COMMENT: IMHO this drug, given its dangerous side-effects of brain swelling and unproven effectiveness (Only one out of two limited trials showed any sign of improvement.), should not have been approved yet without more and wider testing. The FDA did approve it against the advice it received.)
The Alzheimer’s treatment will cost $56,000 per patient, and millions may use it. The result: “crazy numbers” for Medicare.
By Josh Katz, Sarah Kliff and Margot Sanger-Katz
June 22, 2021
Updated 10:09 a.m. ET
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A newly approved drug to treat Alzheimer’s disease is expected to become a multibillion dollar expense for Medicare. By one projection, spending on the drug for Medicare’s patients could end up being higher than the budgets for the Environmental Protection Agency or NASA.
There’s little evidence that the drug, Aduhelm, slows the progression of dementia, but the Food and Drug Administration approved it this month. Analysts expect that Medicare and its enrollees, who pay a share of their prescription drug costs, will spend $5.8 billion to $29 billion on the drug in a single year.
“It’s unfathomable,” said Tricia Neuman, executive director of the Kaiser Family Foundation’s program on Medicare policy. “These are crazy numbers.”
Plenty of other drugs cost more than Aduhelm, which is made by Biogen and will be priced at $56,000 annually. What makes it different is that there are millions of potential customers, and the drug is expected to be taken for years.
The drug’s approval has aroused criticism from health policy experts and pharmaceutical researchers for its lack of proven effectiveness. Effective or not, if widely prescribed, it could have an overwhelming impact on Medicare’s budget because the public program covers the vast majority of the nearly six million Americans with an Alzheimer’s diagnosis.
There is little precedent for a sudden spending jolt of this size. Even at the low end of projections, Aduhelm would become one of Medicare’s most expensive drugs.
At the high end, analysts say the new drug could cause a 50 percent increase in Medicare’s annual spending on drugs delivered in hospitals and doctor’s offices (as Aduhelm, which is given intravenously, would need to be).
The comparisons here are approximate: A third of Medicare enrollees are covered through private Medicare Advantage plans that do not release detailed information on the drugs provided in doctor’s offices. To estimate that spending, we used the drug spending data for Medicare enrollees in the traditional public program and increased it to account for the missing share.
Spending on this scale, so suddenly, could have far-reaching impacts for Medicare, its users and taxpayers. The addition of $29 billion a year to Medicare’s budget would be paid for by increases in both taxpayer spending and in the premiums paid by all Medicare users. Premiums might also go up for supplemental plans many Medicare beneficiaries buy to offset costs the program doesn’t pay directly. And the costs are likely to spill over into state budgets, where Medicaid pays premiums for low-income Medicare enrollees.
Congress, budget experts and several White Houses have spent years suggesting ways to trim spending in Medicare, a large and growing share of the federal budget. But many of these proposals are politically difficult to achieve — and most would save less than the projected cost of Aduhelm.
“It’s so much work to get savings that are really much smaller than this one drug would cost,” said Joshua Gordon, the director of health policy at the Committee for a Responsible Federal Budget, who says he has found himself thinking nonstop about the challenges raised by Aduhelm since its approval.
Cost predictions vary because analysts aren’t sure how many patients will ultimately use the new drug. The F.D.A.’s approval could apply to everyone diagnosed with Alzheimer’s disease. But the drug was developed for a smaller group of around 1.5 million patients who are in the early stages of the disease. Analysts aren’t yet sure whom doctors will recommend the treatment for, and which families will want to try it. The F.D.A. has asked Biogen to continue studying the drug until 2030, but prescribing could become widespread before there are any further public results on how well it works.
Allison Parks, a Biogen spokeswoman, said in an email that the company would focus on reaching the type of patients who were studied in the company’s clinical trials, “in the early symptomatic stage of the disease.”
The range reflects a variety of reasonable expert estimates. The high estimate, drawing on a Kaiser paper, assumes that about a quarter of the two million Medicare enrollees who currently take an Alzheimer’s treatment will take this one. The low one is based on a Cowen and Company analyst estimate of $7 billion in total sales by 2023.
Estimating how many patients will use the drug is challenging. Aduhelm is not just expensive, but also somewhat hard to take, requiring monthly in-person visits to an infusion center for treatment. Patients who take it will be required to get multiple brain scans during their treatments to look for side effects.
And the side effects themselves — about 40 percent of patients in one clinical trial showed signs of brain swelling — may discourage some patients from trying the drug, and prompt others to stop taking it. (The many scans — and treatments for more serious side effects — would also be covered by Medicare.)
There are six million Medicare enrollees who do not purchase supplemental coverage who could have to pay 20 percent of the drug’s cost, in this case $11,200 a year.
Demand may nevertheless be high from families who see an opportunity to intervene when faced with a devastating diagnosis. Until now, there have been few treatment options available for patients hoping to forestall cognitive decline from the disease.
“There is something intrinsically hard about having a loved one, seeing the clock ticking, and saying, Well, let’s just wait,” said Dr. Steven Pearson, a primary care physician and the president of the Institute for Clinical and Economic Review (ICER). “It’s very hard to ignore the drive to do something.”
Doctors, who would administer this drug and be paid a percentage of the drug’s high price by Medicare for that work, may face financial incentives to say yes when patients ask for it.
“The implications of this one drug and the associated set of procedures are enormous,” said Rachel Sachs, a law professor at Washington University in St. Louis and an author of a recent essay in The Atlantic asserting that the drug could “break American health care.”
Private insurers may erect roadblocks to treatment, requiring patients to get additional tests or prove that other options haven’t worked. But in normal circumstances, Medicare covers drugs that are approved by the F.D.A. Medicare decides what drugs to cover based on whether they are “reasonable and necessary,” not on how much they cost.
Medicare is initially required to pay for this type of drug at its list price in addition to a 3 percent fee to the doctor who gives it. And then, after about a year on the market, it pays the average sales price plus 6 percent. For drugs with competition, that average price can be substantially lower than the sticker price. But for a drug like Aduhelm, which is the first of its kind, the drugmaker may not offer doctors discounts.
Medicare, which covers 61 million Americans 65 and over, does have some tools to contain costs. It could decide to cover the drug in a way that is more limited than the F.D.A. approval, a break from its normal practice.
Or it could do something even more unusual: An unexpected alliance of advocates has suggested that Medicare put the drug into a randomized experiment to evaluate how well it works — paying to cover the drug in some parts of the country, but not others. Such policy experiments were authorized under the Affordable Care Act, but one has never been used to limit coverage of a drug in this way.
Other countries will most likely control the cost of Aduhelm by negotiating with Biogen for a lower price, or simply decline to buy it at all. Most will consider the drug’s effectiveness when deciding what they are willing to pay. So far, the drug has not been approved for use anywhere else in the world.
Medicare can’t do that. Because of the way it pays for drugs under current law, it has no way to bargain down the price. Democrats increasingly support legislation to change that. The House passed legislation in 2019 that would give Medicare the authority to negotiate some prices, but it died in the Senate. Legislators reintroduced the same bill in the House in April.
President Biden supports allowing Medicare to negotiate drug prices but did not include the policy in his proposed American Families Plan.
Dr. Pearson of ICER has estimated that if the new drug’s effectiveness were taken into account, a fair price would be $2,500 to $8,300.
“It will be interesting to see if this starts a discussion about fair pricing in the United States,” he said. “To most people’s eyes, this looks like an outstanding example of a price that just does not match up with the evidence.”
https://www.nytimes.com/2021/06/22/upsh ... e=Homepage
New Drug Could Cost the Government as Much as It Spends on NASA
Re: New Drug Could Cost the Government as Much as It Spends on NASA
A New Alzheimer’s Drug Offers More Questions Than Answers
The approval of Aduhelm to treat Alzheimer’s disease has raised hope among older adults, but many doctors wonder if it is warranted.
By Paula Span
July 7, 2021
Dr. Kenneth Koncilja, a geriatrician at the Cleveland Clinic, saw the announcement from the Food and Drug Administration on June 7, on Twitter: The agency had approved Aduhelm (aducanumab), the first drug to treat Alzheimer’s disease to be approved in nearly 20 years.
The calls from patients’ spouses and family members began within the hour, and have not stopped. “I was shocked at how fast the word spread — ‘Hey, is this something we can use? When can we get it?’” Dr. Koncilja recalled. “There’s a mix of excitement, anxiety and desperation.”
His first call that morning came from Joan Morehouse, 78, who has been caring for her 71-year-old husband, James, in their home in North Perry, Ohio, since his Alzheimer’s diagnosis four years ago. She has watched him get lost on familiar drives and forget their grandchildren’s names.
When her brother and her son both emailed her a news article about the F.D.A. action, she recalled, “I said, ‘Oh, my God, my prayers have been answered.’”
It fell to Dr. Koncilja to explain the complexities: That Aduhelm is not yet widely available. That protocols determining which patients qualify have yet to be developed. That the clinical trial data was ambiguous and that the drug might bring no noticeable improvements in daily life. That its side effects include brain swelling and bleeding.
And that its maker, Biogen, estimates the annual cost of monthly intravenous infusions at $56,000, plus expensive scans and tests.
“It’s a more difficult question than I’ve ever had before,” Dr. Koncilja said. Patients ask him how their lives will change, “and I don’t know how to answer.”
In the weeks since the F.D.A.’s action, which places virtually no restrictions on prescribing the drug, geriatricians, neurologists and other doctors across the country have been fielding similar questions.
Aduhelm has generated intense controversy. Biogen stopped two trials in 2019 because they demonstrated no benefit, then submitted an F.D.A. application after a later analysis of one trial showed slightly slower cognitive decline at high doses.
In a letter to the F.D.A., the American Geriatrics Society argued that approval was “premature given the lack of sufficient evidence.” The Society for Post-Acute and Long-Term Care Medicine later came to a similar conclusion.
The F.D.A.’s own advisory committee strongly recommended against approval, and three member scientists resigned in protest when the agency overrode its advice. A new survey of 200 neurologists and primary care doctors has found that most disagreed with the F.D.A. decision.
Senators Elizabeth Warren, Democrat of Massachusetts, and Bill Cassidy, Republican of Louisiana, have called for a hearing, concerned that spending billions on Aduhelm could undermine Medicare. The House Committee on Oversight and Reform has announced an investigation into the drug’s approval and pricing.
Given all that, should older adults consider Aduhelm? “The F.D.A. has passed the determination along to the American family,” said Dr. Jason Karlawish, co-director of the Penn Memory Center, who, with a number of other doctors, publicly opposed the drug’s approval.
Penn Memory doctors are receiving anxious inquiries, too. Geeta Simons, a musician in Philadelphia whose 80-year-old father has Alzheimer’s, messaged her father’s neurologist there. “I wanted to believe that this was that magical save,” she said.
Such doctors face “a dilemma,” Dr. Karlawish said, “a moment when there’s no decision that resolves all the uncertainties and settles the ethical concerns.”
“It puts us in a bad place,” agreed Dr. Karina Bishop, a geriatrician at the University of Nebraska Medical Center. Ethically, she added, “if this drug was available right now, I would not feel able to prescribe it.”
Even as individual doctors grapple with advising patients, hospitals and health systems are devising protocols for when Aduhelm becomes more widely available, probably within weeks.
At the Mayo Clinic, said Dr. Ronald Petersen, a neurologist who directs the Alzheimer’s Disease Research Center there, “we’re going to stick pretty close to the inclusion and exclusion criteria used in the trial.”
That means only patients with mild cognitive impairment or early Alzheimer’s disease would qualify, after an M.R.I. to rule out certain conditions and risks, and a P.E.T. scan or lumbar puncture to confirm the presence of amyloid. The Mayo protocols, like the clinical trials, would exclude people taking blood thinners like Warfarin or Eliquis.
“It’s not like you come in and say, ‘I’m a little forgetful,’ and we say, ‘Here’s this drug,’” said Dr. Petersen. But not every provider, he acknowledged, will employ such safeguards.
Dr. Eric Widera, a geriatrician at the University of California, San Francisco, expressed a similar concern: “If doctors were extremely cautious and limited this drug to the very specific population included in the study, with very careful monitoring, it would be the first time in medicine that was ever done.”
He pointed out another consequence of federal approval: a rift between some doctors and the Alzheimer’s Association, the national advocacy group, which this spring mounted a campaign it called More Time. Intended to demonstrate public support for approval of aducanumab, the effort included newspaper ads and social media posts.
Now Dr. Widera, who has worked with a local chapter to train medical students and residents, is seeking an alternate source of information to which to refer patients. He has come to mistrust the Alzheimer’s Association, calling it “a big promoter, almost a marketer, for Biogen,” which, like other pharmaceutical firms, helps underwrite the organization and contributed $275,000 to it last year.
The association said in an email that “history has shown us that approvals of the first drug in a new category will invigorate the field, increase investments in new treatments and generate greater innovation.”
One major unpredictable factor in Aduhelm’s future: insurance coverage. Medicare could decide to authorize coverage as “reasonable and necessary,” to deny or limit it, or to delay a decision. A spokesman at the Centers for Medicare and Medicaid Services said it was reviewing the F.D.A.’s decision and would have more information soon.
Given the drug’s announced price tag, a restrictive Medicare policy could put it beyond reach for most older Americans.
Eventually, the F.D.A. might also take action against Aduhelm. Its “accelerated approval” process requires Biogen to undertake a new clinical trial; if that shows no benefit, the agency could withdraw approval. But Biogen has until 2030 to report those results; by then, thousands of hopeful patients might already be taking Aduhelm.
For now, doctors are wrestling with how to respond.
“One of my core principles is respect for patient autonomy, especially for this disease, which degrades a patient’s ability to exercise self-determination,” said Dr. Karlawish. Slightly softening his published opposition to Aduhelm, he said that he now would prescribe it, after extensive discussions with patients, “but I’d be a reluctant prescriber.”
Several doctors described gently dissuading patients by noting the uncertainty that the drug would help, the potentially disabling side effects and the many unknowns. “They have been open to waiting and getting more information,” Dr. Bishop said.
Ms. Morehouse, for instance, had heard nothing about Aduhelm before the F.D.A. acted. “We are on a horrible journey,” she said of her husband and herself. Perhaps with the new drug, “we could have maybe not a normal life, but a better life.”
During their phone call, she listened as Dr. Koncilja noted that the science was exciting but that Aduhelm was no miracle drug. She heard for the first time about brain swelling or bleeding, “and that scared me,” she said. “Would I ever want to put Jim through that?” She was staggered by the price, which she cannot pay.
Her excitement has abated. “But Dr. Koncilja didn’t take away all my hope,” she said. “He told me, ‘Let’s see the potential through the summer, and we’ll confer again in the fall.’”
https://www.nytimes.com/2021/07/07/heal ... e=Homepage
The approval of Aduhelm to treat Alzheimer’s disease has raised hope among older adults, but many doctors wonder if it is warranted.
By Paula Span
July 7, 2021
Dr. Kenneth Koncilja, a geriatrician at the Cleveland Clinic, saw the announcement from the Food and Drug Administration on June 7, on Twitter: The agency had approved Aduhelm (aducanumab), the first drug to treat Alzheimer’s disease to be approved in nearly 20 years.
The calls from patients’ spouses and family members began within the hour, and have not stopped. “I was shocked at how fast the word spread — ‘Hey, is this something we can use? When can we get it?’” Dr. Koncilja recalled. “There’s a mix of excitement, anxiety and desperation.”
His first call that morning came from Joan Morehouse, 78, who has been caring for her 71-year-old husband, James, in their home in North Perry, Ohio, since his Alzheimer’s diagnosis four years ago. She has watched him get lost on familiar drives and forget their grandchildren’s names.
When her brother and her son both emailed her a news article about the F.D.A. action, she recalled, “I said, ‘Oh, my God, my prayers have been answered.’”
It fell to Dr. Koncilja to explain the complexities: That Aduhelm is not yet widely available. That protocols determining which patients qualify have yet to be developed. That the clinical trial data was ambiguous and that the drug might bring no noticeable improvements in daily life. That its side effects include brain swelling and bleeding.
And that its maker, Biogen, estimates the annual cost of monthly intravenous infusions at $56,000, plus expensive scans and tests.
“It’s a more difficult question than I’ve ever had before,” Dr. Koncilja said. Patients ask him how their lives will change, “and I don’t know how to answer.”
In the weeks since the F.D.A.’s action, which places virtually no restrictions on prescribing the drug, geriatricians, neurologists and other doctors across the country have been fielding similar questions.
Aduhelm has generated intense controversy. Biogen stopped two trials in 2019 because they demonstrated no benefit, then submitted an F.D.A. application after a later analysis of one trial showed slightly slower cognitive decline at high doses.
In a letter to the F.D.A., the American Geriatrics Society argued that approval was “premature given the lack of sufficient evidence.” The Society for Post-Acute and Long-Term Care Medicine later came to a similar conclusion.
The F.D.A.’s own advisory committee strongly recommended against approval, and three member scientists resigned in protest when the agency overrode its advice. A new survey of 200 neurologists and primary care doctors has found that most disagreed with the F.D.A. decision.
Senators Elizabeth Warren, Democrat of Massachusetts, and Bill Cassidy, Republican of Louisiana, have called for a hearing, concerned that spending billions on Aduhelm could undermine Medicare. The House Committee on Oversight and Reform has announced an investigation into the drug’s approval and pricing.
Given all that, should older adults consider Aduhelm? “The F.D.A. has passed the determination along to the American family,” said Dr. Jason Karlawish, co-director of the Penn Memory Center, who, with a number of other doctors, publicly opposed the drug’s approval.
Penn Memory doctors are receiving anxious inquiries, too. Geeta Simons, a musician in Philadelphia whose 80-year-old father has Alzheimer’s, messaged her father’s neurologist there. “I wanted to believe that this was that magical save,” she said.
Such doctors face “a dilemma,” Dr. Karlawish said, “a moment when there’s no decision that resolves all the uncertainties and settles the ethical concerns.”
“It puts us in a bad place,” agreed Dr. Karina Bishop, a geriatrician at the University of Nebraska Medical Center. Ethically, she added, “if this drug was available right now, I would not feel able to prescribe it.”
Even as individual doctors grapple with advising patients, hospitals and health systems are devising protocols for when Aduhelm becomes more widely available, probably within weeks.
At the Mayo Clinic, said Dr. Ronald Petersen, a neurologist who directs the Alzheimer’s Disease Research Center there, “we’re going to stick pretty close to the inclusion and exclusion criteria used in the trial.”
That means only patients with mild cognitive impairment or early Alzheimer’s disease would qualify, after an M.R.I. to rule out certain conditions and risks, and a P.E.T. scan or lumbar puncture to confirm the presence of amyloid. The Mayo protocols, like the clinical trials, would exclude people taking blood thinners like Warfarin or Eliquis.
“It’s not like you come in and say, ‘I’m a little forgetful,’ and we say, ‘Here’s this drug,’” said Dr. Petersen. But not every provider, he acknowledged, will employ such safeguards.
Dr. Eric Widera, a geriatrician at the University of California, San Francisco, expressed a similar concern: “If doctors were extremely cautious and limited this drug to the very specific population included in the study, with very careful monitoring, it would be the first time in medicine that was ever done.”
He pointed out another consequence of federal approval: a rift between some doctors and the Alzheimer’s Association, the national advocacy group, which this spring mounted a campaign it called More Time. Intended to demonstrate public support for approval of aducanumab, the effort included newspaper ads and social media posts.
Now Dr. Widera, who has worked with a local chapter to train medical students and residents, is seeking an alternate source of information to which to refer patients. He has come to mistrust the Alzheimer’s Association, calling it “a big promoter, almost a marketer, for Biogen,” which, like other pharmaceutical firms, helps underwrite the organization and contributed $275,000 to it last year.
The association said in an email that “history has shown us that approvals of the first drug in a new category will invigorate the field, increase investments in new treatments and generate greater innovation.”
One major unpredictable factor in Aduhelm’s future: insurance coverage. Medicare could decide to authorize coverage as “reasonable and necessary,” to deny or limit it, or to delay a decision. A spokesman at the Centers for Medicare and Medicaid Services said it was reviewing the F.D.A.’s decision and would have more information soon.
Given the drug’s announced price tag, a restrictive Medicare policy could put it beyond reach for most older Americans.
Eventually, the F.D.A. might also take action against Aduhelm. Its “accelerated approval” process requires Biogen to undertake a new clinical trial; if that shows no benefit, the agency could withdraw approval. But Biogen has until 2030 to report those results; by then, thousands of hopeful patients might already be taking Aduhelm.
For now, doctors are wrestling with how to respond.
“One of my core principles is respect for patient autonomy, especially for this disease, which degrades a patient’s ability to exercise self-determination,” said Dr. Karlawish. Slightly softening his published opposition to Aduhelm, he said that he now would prescribe it, after extensive discussions with patients, “but I’d be a reluctant prescriber.”
Several doctors described gently dissuading patients by noting the uncertainty that the drug would help, the potentially disabling side effects and the many unknowns. “They have been open to waiting and getting more information,” Dr. Bishop said.
Ms. Morehouse, for instance, had heard nothing about Aduhelm before the F.D.A. acted. “We are on a horrible journey,” she said of her husband and herself. Perhaps with the new drug, “we could have maybe not a normal life, but a better life.”
During their phone call, she listened as Dr. Koncilja noted that the science was exciting but that Aduhelm was no miracle drug. She heard for the first time about brain swelling or bleeding, “and that scared me,” she said. “Would I ever want to put Jim through that?” She was staggered by the price, which she cannot pay.
Her excitement has abated. “But Dr. Koncilja didn’t take away all my hope,” she said. “He told me, ‘Let’s see the potential through the summer, and we’ll confer again in the fall.’”
https://www.nytimes.com/2021/07/07/heal ... e=Homepage
Re: New Drug Could Cost the Government as Much as It Spends on NASA
F.D.A. Seeks Investigation of Its Own Alzheimer’s Drug Approval
The agency’s acting head said a review should look into whether any interactions between agency staff and the drug developer, Biogen, broke F.D.A. rules.
By Rebecca Robbins
July 9, 2021
The Food and Drug Administration on Friday called for a federal investigation of the process that led to the approval of a new drug for Alzheimer’s disease that has spurred sharp criticism from lawmakers and the medical community.
In a letter to the Department of Health and Human Services’ independent Office of the Inspector General, the F.D.A.’s acting commissioner, Dr. Janet Woodcock, acknowledged the scrutiny the agency has faced about the approval process for the drug, which is known as Aduhelm and has a $56,000 annual price tag. She pointed to interactions between representatives from the drug’s developer, Biogen, and the agency, saying some “may have occurred outside of the formal correspondence process.”
“To the extent these concerns could undermine the public’s confidence in F.D.A.’s decision, I believe it is critical that the events at issue be reviewed by an independent body,” Dr. Woodcock wrote. She noted that the review should look at whether any of the communication between the agency’s staff and Biogen’s representatives violated F.D.A. rules.
Dana Conti, a spokesman for Biogen, said the company “will, of course, cooperate with any inquiry in connection with a possible review of the regulatory process.”
It is unusual for the agency to request an investigation into its own staff’s decision-making process for an individual drug approval. The move is likely to intensify the controversy that has surrounded Aduhelm. The F.D.A. approved it a month ago, overriding the fierce objections of its own independent advisers and many other scientists, who said there was insufficient evidence to know whether the drug was effective.
On Thursday, the F.D.A. moved to narrow its recommendation about who should receive the drug. After originally recommending it for all Alzheimer’s patients, the agency’s new guidelines say it should be prescribed only to people with mild cognitive problems.
Dr. Aaron Kesselheim, one of three experts who quit an F.D.A. advisory panel last month in protest of the agency’s decision on Aduhelm, said he welcomed the call for a federal investigation.
“It’s really important that an investigation happen publicly and quickly so that we find answers to some of the many outstanding questions swirling around this decision,” said Dr. Kesselheim, a professor of medicine at Harvard Medical School and Brigham and Women’s Hospital.
A number of Alzheimer’s and public health experts have raised concerns about what they characterized as an unusually close collaborative relationship between Biogen and the F.D.A. while the drug was being reviewed. One example was the agency’s move to jointly present with Biogen its glowing review of the evidence supporting the drug in a meeting of the F.D.A.’s independent advisers in November 2019.
Stat, the medical news organization, first reported that in early May 2019, Dr. Billy Dunn, the head of the agency’s neuroscience division, held an off-the-book meeting with a Biogen executive, Dr. Al Sandrock. While it is not unusual for drug company executives to meet frequently with F.D.A. officials, it is unusual to present data that would be part of an F.D.A. application outside a formal setting.
A few months earlier, Biogen had moved to halt two late-stage studies of Aduhelm after an early analysis found that it would not prove to be effective. But Biogen researchers analyzing the data soon concluded that the decision to halt the studies had been premature and that they had reason to believe it might be effective after all.
The May 2019 meeting between Dr. Dunn and Dr. Sandrock was an initial step in restarting the talks that led to last month’s approval. It led to the first of a series of standard formal meetings between Biogen and agency staff. It also led to a more unusual collaboration: That summer, representatives from the company and the agency worked closely together to sort through Biogen’s complicated data from the studies, communicating nearly every day, Stat reported.
Aduhelm was the first drug approved to treat Alzheimer’s in 18 years — and it was the very first approval for a drug designed to attack the biological underpinnings of the disease, instead of just delaying symptoms.
https://www.nytimes.com/2021/07/09/heal ... ation.html
The agency’s acting head said a review should look into whether any interactions between agency staff and the drug developer, Biogen, broke F.D.A. rules.
By Rebecca Robbins
July 9, 2021
The Food and Drug Administration on Friday called for a federal investigation of the process that led to the approval of a new drug for Alzheimer’s disease that has spurred sharp criticism from lawmakers and the medical community.
In a letter to the Department of Health and Human Services’ independent Office of the Inspector General, the F.D.A.’s acting commissioner, Dr. Janet Woodcock, acknowledged the scrutiny the agency has faced about the approval process for the drug, which is known as Aduhelm and has a $56,000 annual price tag. She pointed to interactions between representatives from the drug’s developer, Biogen, and the agency, saying some “may have occurred outside of the formal correspondence process.”
“To the extent these concerns could undermine the public’s confidence in F.D.A.’s decision, I believe it is critical that the events at issue be reviewed by an independent body,” Dr. Woodcock wrote. She noted that the review should look at whether any of the communication between the agency’s staff and Biogen’s representatives violated F.D.A. rules.
Dana Conti, a spokesman for Biogen, said the company “will, of course, cooperate with any inquiry in connection with a possible review of the regulatory process.”
It is unusual for the agency to request an investigation into its own staff’s decision-making process for an individual drug approval. The move is likely to intensify the controversy that has surrounded Aduhelm. The F.D.A. approved it a month ago, overriding the fierce objections of its own independent advisers and many other scientists, who said there was insufficient evidence to know whether the drug was effective.
On Thursday, the F.D.A. moved to narrow its recommendation about who should receive the drug. After originally recommending it for all Alzheimer’s patients, the agency’s new guidelines say it should be prescribed only to people with mild cognitive problems.
Dr. Aaron Kesselheim, one of three experts who quit an F.D.A. advisory panel last month in protest of the agency’s decision on Aduhelm, said he welcomed the call for a federal investigation.
“It’s really important that an investigation happen publicly and quickly so that we find answers to some of the many outstanding questions swirling around this decision,” said Dr. Kesselheim, a professor of medicine at Harvard Medical School and Brigham and Women’s Hospital.
A number of Alzheimer’s and public health experts have raised concerns about what they characterized as an unusually close collaborative relationship between Biogen and the F.D.A. while the drug was being reviewed. One example was the agency’s move to jointly present with Biogen its glowing review of the evidence supporting the drug in a meeting of the F.D.A.’s independent advisers in November 2019.
Stat, the medical news organization, first reported that in early May 2019, Dr. Billy Dunn, the head of the agency’s neuroscience division, held an off-the-book meeting with a Biogen executive, Dr. Al Sandrock. While it is not unusual for drug company executives to meet frequently with F.D.A. officials, it is unusual to present data that would be part of an F.D.A. application outside a formal setting.
A few months earlier, Biogen had moved to halt two late-stage studies of Aduhelm after an early analysis found that it would not prove to be effective. But Biogen researchers analyzing the data soon concluded that the decision to halt the studies had been premature and that they had reason to believe it might be effective after all.
The May 2019 meeting between Dr. Dunn and Dr. Sandrock was an initial step in restarting the talks that led to last month’s approval. It led to the first of a series of standard formal meetings between Biogen and agency staff. It also led to a more unusual collaboration: That summer, representatives from the company and the agency worked closely together to sort through Biogen’s complicated data from the studies, communicating nearly every day, Stat reported.
Aduhelm was the first drug approved to treat Alzheimer’s in 18 years — and it was the very first approval for a drug designed to attack the biological underpinnings of the disease, instead of just delaying symptoms.
https://www.nytimes.com/2021/07/09/heal ... ation.html
Re: New Drug Could Cost the Government as Much as It Spends on NASA
Cleveland Clinic and Mount Sinai Won’t Administer Aduhelm to Patients
The rejection of the new Alzheimer’s drug by the two major medical centers is one of the starkest signs of concern over its approval by the F.D.A.
By Pam Belluck
July 14, 2021
In a striking reflection of concern over the approval of the controversial new Alzheimer’s drug Aduhelm, two major American health systems have decided that they will not administer it to patients.
The Cleveland Clinic, one of the largest and most respected medical centers in the country, said in a statement that a panel of its experts had “reviewed all available scientific evidence on this medication,” which is also called aducanumab.
“Based on the current data regarding its safety and efficacy, we have decided not to carry aducanumab at this time,” the statement said.
A spokeswoman for the clinic said that individual physicians there could prescribe Aduhelm to patients, but those patients would have to go elsewhere to receive the drug, which is administered as a monthly intravenous infusion.
Mount Sinai’s Health System in New York City has also decided not to administer Aduhelm, said Dr. Sam Gandy, director of the Mount Sinai Center for Cognitive Health.
Dr. Gandy, who is also a professor of psychiatry and neurology, said the decision was driven by the fact that there have been calls for a federal investigation to look into the F.D.A. decision and the agency’s relationship with Biogen, the drug’s manufacturer. He said, “Aduhelm will not be considered for infusion into patients on any of its campuses until and unless” an investigation by the inspector general of the Department of Health and Human Services, “affirms the integrity of the F.D.A.-Biogen relationship and goes on to reaffirm” the F.D.A.’s basis for approving the drug.
The rejection by the major medical centers is the latest fallout from the Food and Drug Administration’s approval of the drug on June 7, a decision that has also spurred congressional investigations.
Many Alzheimer’s experts and other scientists have said that it is unclear that the drug works to help slow cognitive decline and that in the best-case scenario, the evidence suggested only a slight slowing while also showing that Aduhelm could cause brain swelling or brain bleeding.
Dr. Gandy said in his private practice he tells patients that “no one was improved by Aduhelm and patients’ cognition always continued to decline at some rate.”
The drug is also expensive. Biogen, the maker, has set its price at $56,000 a year.
In a recent survey of nearly 200 neurologists and primary care doctors, most said they disagreed with the F.D.A. decision and did not plan to prescribe the drug to their patients.
Last week, in response to growing criticism, Dr. Janet Woodcock, the acting F.D.A. commissioner, called for an independent federal investigation into the agency’s approval process, writing that “to the extent these concerns could undermine the public’s confidence in F.D.A.’s decision, I believe it is critical that the events at issue be reviewed by an independent body.”
Two nearly identical clinical trials of Aduhelm were stopped early because an independent data monitoring committee concluded that the drug didn’t appear to be helping patients. A later analysis by Biogen found that participants receiving the high dose of the drug in one trial had experienced a very slight slowing of cognitive decline — 0.39 on an 18-point scale — but that participants in the other trial had not benefited at all.
About 40 percent of trial participants developed brain bleeding or brain swelling, and while most of those cases were mild or manageable, about 6 percent of participants dropped out of the trials because of serious adverse effects from those conditions. Dr. Gandy said that one patient in his private practice had to drop out because the patient suffered 10 brain microhemorrhages.
After evaluating the data late last year, an F.D.A. advisory committee of outside experts strongly recommended against approval, and three of its members resigned in protest last month when the agency bucked the advisory committee’s advice. The American Geriatrics Society had also urged the agency not to approve the drug, saying it would be “premature given the lack of sufficient evidence.”
Last week, in response to widespread criticism that it had approved Aduhelm for anyone with Alzheimer’s, the F.D.A. sharply narrowed the drug’s recommended use, saying it should be used only for people with mild memory or thinking problems because there was no data on Aduhelm’s use in later stages of Alzheimer’s.
Pam Belluck is a health and science writer whose honors include sharing a Pulitzer Prize and winning the Nellie Bly Award for Best Front Page Story. She is the author of Island Practice, a book about an unusual doctor.
https://www.nytimes.com/2021/07/14/heal ... uhelm.html
The rejection of the new Alzheimer’s drug by the two major medical centers is one of the starkest signs of concern over its approval by the F.D.A.
By Pam Belluck
July 14, 2021
In a striking reflection of concern over the approval of the controversial new Alzheimer’s drug Aduhelm, two major American health systems have decided that they will not administer it to patients.
The Cleveland Clinic, one of the largest and most respected medical centers in the country, said in a statement that a panel of its experts had “reviewed all available scientific evidence on this medication,” which is also called aducanumab.
“Based on the current data regarding its safety and efficacy, we have decided not to carry aducanumab at this time,” the statement said.
A spokeswoman for the clinic said that individual physicians there could prescribe Aduhelm to patients, but those patients would have to go elsewhere to receive the drug, which is administered as a monthly intravenous infusion.
Mount Sinai’s Health System in New York City has also decided not to administer Aduhelm, said Dr. Sam Gandy, director of the Mount Sinai Center for Cognitive Health.
Dr. Gandy, who is also a professor of psychiatry and neurology, said the decision was driven by the fact that there have been calls for a federal investigation to look into the F.D.A. decision and the agency’s relationship with Biogen, the drug’s manufacturer. He said, “Aduhelm will not be considered for infusion into patients on any of its campuses until and unless” an investigation by the inspector general of the Department of Health and Human Services, “affirms the integrity of the F.D.A.-Biogen relationship and goes on to reaffirm” the F.D.A.’s basis for approving the drug.
The rejection by the major medical centers is the latest fallout from the Food and Drug Administration’s approval of the drug on June 7, a decision that has also spurred congressional investigations.
Many Alzheimer’s experts and other scientists have said that it is unclear that the drug works to help slow cognitive decline and that in the best-case scenario, the evidence suggested only a slight slowing while also showing that Aduhelm could cause brain swelling or brain bleeding.
Dr. Gandy said in his private practice he tells patients that “no one was improved by Aduhelm and patients’ cognition always continued to decline at some rate.”
The drug is also expensive. Biogen, the maker, has set its price at $56,000 a year.
In a recent survey of nearly 200 neurologists and primary care doctors, most said they disagreed with the F.D.A. decision and did not plan to prescribe the drug to their patients.
Last week, in response to growing criticism, Dr. Janet Woodcock, the acting F.D.A. commissioner, called for an independent federal investigation into the agency’s approval process, writing that “to the extent these concerns could undermine the public’s confidence in F.D.A.’s decision, I believe it is critical that the events at issue be reviewed by an independent body.”
Two nearly identical clinical trials of Aduhelm were stopped early because an independent data monitoring committee concluded that the drug didn’t appear to be helping patients. A later analysis by Biogen found that participants receiving the high dose of the drug in one trial had experienced a very slight slowing of cognitive decline — 0.39 on an 18-point scale — but that participants in the other trial had not benefited at all.
About 40 percent of trial participants developed brain bleeding or brain swelling, and while most of those cases were mild or manageable, about 6 percent of participants dropped out of the trials because of serious adverse effects from those conditions. Dr. Gandy said that one patient in his private practice had to drop out because the patient suffered 10 brain microhemorrhages.
After evaluating the data late last year, an F.D.A. advisory committee of outside experts strongly recommended against approval, and three of its members resigned in protest last month when the agency bucked the advisory committee’s advice. The American Geriatrics Society had also urged the agency not to approve the drug, saying it would be “premature given the lack of sufficient evidence.”
Last week, in response to widespread criticism that it had approved Aduhelm for anyone with Alzheimer’s, the F.D.A. sharply narrowed the drug’s recommended use, saying it should be used only for people with mild memory or thinking problems because there was no data on Aduhelm’s use in later stages of Alzheimer’s.
Pam Belluck is a health and science writer whose honors include sharing a Pulitzer Prize and winning the Nellie Bly Award for Best Front Page Story. She is the author of Island Practice, a book about an unusual doctor.
https://www.nytimes.com/2021/07/14/heal ... uhelm.html
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