Jeff Borghoff knew something wasn’t going right after his face developed a contract, after a fall. When at the age of only 51, he received the devastating diagnosis of Alzheimer’s disease at an early stage, his greatest fear was a rapid deterioration of his mental powers.
Desperate to keep the condition at bay, the former IT executive stopped by for the clinical trial of Biogen’s experimental drug, Alzanumab. Six years later, Borghoff attributed the controversial treatment to a precious extra time with his wife and three children.
“Our fear all the time was that there was going to be a sharp decline in all my mental faculties, but to date that has not been the case,” he says. “I’ve had some symptomatic problems… But the medication is really longer, more time with my loved ones, they’re critical things.”
Now, following the approval of the drug this week by the U.S. drug regulator, many of the estimated 35m people living with Alzheimer’s around the world are waiting in line for their own infusion of hope.
However, there is a punctuation on what may seem like good news without ambiguity for patients. A range of problems, ranging from the cost of the drug to questions about evidence of its effectiveness, will accumulate pressure on health systems already battered by the pandemic and which may be ill-equipped to meet expectations. which have been raised.
Drug approval risks opening up a political and trade divide between the United States – where a price of $ 56,000 a year has been set – and Europe, where many governments may be much more skeptical about to its value.
Completing the debate is a growing chorus of anger for the FDA green light decision to drug first, given the lack of evidence of its effectiveness. Three top scientists resigned from their advisory board this week, in an extraordinary reversal of an objective assumption and data-driven approval process.
Biogen initially abandoned the drug after a futility analysis suggested it was ineffective. But when he examined a larger data set, he found patients taking a higher dose “have experienced significant benefits on measures of cognition and function such as memory, orientation and language.”
Craig Garthwaite, a professor of hospital and health services at Northwestern University, says it’s “terrible” that the FDA has bowed to patients’ pressure groups and has not listened to its scientific advisors. He says he is “perplexed” by “this idea that even if we don’t know it or even if it doesn’t work, we have to try it to give hope to people.”
However, experts believe that health insurers feel compelled to cover the drug now that it has been approved by the FDA. In an unexpected turn, the regulator approves it for all patients with the disease – rather than just those who have the initial stage of the disease, who have been studied in the clinical trial.
Most American patients will be on Medicare, the public health insurance company for more than 65 years, which Biogen told investors expects to cover the “vast majority” of patients. Some experts predict that it will quickly become the largest Medicare spending for administered drugs.
The potential costs go far beyond just medicine. Management needs seem willing to challenge established models of care for those with dementia, creating a need for different types of staff and expensive equipment that are not typically associated with the treatment of the disease.
The cost of intravenous infusion of medicine, radiology and imaging could add between $ 2,000 and $ 15,000 or even more on the price of the drug, according to estimates by Premier, a group that represents more than 4,000 American hospitals.
David Thomas, head of policy for Alzheimer’s Research UK, says “a lot of care for people with dementia [in the UK] it is done by memory clinics, which are largely customized by elderly psychiatrists. ”Unlike a neurologist, these doctors“ often do not have the diagnostic and follow-up experience needed to administer disease-modifying treatments. ” .
The other big challenge, he notes, is securing the necessary equipment. Diagnosis for those suspected of having the disease usually involves low-key cognitive tests, which do not require special hardware.
To be eligible for adducanumab, a patient must have a certain level in their brain of amyloid, a protein that can accumulate in tissues or organs, which is established both with a positron emission tomography (PET) and at most. invasive, but typically more economical, lumbar tap, which involves the removal of fluid from the spine.
Admissible patients should then undergo intravenous infusion once every four weeks with adducanumab and obtain regular MRI scans that may reveal dangerous side effects such as swelling of the brain and bleeding.
When Alzheimer’s Research UK recently interviewed psychiatrists in the country, “the majority said it would take up to five years to be ready to administer treatment – only a third thought they could do it in a year.” says Thomas.
Sanjiv Sharma, Dr. Borghoff and founder of the New Jersey Institute of Advanced Memory, recognizes the obstacles ahead to reach all those who could benefit. But he has no doubt that the United States should pave the way. “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 drugs”
Soeren Mattke, director of the Center for Improving the Treatment of Chronic Diseases at the University of Southern California, argues that few developed nations are currently well prepared to administer the drug – even fewer middle-income and emerging nations such as and China and Brazil with much less sophisticated health infrastructure.
However, in the United States, ready-to-treat funding will generate their own business models, he suggests. Medicare pays doctors a 6 percent tax on the price of the drug, plus infusion and visitation fees.
“The United States is very entrepreneurial so once… Neurologists or psychiatrists or geriatricians realize, ‘well, I can really make a good living out of this just by distributing the drug because of my practice,’ it’s a very powerful tool for set up diagnostic structures to find patients, ”he says.
This can lead to their conflicts of interest, suggests Garthwaite of Northwestern. “There’s a real financial incentive to use the drug even if you don’t think it’s going to work.”
Mattke also acknowledges that the same incentives do not exist in more registered, publicly funded health systems such as Canada and the United Kingdom, where long approvals and limited budgets may limit the purchase of equipment, or the ability to employ new staff.
“I’m a little nervous about the change instituted by the government because we all know that governments don’t act very fast,” he adds.
In the United States, Biogen has helped prepare more than 900 infusion sites in the United States to supply the drug. He anticipates “modest” returns in 2021, but after a multimillion-dollar opportunity in the years to come, as could be given to between 1m and 2m of patients in the United States alone.
Roni Christopher, vice president of conception and intervention in the Premier’s analytical group, spent the last year examining everything from developing standard assessments of a patient’s cognitive status to training radiologists to detect side effects, as well as assess whether infusion sites have sufficient places.
Even with these logistical insights, the most experienced health systems can 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, says Christopher.
“Consumer or patient pressure will dictate a lot here, because it’s such a devastating disease,” he adds.
Value for money?
Bigger, though, than the question of how the drug should be delivered is how much the health systems – and the taxpayers who fund them – will be willing to pay for a drug with such a unproven impact.
In parts of Europe, where so-called “health technology assessments” are being used to assess the value for money of a new medicine, the battle has not yet been seriously united to know whether the drug should be offered to patients. In the UK, where its National Institute for Excellence in Health and Care carries a lot of international influence, officials are privately concerned that expectations are being raised which will not be easily met.
Umer Raffat, an analyst at Evercore ISI, expects that even if indiranumab receives the green light from European authorities, it will not “allow a price of more than $ 10,000” a year, which will put Biogen in the difficult position of deciding if to sell to the United States at five times the European price.
This difference in approach seems to inflame Republican politicians who believe that other countries that negotiate lower drug prices are in fact in charge of American innovation.
On the other side of the political spectrum, some Democrats use the price that Biogen has set as proof that Medicare should be allowed to negotiate prices – a potential bargain with the drug industry that the government chooses not to. too much exercise.
Murray Aitken, executive director of the IQVIA Institute for Human Data Science in New Jersey, believes that the question of how much health care systems should pay for a drug that, while defective, offers a rare well of hope, may induces a long public bill if European governments are to spend a higher share of gross domestic product on health.
He adds: “It can precipitate a wider conversation for us as a civil society, in terms of how we attribute our wealth…. It can initiate this broader social debate and discussion, which I will say is probably delayed. “.
At home in New Jersey, Jeff Borghoff has no doubt about the value of medicine which he believes has extended his tenure for a significant life. “I know it’s not a cure and it probably won’t fix everything that has been damaged in my brain by Alzheimer’s,” says Borghoff. He added: “I am 57 years old. I will be fine if I live to be in my 70s, it will be a good race and I hope the medicine will help me do it.”