Tag Archives: Biogen

Former FDA adviser calls for wider probe into Biogen Alzheimer’s drug approval

By Julie Steenhuysen and Deena Beasley

(Reuters) – A federal probe of the U.S. Food and Drug Administration’s approval of a controversial new Alzheimer’s disease drug should look into why that decision was made without clear evidence of patient benefit, a former adviser to the agency said on Tuesday.

Acting FDA Commissioner Janet Woodcock earlier this month asked the Office of the Inspector General at the Department of Health and Human Services to investigate whether FDA representatives’ interactions with drugmaker Biogen Inc were inconsistent with agency policies.

“I happen to think that there’s a lot more to investigate than just that,” said Dr. Aaron Kesselheim of Harvard-affiliated Brigham and Women’s Hospital, who resigned as a member of the FDA advisory panel that reviewed and voted against approval of Biogen’s drug prior to the FDA green light.

Speaking at a live event sponsored by The Forum at the Harvard T.H. Chan School of Public Health, he said more information is needed on FDA’s role in Biogen’s evaluation of clinical trial data, as well as why the agency decided to base its approval on a “surrogate” biomarker rather than the drug’s impact on cognitive function.

The FDA in early June approved Biogen’s Aduhelm, citing evidence that it can reduce brain plaques, a likely contributor to Alzheimer’s disease, rather than proof that it slows progression of the lethal mind-wasting condition.

“If the FDA knows about some really clear scientific evidence … they should share that because I don’t think there is a lot of clarity around that in the field right now,” Kesselheim said.

He was one of three experts who resigned from the FDA advisory panel that recommended late last year that the agency turn down Biogen’s application to market Aduhelm, which the company has priced at about $56,000 a year.

“I think that by all objective measures it is an excessive cost,” Kesselheim said.

(Reporting by Julie Steenhuysen in Chicago; Writing by Deena Beasley in Los Angeles; Editing by Bill Berkrot)

Biogen Alzheimer’s drug hits roadblocks with some hospitals, insurers

By Deena Beasley

(Reuters) -The rollout of Biogen Inc’s Alzheimer’s drug is hitting new roadblocks as some large hospitals decide not to use it and many health insurers await coverage terms from Medicare, the U.S. health plan for people aged 65 and older, before setting their own policies.

Cleveland Clinic, one of the country’s best-known health systems, and New York’s Mount Sinai Health System on Thursday confirmed they had decided not to carry the new drug, called Aduhelm.

“The tide turned on Friday when the inspector general investigation was announced, and the potential allegation of irregularity in the FDA/Biogen relationship,” Dr. Sam Gandy, director of the Mount Sinai Center for Cognitive Health, told Reuters.

The FDA called last week for an independent federal probe into its representatives’ interactions with Biogen.

Biogen shares fell nearly 8% on Thursday, or $25.21, to $324.85. Guggenheim analyst Yatin Suneja attributed the stock slump to the decision by the two hospital systems not to use the drug.

In mid-June, the Washington, D.C., Neurology Center said it would not recommend the treatment, which is given as a monthly infusion, for any of its patients due to concerns about efficacy, safety and cost.

The U.S. Food and Drug Administration approved the drug, also known as aducanumab, in early June despite mixed clinical trial results. The agency said it was convinced that evidence of Aduhelm’s ability to clear amyloid brain plaques would benefit Alzheimer’s patients.

Biogen, which priced Aduhelm at $56,000 a year, said in a statement on Thursday that clinical data supported the drug’s approval and patients who are denied access should contact the company for help.

INSURERS ON HOLD

Insurers representing millions of American enrolled in private Medicare plans said the drug has yet to meet their bar for coverage based on the data.

UnitedHealth Group, the largest private insurer offering Medicare Advantage coverage to seniors, on Thursday said it was still reviewing the drug and awaiting input from Medicare.

“This has some way to go before we get to real clarity. So I wouldn’t guide you to expect a very rapid decision-making on this piece,” CEO Andrew Witty said.

Humana, the second largest provider of Medicare Advantage plans, also said it has not finalized coverage for Aduhelm as it awaits guidance from the Centers for Medicare and Medicaid Services (CMS).

Several Blue Cross Blue Shield health insurance plans, including those in Michigan, North Carolina and Pennsylvania, have said there is insufficient evidence of Aduhelm’s benefit for patients and they will not provide coverage for the drug.

Biogen said in a statement that the several Blues plans’ “characterization of Aduhelm as experimental and investigational is inaccurate and misleading.”

CMS on Monday began a national review process it said would take nine months to complete. Until then, the agency said coverage determinations for aducanumab are being made at the local level by 12 regional contractors.

SVB Leerink this week said a survey of 57 U.S. neurologists who treat high volumes of Alzheimer’s patients found that 44% of them would use Aduhelm in patients with early Alzheimer’s disease who have evidence of amyloid plaques.

The Wall Street firm estimates sales of the drug at $65 million this year, $1.1 billion next year and $5 billion by 2025.

The Institute for Clinical and Economic Review, an influential pricing group, was holding a meeting on Thursday of doctors, patients and other stakeholders to discuss how Aduhelm’s cost stacks up against potential benefits to patients.

(Reporting by Deena Beasley; Additional reporting by Manas Mishra in Bangalaru and Julie Steenhuysen in Chicago; Editing by Howard Goller)

U.S. experts to review Biogen drug that could be first new Alzheimer’s treatment in decades

By Deena Beasley

(Reuters) – U.S. health experts this week will decide whether to recommend approval for Biogen Inc’s Alzheimer’s drug, which could become the first new treatment for the mind-wasting disease in decades even as serious questions persist over whether data show if it works.

In a field littered with unrelenting failure, Biogen believes in aducanumab it has the first drug that can treat an underlying cause, and therefore slow progression, of Alzheimer’s. But its path to approval has been anything but smooth or assured.

Biogen abruptly ended clinical trials of aducanumab last year after an early look at trial results showed it was not effective. Last October, the company shocked many Alzheimer’s experts by reversing course, saying that a new analysis showed aducanumab could help patients with early-stage disease preserve their ability to function independently for longer. In July, Biogen filed for approval from the Food and Drug Administration.

Now the agency faces tremendous pressure to approve a treatment option for millions of Americans suffering from Alzheimer’s and the millions more expected to face it in coming years.

Patient advocates say the need for a new Alzheimer’s treatment that could help people remain independent is heightened by the coronavirus pandemic, which has killed more than 229,000 people in the United States, including tens of thousands of seniors in nursing homes.

“The pandemic came and it changed everything,” said Russ Paulsen, chief operating officer at patient advocacy group UsAgainstAlzheimer’s. “We need something to keep people out of nursing homes.”

A committee of outside advisers to the FDA will discuss aducanumab on Nov. 6. The agency’s final decision is expected by March. European health regulators have also accepted the drug for review.

Charles Flagg, a 79-year-old retired minister from Jamestown, Rhode Island, had been enrolled for years in a trial of aducanumab before it was stopped. He started receiving the drug again in August as part of a follow-up study, according to his wife Cynthia Flagg.

“He’s not 100 percent himself, but overall I’m not dealing with someone that needs to be led around or be in a care home,” Flagg said.

Aducanumab, an antibody designed to remove amyloid plaques from the brain – a strategy tried with many failed Alzheimer’s drugs – would reap billions of dollars in sales if approved.

Biogen, along with partner Eisai Co Ltd <4523.T>, is one of the last large drugmakers pursuing treatments for a disease that afflicts nearly 6 million Americans and millions more worldwide. Biogen estimates about 1.5 million people with early Alzheimer’s in the United States could be candidates for the drug.

‘CLINICALLY MEANINGFUL’

Late last year, Biogen said one of its two pivotal studies of aducanumab showed a statistically significant benefit at slowing cognitive and functional decline in patients with early Alzheimer’s. A second trial failed to achieve that goal, but did show a benefit for a subset of patients who were given a high dose for at least 10 months.

In March, it opened a follow-up long-term safety study to 2,400 people who had previously participated in trials of aducanumab, like Flagg.

Many Alzheimer’s researchers say Biogen should conduct a third large study to prove aducanumab works. They worry about its possible side-effects, such as brain-swelling, and the potential cost.

“Aducanumab’s efficacy as a treatment for the cognitive dysfunction in Alzheimer’s disease cannot be proven by clinical trials with divergent outcomes,” said Mayo Clinic neurologist Dr. David Knopman, who was recused from the expert panel because he helped conduct the trials.

Others believe the FDA could approve the drug without another trial.

Previous clinical studies had largely targeted patients in later stages of the disease, while many experts now believe attacking Alzheimer’s as early as possible may be the key to success. But there was a lack of clarity on how to assess a drug’s success when functional deficits are less pronounced.

In 2018, the FDA revised its standard of proof guidance for reviewing Alzheimer’s drugs by essentially combining what had been separate goals for cognition, or memory, and day-to-day function. The new guidance stressed the need for a drug to show “clinically meaningful” benefits, a term it has not clearly defined.

Alzheimer’s advocacy groups are pushing for a broad definition, saying it should include preserving the ability to perform daily activities such as shopping independently or remembering to turn off a stove.

“They have been trying to lower the bar and help any company to get a drug approved,” said Dr. Marwan Sabbagh, from the Cleveland Clinic Lou Ruvo Center for Brain Health in Las Vegas. “How much (improvement) do you need to be clinically meaningful? Naturally this is very subjective.”

The FDA did not immediately respond to a request for comment.

(Reporting by Deena Beasley; Editing by Caroline Humer and Bill Berkrot)