cautious. The largest health insurance lobby group, AHIP, asked Medicare officials to come up with a uniform national policy on how to cover Aduhelm, and several major insurers, including UnitedHealthcare, Humana, CVS Health, Cigna and Anthem, did not respond. STAT inquiries about your plans.
Ceci Connolly, President and CEO of the Alliance of Community Health Plans, said the decision of an expert advisory panel not to recommend Aduhelm’s approval and public announcements that major medical centers, including the Cleveland Clinic and Mount Sinai Health System, would not administer the drug contributed to insurers’ hesitation to cover it.
“It gives all of us pause, and at the very least suggests we need to keep investigating,” Connolly said.
While many plans wait, some advance. An apparent draft Centene’s policy has been posted online, which would provide 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 Harvard Pilgrim Health Care and Tufts combined health plan has threatened not to cover Aduhelm unless Biogen reduces its price. The plan has more resources and experience to review the drug faster than some plans with less institutional support, and Connolly said smaller plans will likely depend on the federal government’s decision.
Some Blue Cross Blue Shield affiliates have decided not to cover Aduhelm, saying the drug is still investigational and experimental, although Biogen has argued that the classification is a mischaracterization.
Although the majority of Aduhelm-eligible patients will be in the Medicare program, Chambers said the price and population of the drug still make it likely that commercial plans will eventually have an explicit policy on how to cover the drug, though he hopes that it will join Medicare contractors in waiting until after Medicare’s decision.
“State health insurance [decisions like this] they are generally reserved for high-priced drugs where there is the possibility of a real inconsistency, ”Chambers said. “With such a controversial drug as Aduhelm, I would be surprised if anyone wanted to get ahead.”