Jeff Burghoff knew something was wrong after his face got ecstatic and then drooped. When he was just 51 years old, receiving the devastating early Alzheimer’s diagnosis, his biggest fear was the rapid decline in his mental powers.
In a desperate attempt to keep the condition at bay, the former IT executive signed up for the clinical trial of Biogen’s experimental Alzheimer’s drug, aducanumab. Six years later, Burghoff credits the controversial treatment to precious extra time with his wife and three children.
“Our fear all along was that there was a severe deterioration in all my mental faculties, but so far it hasn’t been,” he says. “I’ve had some issues with symptoms…but the medication is really taking longer, more time with my loved ones, these are very important things.”
Now, with the drug approved this week by the US drug regulator, many of the estimated 35 million people with Alzheimer’s disease worldwide will wait in line for an infusion of hope.
However, there is a sting in the tail about what may seem to be unequivocal good news for patients. A series of issues, ranging from the cost of a drug to questions about evidence of its efficacy, will put more pressure on health systems already hit by the pandemic that may be ill-equipped to meet the expectations raised.
Approval of the drug threatens to open a political and commercial chasm between the United States – where the price has been set at $56,000 a year – and Europe, where many governments may be more skeptical of its value.
Complicating the debate is the growing chorus of anger at the Food and Drug Administration The decision to give the green light to the drug Primarily, given the paucity of evidence for its effectiveness. Three prominent scientists resigned from its advisory board this week, in an unusual rejection of an objective, data-driven presumed consent process.
Biogen originally abandoned the drug after a non-viability analysis indicated it was ineffective. But when she examined a larger set of data, she found that patients who took the higher dose “experienced significant benefits on measures of cognition and function such as memory, orientation, and language.”
It’s “terrible” that the FDA has acquiesced to patient pressure groups and not listened to their scientific advisors, says Craig Garthwaite, a professor of Hospital and Health Services at Northwestern University. He says he’s “baffled” by “this idea that even if we don’t know or even if it doesn’t work, we should try to give people hope.”
However, experts believe that health insurance companies will likely feel compelled to cover the drug now that it has been approved by the Food and Drug Administration. In an unexpected development, the regulator has approved it for all patients with this condition — not just those with early-stage disease, who were studied in the clinical trial.
Most patients in the United States will be on Medicare, the public health insurer for the over-65s, which Biogen told investors it expects to cover the “vast majority” of patients. Some experts predict that it will become more and more quickly Medicare spending on physician-administered medications.
The potential costs go beyond just medication. The exigencies of his administration appear to challenge established patterns of care for people with dementia, creating a need for different types of staff and expensive equipment not usually associated with treating the disease.
The cost of intravenous medications, radiology, and imaging can add between $2,000 and $15,000 or more on top of the drug’s price, according to estimates by Premier, a group that represents more than 4,000 hospitals in the United States.
There is a lot of care for people with dementia, says David Thomas, head of policy at Alzheimer’s Research UK. [in the UK] It is performed out of memory clinics, which are staffed largely by aging psychiatrists.” Unlike a neurologist, these doctors “often do not have the expertise in diagnosis and monitoring required to administer disease-modifying therapies.”
He points out that the other big challenge is securing the necessary equipment. Diagnosis of people suspected of having the disease usually includes low-level cognitive tests, and does not require special equipment.
In order for a patient to be eligible for Aducanumab, the patient must have a certain level in their brain of amyloid, a protein that can build up in tissues or organs, created either through a positron emission tomography (PET) scan or a more invasive scan, but usually What is the cheapest lumbar puncture, which involves removing fluid from the spine.
Eligible patients should then undergo an intravenous infusion every four weeks with aducanumab and have MRI scans that can detect serious side effects such as brain swelling and bleeding.
When Alzheimer’s Research UK recently surveyed psychiatrists in the country, Thomas said: “The majority said it would take up to five years to be ready to administer treatment – only a third thought they could do so within a year.”
Sanjeev Sharma, MD, a Borghoff physician and founder of the Institute for Advanced Memory in New Jersey, acknowledges the obstacles that lie ahead if all those who could benefit are to be reached. But he does not doubt that the United States should lead the way. “If we can’t do that [here]As the most advanced country in the world, where can we do this? ‘, he says.
A financial incentive to use the drug
Soeren Mattke, director of the Center for Improved Chronic Disease Care at the University of Southern California, argues that few developed countries are currently well prepared to administer the drug — let alone middle-income and emerging countries like China and Brazil with much less development. health infrastructure.
However, it is suggested that treatment-ready funding in the United States will generate business models of its own. Medicare pays doctors a fee of 6 percent of the drug’s price, plus infusion and visit fees.
“The United States is a very entrepreneurial country so once upon a time . . . neurologists or psychiatrists or geriatricians realize, ‘Well, I can actually make a good living out of this just by distributing medicine across my practice,’ and that’s a very strong pull for establishing diagnostic facilities to find patients,” he says.
This could lead to his own conflict of interest, suggests Northwestern’s Garthwaite. “There is an actual financial incentive to use the drug even if you don’t think it will work.”
Matkey also acknowledges that the same incentives do not exist in more regulated and publicly funded health systems such as Canada and the United Kingdom, where lengthy approvals and tight budgets may restrict equipment purchases or the ability to hire new staff.
“I’m a bit concerned about the government-imposed change because we all know governments don’t act very quickly,” he adds.
In the United States, Biogen has helped set up more than 900 infusion sites across the United States for drug delivery. It expects “modest” revenue in 2021, but then a multibillion-dollar opportunity in the coming years, as it could be awarded to between one and two million patients in the United States alone.
Ronnie Christopher, vice president of design and intervention in the Analytics Group at Premier, has spent the past year examining everything from developing standard assessments of a patient’s cognitive status to training radiologists to spot side effects, to assessing whether injection sites have enough seating.
Even with these logistical insights, the most experienced health systems may not be ready to treat patients until the fall. Christopher says the FDA’s decision to open the drug to all Alzheimer’s patients, will lead to a “bigger deluge” of interest.
“Consumer or patient pressure is going to dictate a lot here, because it’s a devastating disease,” she adds.
value for money?
The bigger, and even more so, question of how the drug will be delivered is how much health systems — and the taxpayers who fund them — are willing to pay for a drug with little proven effect.
In parts of Europe, where so-called “health technology assessments” are used to assess a new drug’s value for money, the battle has yet to be seriously joined over whether the drug should be offered to patients. In the UK, whose National Institute for Healthcare Excellence and much international influence, officials are particularly concerned that expectations being raised will not be easily met.
Omar Raafat, an analyst at Evercore ISI, predicts that even if aducanumab gets the go-ahead from European authorities, they “would not allow a price of more than $10,000” a year, which would put Biogen in a difficult position to decide whether to sell it to the United States at five times the price. European.
This difference in approach appears to stoke Republican politicians who believe that other countries negotiating cheaper drug prices are actually dispensing with American innovation.
On the other side of the political aisle, some Democrats are using the price set by Biogen as evidence that Medicare should be allowed to negotiate prices — a potential bargaining power with the drug industry that the government chooses not to exercise either.
Murray Aitken, executive director of the IQVIA Institute for Human Data Science in New Jersey, believes that the question of how much health systems should pay for a drug, however flawed, offers a rare glimmer of hope, which may prompt people to consider their long-awaited reckoning. Whether European governments should spend a larger share of GDP on health.
He adds, “It might precipitate a larger conversation for us as a civil society, as to how we should allocate our wealth . . . it might lead to broader social debate and discussion, which I would say may be overdue.”
In his New Jersey home, Jeff Burghoff has no doubts about the value of the drug he believes has extended his lease for a meaningful life. “I know it’s not a cure, and it probably won’t cure everything that was damaged in my brain from Alzheimer’s,” Burghoff says. He adds, “I’m 57. I’d be fine if I lived in my 70s, that would be fine and I hope medication helps me do that.”