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Jeff Borghoff knew something was wrong after his face contracted, then a fall. When at the age of only 51 he received the devastating diagnosis of early-onset Alzheimer’s disease, his main fear was a rapid deterioration of his mental powers.
Desperate to keep the condition at bay, the former IT executive enrolled in the clinical trial of Biogen’s Alzheimer’s experimental drug, aducanumab. Six years later, Borghoff attributes the controversial treatment to valuable extra time with his wife and three children.
“Our fear throughout everything was that there would be a sharp decline in all my mental faculties, but so far it hasn’t been that way,” he says. “I had some symptomatic problems. . . but the medication is really longer, longer with my loved ones, this is a critical thing ”.
Now, after the drug was approved this week by the U.S. drug regulator, many of the estimated 35 million living with Alzheimer’s worldwide will be waiting for their own infusion of hope.
Still, there is a sting in the tail of what might seem like unambiguous good news to patients. A range of issues, ranging from the cost of the drug to questions about the evidence for its effectiveness, will put pressure on health systems already affected by the pandemic and which may be ill-equipped to meet the expectations raised.
Drug approval runs the risk of opening up a political and business divide between the United States (where it has been priced at $ 56,000 a year) and Europe, where many governments may be much more skeptical about its value.
Complicating the debate is a growing heart of rage for the FDA decision to give the green light to the drug first, given the scant evidence of its effectiveness. Three prominent scientists resigned from their advisory board this week, in an extraordinary repudiation of a supposedly objective, data-based approval process.
Biogen originally abandoned the drug after a futility analysis suggested it was ineffective. But when he examined a larger data set, he found that patients taking a higher dose “experienced significant benefits in measures of cognition and function, such as memory, orientation, and language.”
Craig Garthwaite, a professor of hospitals and health services at Northwestern University, says it’s “terrible” that the FDA has bowed to patients’ pressure groups and hasn’t listened to their scientific advisors. He says he’s “baffled” by “this idea that even if we don’t know it or even if it doesn’t work, we should try it to give people hope.”
Still, experts believe health insurers are likely to be forced to cover the drug now that it has been approved by the FDA. In an unexpected turn, the regulator approved it for all patients with the disease, rather than only for those with an early stage of the disease, who were studied in the clinical trial.
Most U.S. patients will attend Medicare, the public health insurer for people over 65, which Biogen told investors it hopes to cover the “vast majority” of patients. Some experts predict it will quickly become Medicare’s largest spending on doctor-administered drugs.
The potential costs go far beyond medicine. The demands of administering it appear to challenge established patterns of care for people with dementia, creating the need for different types of expensive staff and equipment that are not usually associated with the treatment of the disease.
According to estimates by Premier, a group representing more than 4,000 American hospitals, the cost of intravenous infusion of medicine, radiology and imaging could add between $ 2,000 and $ 15,000 or even more to the price of the drug. .
David Thomas, head of policy at Alzheimer’s Research UK, says that “a lot of attention is paid to people with dementia [in the UK] it is done from memory clinics, which are largely attended to by psychiatrists of old age ”. Unlike a neurologist, these doctors “often have no experience in the diagnosis and supervision needed to administer treatments to modify the disease.”
The other big challenge, he points out, is getting the necessary equipment. Diagnoses for people suspected of having the disease usually involve low-level cognitive tests, which do not require any special hardware.
To be eligible for aducanumab, the patient must have a certain level in the brain of amyloids, proteins that can accumulate in tissues or organs, which is established by positron emission tomography (PET) or more. invasive, but usually cheaper lumbar faucet, which involves removing fluid from the spine.
Eligible patients should then undergo intravenous infusion once every four weeks with adducanumab and obtain periodic MRI scans that may detect dangerous side effects such as brain swelling and bleeding.
When Alzheimer’s Research UK surveyed psychiatrists in the country recently, “most said it would take five years to be ready to administer treatment; only a third thought they could do it in a year,” says Thomas.
Sanjiv Sharma, Borghoff’s physician and founder of the New Jersey Institute for Advanced Memory, recognizes the obstacles that remain to reach all those who could potentially benefit. But he has no doubt that the United States must begin the path. “If we can’t do it [here], as the most developed country in the world, where can we do it? ”He says.
“A financial incentive to use the drug”
Soeren Mattke, director of the University of Southern California’s Center for Improving Chronic Disease Care, argues that few developed nations are currently well-prepared to administer the drug, let alone emerging nations such as China and the United States. Brazil, with less sophisticated health infrastructure countries.
However, in the United States, ready-for-treatment funding will generate its own business models, he suggests. Medicare pays doctors a fee of 6% of the price of the drug, plus infusion and visitation fees.
“The United States is very enterprising, so once. . . neurologists, psychiatrists, or geriatricians realize that “well, I can really make a living from this just by distributing the drug through my consultation,” is a very powerful attraction for establishing diagnostic facilities for finding patients, ”he says.
This can lead to their own conflicts of interest, suggests Garthwaite of Northwestern. “There’s a real financial incentive to use the drug, even if you don’t think it works.”
Mattke also acknowledges that there are not the same incentives in more regimented and publicly funded health systems, such as Canada and the United Kingdom, where long approvals and limited budgets can limit the purchase of equipment or the ability to hire new staff. .
“I’m a little nervous about the change instituted by the government, because we all know that governments don’t act very quickly,” he adds.
In the United States, Biogen has helped prepare more than 900 infusion sites across the United States to administer the drug. It predicts “modest” revenue in 2021, but then a multimillion-dollar opportunity in the coming years, as it could give only 1 to 2 million patients in the U.S.
Roni Christopher, vice president of design and intervention for the Premier analysis group, has spent the past year examining everything from developing standard assessments of a patient’s cognitive status to training radiologists to detect side effects and even assess whether the infusion sites have sufficient capacity.
Even with these logistical ideas, more experienced health systems may not be ready to treat patients until the fall. The FDA’s decision to open the drug to all Alzheimer’s patients will lead to a “bigger flood” of interest, Christopher says.
“Consumer or patient pressure will dictate a lot here, because it’s such a devastating disease,” he adds.
Quality price value?
Even bigger than the question of how the drug should be administered is how many health systems – and the taxpayers who fund them – will be willing to pay for a drug with such a low impact.
In some areas of Europe, where so-called “health technology assessments” are used to assess the value for money of a new drug, the battle has not yet been seriously united over whether the drug should be offer to patients. In the UK, the National Institute for Health and Care Excellence has a lot of international influence, officials are privately concerned that expectations are being raised that are not easily met.
Umer Raffat, an analyst at Evercore ISI, hopes that even if aducanumab receives the green light from European authorities, “they will not allow a price of more than $ 10,000 a year”, which will put Biogen in the difficult position to decide whether to sell in the US at five times the European price.
This difference in approach seems to strengthen Republican politicians who believe that other countries that negotiate lower drug prices are in effect releasing American innovation.
Across the political aisle, some Democrats use the price Biogen has set as proof that Medicare should be allowed to negotiate prices, a potential bargaining power with the pharmaceutical industry the government chooses not to exercise too much.
Murray Aitken, executive director of the IQVIA Institute for Human Data Science in New Jersey, believes the question of how much health systems should pay for a drug that, while defective, offers an infrequent line of hope, it can lead to long-standing public recognition if European governments should spend a larger share of gross domestic product on health.
And he adds: “It can precipitate a larger conversation for us as a civil society, in terms of how we allocate our wealth… It can trigger this broader social debate and discussion, which I would say has probably lagged behind.”
At home in New Jersey, Jeff Borghoff has no doubt about the value of medicine, which he says has extended his lease for a meaningful life. “I know it’s not a cure and it probably won’t fix everything that’s been caused in my brain because of Alzheimer’s,” Borghoff says. And he adds, “I’m 57. I’ll be fine if I’m 70, it’ll work well and I hope the medication helps me do that.”
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