Jeff Borghoff’s face began to twitch and then drooped, knowing what was wrong. When he was only 51 years old, he was diagnosed with early-onset Alzheimer’s disease. His biggest fear at the time was that his intelligence deteriorated rapidly.
Desperate to control the condition, the former IT executive signed up for a clinical trial of Biogen’s experimental Alzheimer’s drug aducanumab. Six years later, Borghoff attributed the controversial treatment to the precious extra time he spent with his wife and three children.
“We have always worried that all my intelligence will drop sharply, but so far this has not been the case,” he said. “I have some symptom problems… But medication does require more time, more time with the person I love, these are all vital things.”
Now, after the U.S. drug regulatory agency approves the drug this week, many of the estimated 35 million Alzheimer’s patients worldwide will wait in line for their own hope.
However, for patients, this seems to be a definite good news. A range of issues, from the cost of medicines to questioning evidence of its efficacy, will put pressure on a health system that has been hit by the pandemic and may not be able to meet the expectations raised by people.
The approval of this drug may cause political and commercial disagreements between the United States (US$56,000 per year) and Europe, and many European governments may be skeptical of its value.
Complicating the debate is the growing outrage against the FDA Decided to give the green light to the drug First, given that there is little evidence for its efficacy. This week, three top scientists resigned from their advisory board, which is an extraordinary denial of the so-called objective and data-driven approval process.
Biogen initially abandoned the drug after an ineffective analysis showed that it was ineffective. But when it examined a larger data set, it found that patients taking higher doses “experienced significant benefits in cognitive and functional measures such as memory, orientation, and language.”
Northwestern University Hospital and Health Services Professor Craig Garthwaite (Craig Garthwaite) said that the FDA’s bowing to patient pressure groups and not listening to the opinions of its scientific advisers is “horrible”. He said he was “confused” by the “thought that we should try to give people hope even if we don’t know it or even if it doesn’t work.”
However, experts believe that now that the drug has been approved by the FDA, the health insurance company may feel obliged to cover the drug. Unexpectedly, the regulatory agency approved it for all patients with this disease—not just for early-stage disease patients studied in clinical trials.
Most American patients will use Medicare, a public health insurance company for people over 65, and Biogen told investors that it expects to cover “the vast majority” of patients. Some experts predict that this will soon become the largest medical insurance expenditure for doctors on medication.
The potential cost goes far beyond drugs. The urgency of managing it seems to challenge the existing dementia care model, requiring different types of staff and expensive equipment, which are usually not related to treating the disease.
According to Premier, who represents more than 4,000 US hospitals, the cost of intravenous infusion of drugs, radiology, and imaging may increase by US$2,000 to US$15,000 or more.
David Thomas, head of Alzheimer’s research policy in the United Kingdom, said, “A lot of care for patients with dementia [in the UK] It is done in memory clinics, which are mainly composed of geriatric psychiatrists.” Unlike neurologists, these doctors “usually do not have the diagnosis and monitoring experience needed to manage disease and improve treatment.”
He pointed out that another major challenge is to ensure the safety of the necessary equipment. The diagnosis of a person suspected of having this disease usually involves low-key cognitive tests and does not require special hardware.
In order to be eligible for aducanumab, the patient’s brain must have a certain level of amyloid, which can accumulate in tissues or organs, which is determined by a positron emission tomography (PET) scan or a more invasive scan Yes, but usually cheaper lumbar faucets, which involve removing fluid from the spine.
Eligible patients must receive an intravenous infusion of adukumab every 4 weeks and regular MRI scans to detect dangerous side effects such as brain swelling and bleeding.
Recently, the Alzheimer’s Research Center in the United Kingdom conducted a poll of psychiatrists in the country. “Most people say it takes up to five years to prepare for treatment-only one-third think they can Complete within one year,” Thomas said.
Sanjeev Sharma, a Boghof doctor and founder of the Advanced Memory Institute in New Jersey, admits that the obstacles ahead exist if all those who might benefit are to be benefited. But he firmly believes that the United States must blaze a trail. “If we can’t [here]As the most developed country in the world, where can we do it? “,He says.
“Financial incentives to use this drug”
Soeren Mattke, director of the Center for Improved Chronic Care at the University of Southern California, believes that few developed countries are currently fully prepared for the use of this drug-let alone middle-income and emerging countries, such as China and Brazil, whose technology is far less mature. . Health infrastructure.
However, he said that in the United States, funding for treatment will give birth to its own business model. Medicare pays the doctor 6% of the price of the drug, plus the cost of infusions and medical visits.
“The United States used to be very entrepreneurial… Neurologists, psychiatrists or geriatricians realized,’Well, I can actually live a good life by distributing drugs through my practice.’ This is the establishment The diagnostic facility is very attractive to find patients,” he said.
Northwestern’s Garthwaite said this may bring its own conflict of interest. “Even if you think it won’t work, there are practical economic motivations for using this drug.”
Matek also acknowledged that the same incentives do not exist in more stringent, publicly funded health systems such as Canada and the United Kingdom, where lengthy approvals and limited budgets may limit the purchase of equipment or the hiring of new employees. ability.
“I am a little nervous about the changes initiated by the government because we all know that the government will not act quickly,” he added.
In the United States, Biogen has helped prepare more than 900 infusion points in the United States to deliver drugs. It expects revenue in 2021 to be “moderate”, but there will be a multi-billion dollar opportunity in the next few years, as the United States alone has 1 to 2 million patients.
Roni Christopher, vice president of design and intervention of Premier’s analysis group, has been researching for the past year from developing standard assessments of patients’ cognitive status to training radiologists to detect side effects, and even assessing whether there are enough seats for infusion points.
Even with these logistical insights, the most experienced health systems may not be ready to treat patients until autumn. Christopher said that the FDA’s decision to open the drug to all patients with Alzheimer’s disease will lead to “a greater flood of interest.”
“Consumer or patient pressure will have a big impact here because this is a devastating disease,” she added.
Even more important than the question of how to provide the medicine is how many health systems — and the taxpayers who fund it — are willing to pay for this medicine with little proven impact.
In parts of Europe, the so-called “health technology assessment” is used to evaluate the cost-effectiveness of a new drug, but it has not been seriously involved in the issue of whether the drug should be provided to patients. In the UK, its National Institute of Health and Care Excellence has great international influence, and officials privately worry that rising expectations will not be easily realized.
Evercore ISI analyst Umer Raffat predicts that even if aducanumab is approved by the European authorities, they will “not allow a price of more than $10,000 per year”, which will put Biogen in a dilemma and decide whether to sell it to the United States at five times the European price. .
This difference in approach seems to anger Republican politicians, who believe that other countries negotiating cheaper drug prices are actually taking advantage of American innovations for free.
On the other side of the political channel, some Democrats are using the price set by Biogen as evidence that medical insurance should be allowed to negotiate prices — the government has chosen not to over-exercise its potential bargaining power with the pharmaceutical industry.
Murray Aitken, executive director of the Human Data Science Institute of IQVIA, New Jersey, believes that the question of how much the health system should pay for a drug, despite its flaws, offers rare hope, which may trigger The long overdue public liquidation is whether European governments should devote a higher proportion of GDP to health care.
He added: “In terms of how we distribute our wealth, this may prompt us as a civil society to engage in a larger dialogue… It may trigger a wider social debate and discussion, which I think may have expired. .”
At his home in New Jersey, Jeff Borghoff had no doubts about the value of the drug, and he believed it had prolonged his meaningful life. “I know this is not a cure, and it may not repair everything in my brain damaged by Alzheimer’s,” Burghof said. He added: “I am 57 years old. If I live to be in my 70s, I will be fine. It will be a good run. I hope the medicine will help me do this.”