Face ef Borghoff knew that something was not right after his face was cracked and then he fell. When he was diagnosed with Alzheimer’s disease at an early age of 51, his greatest fear was a rapid deterioration in his mental capacity.
In desperation to keep the situation afloat, the former head of IT signed up for a clinical trial of aducanumab, a biogenic experimental Alzheimer’s drug. Six years later, Borghoff considers the controversial relationship with his wife and three children a valuable extra time.
“Our fear all along was that there would be a sharp decline in all my mental abilities, but so far that has not been the case,” he said. “I have had some symptomatic problems. “But medicine is actually more time, more time with my loved ones, they are important things.”
Now, after the US drug regulator approved the drug this week, many of the 35 million people with Alzheimer’s worldwide are waiting in line for their own hope injection.
But there is a sting in the tail that may seem like good news to patients. A number of issues, from the cost of drugs to questions about their effectiveness, will put pressure on health systems already affected by the epidemic, which may be poorly equipped to raise expectations.
Drug approval runs the risk of opening political disputes between businesses and the United States (where $ 56,000 a year is set) between Europe and Europe, where many governments may be much more skeptical of its value.
Complicating the debate is a growing anger at the FDA decision to put the medicine in the green light first of all, given the limited evidence of its effectiveness. Three leading scholars resigned from their advisory board this week, ostensibly objectively rejecting the data-based approval process.
Biogen initially abandoned the drug after a useless analysis suggested it was ineffective. But when he looked at a larger body of data, he found that patients taking higher doses “had significant benefits over cognitive functions such as memory, orientation, and language.”
Craig Garthwaite, a professor of hospital services at Northwestern University, said it was “appalling” that the FDA bowed to patients’ pressure groups and did not listen to its scientific advisers. He says he is “puzzled” by “the idea that even if we do not know or even if it does not work, we should try to give people hope.”
However, experts believe that health insurers will most likely feel obligated to cover the drug now that it has been approved by the FDA. Unexpected reversal was confirmed by the regulator for not all patients with early-onset disease և who have been studied in clinical trials.
Most US patients will be on Medicare, a public health insurer over 65, which Biogen told investors it expects to cover the “overwhelming majority” of patients. Some experts predict that it will quickly become the largest expense for Medicare over-the-counter medications.
Potential costs exceed only drugs. The urgency of running it seems to challenge the caregivers approved for mental care, creating the need for a variety of staff և expensive equipment that is not normally associated with the treatment of the disease.
The cost of an intravenous injection of medication, radiation, or imaging can add up to $ 2,000 to $ 15,000 or more, according to a Premier Group estimate of more than 4,000 hospitals in the United States.
David Thomas, head of Alzheimer’s research policy in the UK, says “a lot of care for people with dementia [in the UK] It is performed by memory clinics, which are mainly run by old age psychiatrists. ” Unlike a neurologist, these doctors “often do not have the diagnostic and monitoring experience needed to treat the disease.”
The other big challenge, he says, is providing the necessary equipment. Diagnosis of suspected disease usually involves low-level cognitive tests that do not require special equipment.
To be eligible for aducanumab, a patient must have certain levels of amyloid, a protein that can accumulate in tissues or organs, which is confirmed by either positron emission tomography (PET) scan or a more invasive but usually cheaper, lumbar valve. is the removal of spinal fluid.
Eligible patients should undergo an intravenous infusion of aducanumab every four weeks, and have regular MRI scans that may detect dangerous side effects, such as swelling of the brain or bleeding.
“When the UK recently conducted a survey of Alzheimer’s patients among psychiatrists in the country, ‘the majority said they would be ready for treatment for up to five years, only a third thought they could do it within a year,'” said Thomas.
Sanjiv Sharma, Dr. Borghoff հիմն Founder of the New Ersey Institute for Advanced Memory, acknowledges the obstacles ahead if he wants to reach everyone who can benefit. But he has no doubt that the United States should burn the trail. “If we can not do it [here]”As the most developed country in the world, where can we do that?” he said.
“Financial incentive to use drugs”
Soren Metke, director of the Center for Chronic Disease Improvement at the University of Southern California, says few developed countries are ready to use the drug, especially in middle-income developing countries such as China and Brazil, which have much more sophisticated health facilities.
However, the United States is ready to create its own business models for treatment funding, he suggests. Medicare pays doctors up to 6% of the cost of the drug plus infusion և visits.
“The United States is an entrepreneur at once. ,, neurologists or psychiatrists or geriatricians realize. “Well, I can make a better living from it just by distributing drugs through my practice,” he said. “This is a very powerful game to create diagnostic tools to find patients,” he said.
This could lead to a conflict of interest, says Garthwaite of Northwester. “There is a real financial incentive to use drugs, even if you do not think it will work.”
Matke acknowledges that the same incentives do not exist in more global, publicly funded health care systems, such as Canada and the United Kingdom, where long-term approvals, limited budgets can limit access to equipment or hire new staff.
“I am a little nervous about the change approved by the government, because we all know that governments do not act very quickly,” he added.
In the United States, Biogen has helped prepare more than 900 infusion sites in the United States to deliver the drug. It forecasts “modest” revenue in 2021, but then a multi-billion dollar opportunity years later, as it could only reach 1 to 2 million patients in the United States.
Ronnie Christopher, vice president of design and intervention for the Prime Minister’s Analytical Group, spent the past year developing everything from standard patient cognitive assessments to training radiologists to detect side effects and even assessing if infusion sites are adequate. ,
Even with these logistical insights, even the most experienced health care systems may not be ready to treat patients until the fall. Christopher says the FDA’s decision to open a drug for Alzheimer’s patients will lead to a “bigger flood” of interest.
“The pressure of the consumer or the patient will dictate a lot here, because it is such a devastating disease,” he added.
Value for money?
Even bigger than the question of how the drug should be delivered is how many health care systems ֆինանս the taxpayers who fund them will be willing to pay for such a drug that has so little proven effect.
In parts of Europe where so-called “health technology assessments” are used to assess the cost of a new drug, there is still a serious struggle over whether the drug should be offered to patients. In the UK, where the National Institutes of Health’s Excellence in Health has great international influence, officials are concerned that expectations are rising that are not easily met.
Evercore ISI analyst Umer Rafat expects that even if aducanumab gets the green light from the European authorities, they “are not going to allow more than $ 10,000 a year”, which will put Biogen in a difficult position to decide to sell in the US. at five times the European price.
This difference in approach seems likely to inflame Republican politicians who believe that other countries negotiating cheaper prices are actually releasing American innovations.
Across the political corridor, some Democrats are using the price that Biogen has set as proof that Medicare should be allowed to negotiate prices. A potential bargaining chip with the drug industry that the government does not use much.
Murray Aitken, executive director of the IQVIA Institute for Human Data Science in New Ersey, believes that the question of how much health care systems should pay for a drug that, while flawed, inspires rare hope, could lead to a long-awaited public outcry. Should European governments spend more of their gross domestic product on health?
He adds: “It simply came to our notice then that we, as a civil society, could talk about how we spend our wealth.” “It could provoke that wider social debate, which I would say is probably out of date.”
Jeff Borghoff has no doubt about the value of the drug, which he believes has extended his rent for a meaningful life. “I know this is not a cure, it probably won’t fix everything that has damaged my brain from Alzheimer’s,” Borghoff said. He adds: “I am 57 years old. “I will be fine if I live to be 70, it will be a good result. I hope the medicine will help me do that.”