Tag: Alzheimers

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Biogen’s controversial Alzheimer’s drug generates $2 million gross sales in first few weeks after approval

Biogen's controversial Alzheimer’s drug generates $2 million sales in first few weeks after approval

Aduhelm from Biogen

Source: Biogen

Biogen’s Alzheimer’s drug Aduhelm made $ 2 million in sales in the first few weeks after its approval, the company said Thursday when it released its second quarter results along with an open letter about the controversial drug.

Biogen increased its sales guidance for the year and expected total sales for the year of $ 10.65 billion to $ 10.85 billion. That’s an increase from its earlier estimates of $ 10.45 billion to $ 10.75 billion. The new forecast assumes “modest” income for Aduhelm in 2021, which will then be ramped up, the company said.

Here’s how Biogen performed in the three months ended June 30, compared to Wall Street’s expectations, according to Refinitiv’s average estimates:

  • Adjusted earnings per share: $ 5.68 versus $ 4.54 expected
  • Revenue: $ 2.78 billion versus an expected $ 2.61 billion

The company’s stock rose slightly in early trading.

Aduhelm was approved by the Food and Drug Administration on June 7th. The drug, scientifically known as aducanumab, offers new hope to friends and families of patients living with the disease and is set to generate billions in revenue for the company.

However, its approval has since been questioned, and the head of the FDA is now calling for a state investigation into the interactions between agency employees and the biotech company.

Biogen’s Chief Research Officer, Dr. Al Sandrock, defended the drug in an open letter released Thursday along with the company’s profits, saying its approval was subject to “extensive misinformation and misunderstanding”.

He said it was “normal” for scientists and clinicians to discuss and debate data from experimental and clinical trials, but added that those discussions had taken a turn “outside the boundaries of legitimate scientific reasoning”.

“We welcome a formal review of the interactions between the FDA and Biogen in the process of obtaining aducanumab approval,” said Sandrock. “A better understanding of the facts is good for everyone involved in building confidence in the therapy and the approval process as we prioritize the issues that affect patients.”

Correction: In an earlier version, Aduhelm was misspelled.

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How an Unproven Alzheimer’s Drug Acquired Authorized

How an Unproven Alzheimer’s Drug Got Approved

The idea of accelerated approval came up briefly toward the end, raised by Dr. Rick Pazdur, head of F.D.A.’s oncology center, who was not a council member. It was not discussed in detail, but after the meeting, given the council’s rejection of standard approval, accelerated approval appeared to be the only way to make the drug available.

On April 26, Dr. Patrizia Cavazzoni, Dr. Dunn’s boss and director of the Center for Drug Evaluation and Research, led a smaller meeting about accelerated approval, which had never been used for Alzheimer’s drugs.

In fact, the F.D.A.’s most recent guidance for Alzheimer’s drugs, issued by Dr. Dunn in 2018, says “the standard for accelerated approval” had not yet been met for the disease, “despite a great deal of research.” The guidance says that is because “there is unfortunately at present no sufficiently reliable evidence” that attacking amyloid plaques or other biomarkers of Alzheimer’s “would be reasonably likely to predict clinical benefit.”

And at the November advisory committee meeting, Dr. Dunn said that in considering whether to approve aducanumab, “we’re not using the amyloid as a surrogate for efficacy.”

Under accelerated approval, while a drug is on the market, a company must conduct an additional trial, a costly undertaking. Biogen said its goal was standard approval, which it believed its data warranted.

At the April 26 meeting, Dr. Cavazzoni invited two officials not involved with neurological drugs who had used accelerated approval frequently: Dr. Pazdur and Dr. Peter Marks, the top vaccine regulator. They and Dr. Cavazzoni voted to grant such approval to aducanumab, as did Dr. Issam Zineh, director of the Office of Pharmacology, and Dr. Jacqueline Corrigan-Curay, who led the internal review of the F.D.A-Biogen collaboration.

The director of the office of translational sciences, Dr. ShaAvhrée Buckman-Garner — who supervises both the pharmacology and biostatistics offices — did not vote yes or no, saying she understood both arguments. The only clear no vote, F.D.A. documents say, was the director of the office of biostatistics, Dr. Sylva Collins, “stating her belief that there is insufficient evidence to support accelerated approval or any other type of approval.”

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Ohio Clinic Says It Will not Administer Alzheimer’s Drug to Sufferers

Ohio Clinic Says It Won't Administer Alzheimer's Drug to Patients

In a conspicuous concern about the approval of the controversial new Alzheimer’s drug Aduhelm, the Cleveland Clinic said Wednesday evening that it would not give it to patients.

The clinic, one of the largest and most respected medical centers in the country, said in a statement a panel of experts had “reviewed all available scientific evidence about this drug,” also called aducanumab.

“Based on the current data on safety and effectiveness, we have decided not to wear aducanumab at the moment,” the statement said.

A spokeswoman for the clinic said individual doctors there could prescribe Aduhelm to patients, but those patients would have to go elsewhere to get the drug, which is given as an intravenous infusion every month.

The stance of the major medical center is the latest fallout from the approval of the drug by the Food and Drug Administration on June 7, a decision that also fueled Congressional investigations.

Many Alzheimer’s experts and other scientists have said that it is unclear that the drug helps slow cognitive decline and that at best the evidence suggests only a slight slowdown while showing that Aduhelm causes brain swelling or hemorrhage could.

Recognition…Biogen, via Associated Press

The drug is also expensive. Biogen, the maker, has set its price at $ 56,000 per year.

In a recent survey of nearly 200 neurologists and primary care physicians, most said they disagreed with the FDA’s decision and did not plan to prescribe the drug to their patients.

Last week, Dr. Janet Woodcock, acting FDA commissioner, in response to growing criticism of an independent state investigation into the agency’s regulatory process, wrote, “To the extent that these concerns could undermine public confidence in the FDA’s decision, I believe” it is critically important that the disputed events are reviewed by an independent body. “

Two almost identical clinical trials with Aduhelm were stopped prematurely because an independent data monitoring committee concluded that the drug did not appear to be helping patients. A later analysis by Biogen found that participants who received the high dose of the drug in one study experienced a very slight slowdown in cognitive decline – 0.39 on an 18-point scale – that participants in the other study however, had not benefited from it at all.

About 40 percent of study participants developed cerebral hemorrhage or swelling, and while most of these cases were mild or manageable, about 6 percent of participants dropped out because of serious side effects from these conditions.

After reviewing the data late last year, an FDA advisory committee strongly recommended outside experts against approval, and three of its members resigned in protest last month when the agency defied the advice of the advisory committee. The American Geriatrics Society had also urged the agency not to approve the drug because it was “premature in the absence of sufficient evidence.”

In response to widespread criticism that Aduhelm was approved for anyone with Alzheimer’s, the FDA last week severely restricted the drug’s recommended use, saying that it should only be used for people with mild memory or thinking problems as it doesn’t have any Data on the use of Aduhelm gave later stages of Alzheimer’s disease.

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F.D.A. Requests Federal Investigation of Alzheimer’s Drug Approval

F.D.A. Requests Federal Investigation of Alzheimer’s Drug Approval

The Food and Drug Administration on Friday called for a federal investigation into the process that led to the approval of a new drug for Alzheimer’s disease that has sparked harsh criticism from lawmakers and the medical community.

In a letter to the independent office of the Inspector General of the Department of Health, Acting Commissioner of the FDA, Dr. Janet Woodcock, the scrutiny the agency has been through regarding the approval process for the drug known as Aduhelm. She pointed to interactions between representatives from drug developer Biogen and the agency, saying that some “may have occurred outside of the formal correspondence process”.

“To the extent that these concerns could undermine public confidence in the FDA’s decision, I believe it is critical that the disputed events be verified by an independent body,” wrote Dr. Woodcock. She noted that the review should investigate whether communications between agency staff and Biogen representatives violated FDA rules.

The unusual request to examine the decision-making process of one’s own employees for an individual drug approval is likely to exacerbate the controversy surrounding the approval of Aduhelm. The FDA approved the drug a month ago, overcoming stout objections from its own independent advisors who said there wasn’t enough evidence to know if the drug was effective.

After the decision, three of these experts left an FDA advisory panel.

Dr. Woodcock’s request for an investigation came a day after the FDA narrowed its recommendations on who should receive the drug. After initially being recommended for all Alzheimer’s patients, the agency’s new guidelines should only prescribe it to people with mild cognitive problems.

Biogen did not immediately respond to a request for comment.

STAT, the medical news organization, first reported that Dr. Billy Dunn, the agency’s head of neuroscience, in early May 2019 held an off-the-book meeting with a Biogen manager, Dr. Al Sandrock, exit. While it is not uncommon for pharmaceutical company executives to meet frequently with FDA officials, it is uncommon to provide data that would be part of an FDA application outside of a formal framework.

A few months earlier, Biogen had discontinued two late-stage studies of the drug after early analysis found the drug would not prove effective. However, the Biogen researchers who analyzed the data soon concluded that the decision to stop the studies was premature and that the drug might be effective after all.

The meeting between Dr. Dunn and Dr. Sandrock was a first step in resuming the talks that led to approval last month.

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A New Alzheimer’s Drug Gives Extra Questions Than Solutions

A New Alzheimer’s Drug Offers More Questions Than Answers

“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 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.

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In a Reversal, F.D.A. Requires Limits on Who Will get Alzheimer’s Drug

In a Reversal, F.D.A. Calls for Limits on Who Gets Alzheimer’s Drug

When the FDA approved Aduhelm a month ago, the original label said the drug was “for the treatment of Alzheimer’s disease.” The surprisingly broad label caused a storm of concern among many Alzheimer’s experts, even those who had supported the drug’s approval.

At a forum sponsored by the Alzheimer’s Association last month that urged Aduhelm to be approved, a panel of Alzheimer’s clinics with differing views agreed on whether the drug should have been approved that its use was strong should be restricted.

Many experts say the drug’s label should not only restrict Aduhelm’s use to mild disease stages, but also require two more stringent clinical trial conditions: that suitable patients have evidence of high levels of a key protein, amyloid, in their brain, and that people with certain diseases (so-called “contraindications”) should be prevented from taking the drug or at least classified as a high-risk group, as this can lead to brain swelling and bleeding.

At the forum, Dr. Stephen Selloway, director of the Memory and Aging Program at Butler Hospital in Providence, RI, who helped conduct the Aduhelm studies and is one of the drug’s most ardent medical advocates, when he saw the label had no contraindications to his The reaction was “oy”.

On Thursday, Dr. Lon Schneider, director of the California Alzheimer’s Disease Center at the University of Southern California, who also worked on one of the Aduhelm studies and spoke out against the approval of the drug, saying that the new labeling was neglected.

The label should have said, among other things, that people with diabetes, high blood pressure and people who take blood thinners are not allowed to take part in the clinical trials and therefore “no extent is known”. of increased risk ”for these patients.

Dr. Jason Karlawish, a co-director of the University of Pennsylvania’s Penn Memory Center, said the new label was based more on what he and other Alzheimer’s experts believe should originally have been written. But the abrupt revision of the label so soon after a much criticized approval decision was worrying, he said.

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New Alzheimer’s Drug Might Value the Authorities as A lot as It Spends on NASA

New Alzheimer’s Drug Could Cost the Government as Much as It Spends on NASA

Es wird erwartet, dass Medicare ein neu zugelassenes Medikament zur Behandlung der Alzheimer-Krankheit in Höhe von mehreren Milliarden Dollar kosten wird. Einer Prognose zufolge könnten die Ausgaben für das Medikament für die Patienten von Medicare am Ende höher sein als die Budgets der Umweltschutzbehörde oder der NASA.

Es gibt wenig Beweise dafür, dass das Medikament Aduhelm das Fortschreiten der Demenz verlangsamt, aber die Food and Drug Administration hat es diesen Monat genehmigt. Analysten gehen davon aus, dass Medicare und seine Teilnehmer, die einen Teil ihrer Kosten für verschreibungspflichtige Medikamente zahlen, in einem einzigen Jahr 5,8 bis 29 Milliarden US-Dollar für das Medikament ausgeben werden.

„Es ist unergründlich“, sagte Tricia Neuman, geschäftsführende Direktorin des Programms zur Medicare-Politik der Kaiser Family Foundation. “Das sind verrückte Zahlen.”

Viele andere Medikamente kosten mehr als Aduhelm, das von Biogen hergestellt wird und jährlich 56.000 US-Dollar kosten wird. Der Unterschied besteht darin, dass es Millionen potenzieller Kunden gibt und das Medikament voraussichtlich über Jahre hinweg eingenommen wird.

Die Zulassung des Medikaments stößt bei Gesundheitspolitikern und Pharmaforschern auf Kritik wegen fehlender nachgewiesener Wirksamkeit. Wirksam oder nicht, wenn es allgemein verschrieben wird, könnte es einen überwältigenden Einfluss auf das Budget von Medicare haben, da das öffentliche Programm die überwiegende Mehrheit der fast sechs Millionen Amerikaner mit einer Alzheimer-Diagnose abdeckt.

Es gibt kaum einen Präzedenzfall für einen plötzlichen Ausgabenruck dieser Größenordnung. Selbst am unteren Ende der Prognosen würde Aduhelm zu einem der teuersten Medikamente von Medicare werden.

Am oberen Ende sagen Analysten, dass das neue Medikament die jährlichen Ausgaben von Medicare für Medikamente, die in Krankenhäusern und Arztpraxen geliefert werden, um 50 Prozent erhöhen könnte (wie es Aduhelm, das intravenös verabreicht wird, sein müsste).

Die Vergleiche hier sind ungefähre Angaben: Ein Drittel der Medicare-Mitglieder ist durch private Medicare Advantage-Pläne abgesichert, die keine detaillierten Informationen zu den in Arztpraxen angebotenen Medikamenten enthalten. Um diese Ausgaben zu schätzen, haben wir die Daten zu den Medikamentenausgaben der Medicare-Teilnehmer des traditionellen öffentlichen Programms verwendet und sie erhöht, um den fehlenden Anteil zu berücksichtigen.

Ausgaben in dieser Größenordnung könnten so plötzlich weitreichende Auswirkungen auf Medicare, seine Nutzer und Steuerzahler haben. Die Hinzufügung von 29 Milliarden US-Dollar ein Jahr des Medicare-Haushalts würde durch Erhöhungen sowohl der Ausgaben der Steuerzahler als auch der von allen Medicare-Nutzern gezahlten Prämien gedeckt. Die Prämien könnten auch für Zusatzpläne steigen, die viele Medicare-Leistungsempfänger kaufen, um Kosten auszugleichen, die das Programm nicht direkt bezahlt. Und die Kosten werden wahrscheinlich auf die Staatshaushalte übergreifen, wo Medicaid Prämien für einkommensschwache Medicare-Mitglieder zahlt.

Kongress, Haushaltsexperten und mehrere Weiße Häuser haben Jahre damit verbracht, Wege zur Reduzierung der Ausgaben für Medicare, einen großen und wachsenden Anteil des Bundeshaushalts, vorzuschlagen. Aber viele dieser Vorschläge sind politisch schwer zu erreichen – und die meisten würden weniger als die prognostizierten Kosten von Aduhelm einsparen.

“Es ist so viel Arbeit, Einsparungen zu erzielen, die wirklich viel kleiner sind, als dieses eine Medikament kosten würde”, sagte Joshua Gordon, der Direktor für Gesundheitspolitik beim Ausschuss für einen verantwortungsvollen Bundeshaushalt, der sagt, dass er sich ständig Gedanken über die Herausforderungen gemacht hat von Aduhelm seit seiner Zulassung erhoben.

Die Kostenprognosen variieren, da Analysten nicht sicher sind, wie viele Patienten das neue Medikament letztendlich verwenden werden. Die Zulassung der FDA könnte für jeden gelten, bei dem Alzheimer diagnostiziert wurde – etwa sechs Millionen Menschen. Das Medikament wurde jedoch für eine kleinere Gruppe von rund 1,5 Millionen Patienten entwickelt, die sich im Frühstadium der Krankheit befinden. Analysten sind sich noch nicht sicher, wem Ärzte die Behandlung empfehlen und welche Familien sie ausprobieren möchten. Die FDA hat Biogen gebeten, das Medikament bis 2030 weiter zu untersuchen, aber die Verschreibung könnte weit verbreitet werden, bevor weitere öffentliche Ergebnisse darüber vorliegen, wie gut es wirkt.

Allison Parks, eine Sprecherin von Biogen, sagte in einer E-Mail, dass sich das Unternehmen darauf konzentrieren werde, die Art von Patienten zu erreichen, die in den klinischen Studien des Unternehmens untersucht wurden, „im frühen symptomatischen Stadium der Krankheit“.

Aktualisiert

21. Juni 2021, 20:11 Uhr ET

Die Bandbreite spiegelt eine Vielzahl von angemessenen Expertenschätzungen wider. Die hohe Schätzung, die sich auf ein Kaiser-Papier stützt, geht davon aus, dass etwa ein Viertel der zwei Millionen Medicare-Eingeschriebenen, die derzeit eine Alzheimer-Behandlung erhalten, diese einnehmen werden. Der niedrige Wert basiert auf einer Schätzung der Analysten von Cowen and Company von einem Gesamtumsatz von 7 Milliarden US-Dollar bis 2023.

Es ist schwierig abzuschätzen, wie viele Patienten das Medikament einnehmen werden. Aduhelm ist nicht nur teuer, sondern auch etwas schwer einzunehmen und erfordert monatliche persönliche Besuche in einem Infusionszentrum zur Behandlung. Patienten, die es einnehmen, müssen während ihrer Behandlungen mehrere Gehirnscans durchführen, um nach Nebenwirkungen zu suchen.

Und die Nebenwirkungen selbst – etwa 40 Prozent der Patienten in einer klinischen Studie zeigten Anzeichen einer Hirnschwellung – können einige Patienten davon abhalten, das Medikament auszuprobieren, und andere dazu veranlassen, die Einnahme abzubrechen. (Die vielen Scans – und Behandlungen für schwerwiegendere Nebenwirkungen – würden auch von Medicare abgedeckt.)

Es gibt sechs Millionen Medicare-Angehörige, die keine Zusatzversicherung abschließen, die möglicherweise 20 Prozent der Arzneimittelkosten bezahlen müssen, in diesem Fall 11.200 USD pro Jahr.

Dennoch kann die Nachfrage von Familien groß sein, die angesichts einer verheerenden Diagnose eine Möglichkeit sehen, einzugreifen. Bisher gab es nur wenige Behandlungsmöglichkeiten für Patienten, die hoffen, den kognitiven Rückgang durch die Krankheit zu verhindern.

„Es ist schon an sich schwer, einen geliebten Menschen zu haben, die Uhr ticken zu sehen und zu sagen: Nun, lass uns einfach warten“, sagte Dr. Steven Pearson, Hausarzt und Präsident des Institute for Clinical and Economic Review (ICER). ). „Es ist sehr schwer, den Drang zu ignorieren, etwas zu tun.“

Bidens Haushalt 202222

    • Ein neues Jahr, ein neues Budget: Das Geschäftsjahr 2022 für die Bundesregierung beginnt am 1. Oktober, und Präsident Biden hat bekannt gegeben, was er ab diesem Zeitpunkt ausgeben möchte. Aber jede Ausgabe erfordert die Zustimmung beider Kammern des Kongresses.
    • Ambitionierte Gesamtausgaben: Präsident Biden möchte, dass die Bundesregierung im Fiskaljahr 2022 6 Billionen US-Dollar ausgibt und die Gesamtausgaben bis 2031 auf 8,2 Billionen US-Dollar steigen. Dies würde die Vereinigten Staaten auf den höchsten anhaltenden Stand der Bundesausgaben seit dem Zweiten Weltkrieg bringen, während sie laufen Defizite von über 1,3 Billionen US-Dollar in den nächsten zehn Jahren.
    • Infrastrukturplan: Das Budget skizziert das gewünschte erste Jahr der Investition des Präsidenten in seinen American Jobs Plan, der darauf abzielt, Verbesserungen von Straßen, Brücken, öffentlichen Verkehrsmitteln und mehr mit insgesamt 2,3 Milliarden US-Dollar über acht Jahre zu finanzieren.
    • Familienplan: Das Budget befasst sich auch mit dem anderen wichtigen Ausgabenvorschlag, den Biden bereits eingeführt hat, seinem American Families Plan, der darauf abzielt, das soziale Sicherheitsnetz der Vereinigten Staaten zu stärken, indem der Zugang zu Bildung erweitert, die Kosten für Kinderbetreuung gesenkt und Frauen in der Arbeitswelt unterstützt werden.
    • Pflichtprogramme: Wie üblich machen obligatorische Ausgaben für Programme wie Social Security, Medicaid und Medicare einen erheblichen Teil des vorgeschlagenen Budgets aus. Sie wachsen, während die Bevölkerung Amerikas altert.
    • Ermessensausgaben: Die Mittel für die einzelnen Budgets der Agenturen und Programme der Exekutive würden im Jahr 2022 rund 1,5 Billionen US-Dollar erreichen, eine Steigerung um 16 Prozent gegenüber dem vorherigen Budget.
    • Wie Biden dafür bezahlen würde: Der Präsident würde seine Agenda weitgehend durch Steuererhöhungen für Unternehmen und Gutverdiener finanzieren, was in den 2030er Jahren beginnen würde, die Haushaltsdefizite zu verringern. Verwaltungsbeamte sagten, Steuererhöhungen würden die Beschäftigungs- und Familienpläne im Laufe von 15 Jahren vollständig ausgleichen, was der Haushaltsantrag unterstützt. In der Zwischenzeit würde das Haushaltsdefizit jedes Jahr über 1,3 Billionen US-Dollar bleiben.

Ärzte, die dieses Medikament verabreichen und für diese Arbeit einen Prozentsatz des hohen Preises des Medikaments von Medicare erhalten, könnten finanzielle Anreize haben, Ja zu sagen, wenn Patienten danach fragen.

“Die Auswirkungen dieses einen Medikaments und der damit verbundenen Verfahren sind enorm”, sagte Rachel Sachs, Rechtsprofessorin an der Washington University in St. Louis und Autorin eines kürzlich in The Atlantic erschienenen Essays, in dem behauptet wird, dass das Medikament “die amerikanische Gesundheit verletzen” könnte Pflege.”

Private Versicherer können Hindernisse für die Behandlung errichten, die von Patienten zusätzliche Tests verlangen oder nachweisen, dass andere Optionen nicht funktioniert haben. Unter normalen Umständen deckt Medicare jedoch Medikamente ab, die von der FDA zugelassen sind. Medicare entscheidet, welche Medikamente abgedeckt werden, basierend darauf, ob sie „angemessen und notwendig“ sind, nicht auf deren Kosten.

Medicare ist verpflichtet, diese Art von Arzneimitteln zunächst zum Listenpreis zuzüglich einer Gebühr von 3 Prozent an den behandelnden Arzt zu zahlen. Und dann, nach etwa einem Jahr auf dem Markt, zahlt es den durchschnittlichen Verkaufspreis plus 6 Prozent. Bei Arzneimitteln mit Konkurrenz kann dieser Durchschnittspreis erheblich unter dem Aufkleberpreis liegen. Aber für ein Medikament wie Aduhelm, das das erste seiner Art ist, darf der Arzneimittelhersteller Ärzten keine Rabatte anbieten.

Medicare, das 61 Millionen Amerikaner ab 65 Jahren abdeckt, hat einige Instrumente, um die Kosten einzudämmen. Es könnte beschließen, das Medikament in einer Weise abzudecken, die eingeschränkter als die FDA-Zulassung ist, eine Abweichung von seiner normalen Praxis.

Oder es könnte etwas noch Ungewöhnlicheres tun: Eine unerwartete Allianz von Befürwortern hat vorgeschlagen, dass Medicare das Medikament einem randomisierten Experiment unterzieht, um zu bewerten, wie gut es wirkt – in einigen Teilen des Landes bezahlen sie für die Abdeckung des Medikaments, in anderen jedoch nicht. Solche politischen Experimente wurden im Rahmen des Affordable Care Act genehmigt, aber noch nie wurde eines verwendet, um die Abdeckung eines Medikaments auf diese Weise einzuschränken.

Andere Länder werden höchstwahrscheinlich die Kosten von Aduhelm kontrollieren, indem sie mit Biogen über einen niedrigeren Preis verhandeln oder einfach den Kauf ablehnen. Die meisten werden die Wirksamkeit des Medikaments berücksichtigen, wenn sie entscheiden, was sie zu zahlen bereit sind. Bisher ist das Medikament nirgendwo sonst auf der Welt zugelassen.

Medicare kann das nicht. Aufgrund der Art und Weise, wie sie nach geltendem Recht für Medikamente bezahlt, hat sie keine Möglichkeit, den Preis herunterzuhandeln. Demokraten unterstützen zunehmend Gesetze, die dies ändern. Das Repräsentantenhaus verabschiedete 2019 ein Gesetz, das Medicare die Befugnis geben würde, einige Preise auszuhandeln, aber es starb im Senat. Im April brachten die Gesetzgeber den gleichen Gesetzentwurf wieder ins Repräsentantenhaus ein.

Präsident Biden unterstützt es, Medicare die Aushandlung von Medikamentenpreisen zu ermöglichen, hat die Richtlinie jedoch nicht in seinen vorgeschlagenen amerikanischen Familienplan aufgenommen.

Dr. Pearson von ICER schätzt, dass, wenn die Wirksamkeit des neuen Medikaments berücksichtigt würde, ein fairer Preis 2.500 bis 8.300 US-Dollar betragen würde.

“Es wird interessant sein zu sehen, ob dies eine Diskussion über faire Preise in den Vereinigten Staaten auslöst”, sagte er. “In den Augen der meisten Leute sieht dies wie ein hervorragendes Beispiel für einen Preis aus, der einfach nicht mit den Beweisen übereinstimmt.”

Methodik: Die geschätzten aktuellen Ausgaben für Medicare-Teil-B-Medikamente wurden vom Centers for Medicare- und Medicaid-Services-Teil-B-Drogenausgaben-Dashboard entnommen und um 54 Prozent angehoben, um Medicare-Leistungsempfänger zu berücksichtigen, die in Medicare Advantage-Plänen eingeschrieben sind. Aufgrund der Demografie, wer an welchem ​​Programm teilnimmt, kann diese Annahme die aktuellen Drogenausgaben überschätzen.)

Die Medikamentenausgaben von Medicare Teil D wurden direkt aus dem CMS Teil D-Drogenausgaben-Dashboard entnommen und stellen möglicherweise eine Überschätzung dar, da diese Zahlen nicht alle an Medikamentenpläne gezahlten Rabatte enthalten.

Die hohe Schätzung der Ausgaben von Aduhelm stammt aus einem Papier der Kaiser Family Foundation. Die niedrige Schätzung wird aus einer Gesamtumsatzschätzung von Cowen and Company abgeleitet und angepasst, um schätzungsweise 80 Prozent der Alzheimer-Patienten zu Beginn ihrer Krankheit zu berücksichtigen, die sich in Medicare eingeschrieben haben – und Medicares anfängliche Zahlung von 3 Prozent an Ärzte für Gemeinkosten und Verwaltung.

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Many Alzheimer’s Consultants Say Use of Aduhelm Ought to Be Sharply Restricted

Many Alzheimer’s Experts Say Use of Aduhelm Should Be Sharply Limited

Dr. Selloway, a research director on the site for studies of the drug, was not paid for this work but has received research and consulting fees from Biogen. He said doctors should only use the drug on patients whose status matches those in clinical trials.

“There is no evidence that it could be beneficial for any other stage of Alzheimer’s disease,” he said.

Mary Sano, director of the Alzheimer’s Research Center at Mount Sinai in New York City, said the criteria she and other panelists outlined were “very important”, saying that “it will be very restrictive and the ability to use this drug with.” Sharing a wide range of people with others will be significantly limited, at least at this point in time. “

For dementia clinicians, treating people with only mild symptoms would mean that “most of your staff is unlikely to be an option in your current practice,” said Dr. Sano.

In its decision, the FDA admitted that there was not the proof of benefit usually required by the authorities. As a result, it is making it available to Aduhelm under a program called accelerated approval, which spearheads the drug’s ability to lower amyloid levels in the brain. But reducing amyloid is not the same as slowing down symptoms of dementia. Many amyloid-lowering drugs failed to slow the decline in clinical trials, a story that makes some experts particularly suspicious of trusting Aduhelm based on the evidence presented so far.

Also, given the agency’s focus on amyloid in its approval decision and the fact that all participants in the clinical trial were required to have high levels of amyloid, experts were surprised that the FDA label does not mandate patient screening for the protein. Doctors at the Alzheimer’s Association forum all said that high levels of amyloid, typically measured by PET scans or spinal puncture, should be a prerequisite for treatment.

Several of the panelists said that relatively few doctors and clinics, at least initially, would be able to adequately diagnose, screen, and treat patients.

“This is not a simple drug,” said Dr. Paul Aisen, director of the Alzheimer’s Therapeutic Research Institute at the University of Southern California and co-author of an article calling on the FDA to approve the drug. “I think identifying the right people to treat and supervise treatment requires knowledge and experience, and there are very few clinicians who have that experience.”

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Biogen’s Alzheimer’s drug may value Medicare billions of {dollars} a yr: report

Biogen's Alzheimer's drug could cost Medicare billions of dollars a year: report

A pedestrian walks past Biogen Inc. headquarters in Cambridge, Massachusetts on Monday, June 7, 2021.

Adam Glanzman | Bloomberg | Getty Images

Biogen’s expensive new Alzheimer’s drug Aduhelm could cost Medicare billions of dollars, according to an analysis published Thursday by the nonprofit Kaiser Family Foundation.

The Food and Drug Administration on Monday approved the company’s drug, the first U.S. regulator-approved drug to slow cognitive decline in people with Alzheimer’s and the first new drug for the disease in nearly two decades.

The biotech company said it charges $ 56,000 for an annual course of the new treatment, which is higher than the $ 10,000-25,000 price some Wall Street analysts were expecting. This is the wholesale price, and the cost that patients actually pay depends on their health insurance plan.

It is estimated that Alzheimer’s disease affects more than 6 million Americans, the vast majority of whom are 65 years of age and older. Biogen estimates that about 80% of Alzheimer’s patients are covered by Medicare, the state health insurance for the elderly.

It is still unclear how many Medicare beneficiaries will take Biogen’s drug, but even a conservative estimate would result in a “substantial increase” in Medicare spending, according to KFF.

In 2017, nearly 2 million Medicare beneficiaries were using one or more Alzheimer’s treatments that are covered under Medicare Part D, according to KFF. The group said if a quarter of those beneficiaries were instead prescribed Aduhelm, and Medicare paid 103% of $ 56,000 in the near future, “the total spending on Aduhelm in a year alone will be nearly $ 29 billion”.

According to the KFF, Aduhelm is covered by Medicare Part B, which generally covers FDA-approved, physician-administered drugs.

“If 1 million Medicare beneficiaries received Aduhelm, which may be on the lower end of Biogen’s expectations, spending for Aduhelm alone would exceed $ 57 billion in a single year – well above anything else covered by Part B. Medication together, ”group said. The total spend for Part B in 2019 was $ 37 billion.

Biogen has been criticized by Wall Street analysts and advocacy groups for questioning how the company could justify the price, especially as medical experts continue to debate whether there is enough evidence that the drug actually works and criticize the industry for drug prices becomes.

On a call to investors Tuesday morning, Evercore ISI analyst Umer Raffat congratulated the Massachusetts-based company on US approval of the drug before asking executives to explain the price.

“I think there is a discrepancy between some of the words you shared in your press releases like responsibility, access, health equity, and price, especially given the basic care population,” he told executives.

Biogen executives said Tuesday the overall price of the new treatment was “underpinned” by the value it is expected to bring to patients, caregivers and society. They insisted that the price was “responsible” and stated that the disease costs the US billions each year.

The company has pledged not to increase the price of the new drug over the next four years. However, executives said they were “open-minded” and suggested reconsidering price as the company assesses demand over the next few years.

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Biogen Alzheimer’s drug and the brand new battle over dementia remedy

Biogen Alzheimer’s drug and the new battle over dementia treatment

Aduhelm from Biogen

Source: Biogen

The FDA approval of Biogen’s Alzheimer’s drug Aduhelm marked a milestone in Dr. Paul Aisen. The director of the Alzheimer’s Therapeutic Research Institute at USC has focused on the treatment of neurodegenerative disease for the past three decades and in recent years has helped guide this particular drug through the various phases of clinical trials.

But as he sat in his sun-drenched San Diego office in early June, he felt slightly puzzled by the way the Food and Drug Administration approved their use in early June on an “accelerated” basis, normally reserved for cancer drugs. This meant that the clinical benefit was considered likely, but approval for long-term use would be the subject of a larger study in a fourth phase of studies.

Aisen, who works as a paid advisor to Biogen, emphasizes the “unusual nature” of the regulator’s green light, as an advisory board of experts voted and publicly opposed the approval, and insists that there were still “many questions the “I have – they still have no answers.”

Three members of the FDA’s panel that oversees the research have resigned since it was approved this week, including Dr. Aaron Kesselheim, a medicine professor at Harvard Medical School, who said in a letter the agency’s decision on Biogen was “probably the worst drug approval decision in history.” recent US history. “

Last November, that panel said in an 8-1 vote that Biogen’s late-stage phase did not provide “strong evidence” showing that aducanumab is effective in treating Alzheimer’s; two other panellists said the data was “uncertain”.

While Aisen sees Aduhelm as an “effective treatment” for a disease that affects millions of Americans, he also has concerns about the impact of the FDA’s decision on the range of other potential treatment options that are in the late stages of development.

An immediate challenge facing other teams working on a broader Alzheimer’s drug pipeline, he said in a recent video call, would be to keep participants in ongoing studies, let alone attract new ones.

“In most cases,” he said, many people with Alzheimer’s disease would drop out of other drug trials to begin treatment with the newly approved Aduhelm. Leaving them would make the study data for these alternative drugs less useful, even if the drugs in question might one day prove to be safer, more effective, or more suitable for different stages of the disease. But perhaps pervertedly, he still regards Aduhelm’s approval as “a boost to these efforts – a powerful boost”.

Over 6 million Americans suffer from Alzheimer’s

In the past few years, some large pharmaceutical companies have abandoned brain disease research efforts, including Pfizer and Boehringer Ingelheim in 2018 – in fact, Biogen Aduhelm had given up at one point during clinical trials in 2019 before reversing its decision – after decades of failure in search of a breakthrough.

The controversy surrounding the Biogen drug, including its potential cost, is hitting a massive, unmet need for dementia treatment and a disease that costs the US up to $ 259 billion annually. The Alzheimer’s Association estimates that more than 6 million Americans have Alzheimer’s or some other form of dementia, and by 2050 that number could reach over 12 million people, which costs a trillion dollars a year.

Because of this, some dementia drug experts are focusing on the new attention and funding, rather than the potential downsides of Biogen approval, said Dr. Jeffrey Cummings, a neurologist at the University of Nevada, Las Vegas, who does an annual review of. publishes Alzheimer’s drug development pipeline. His research consistently showed that drug failure rates prior to Biogen’s approval were 99.6 percent, a stark contrast to 1 in 5 successful cancer drugs (20%).

Cummings says that any short-term adverse side effect for other drug trials “will be overcome, if at all, by increased interest from companies, venture capital, and biotechnology once they see that there is a way to get approval for a particular drug”. Illness.”

In recent history, the National Institutes of Health spent two to three times more research on heart disease and cancer than they did on dementia, and the lack of qualified participants in clinical trials also slowed progress.

Next in the dementia drug pipeline

For the handful of other Alzheimer’s drugs in development hoping to overcome the same regulatory hurdles and prove their effectiveness – including Eli Lillys Donanemab, Roches Gantenerumab, and Eiseis Lecanemab – there could be a silver lining, the first mover advantage to cede to Aduhelm.

After decades of expensive but largely unsuccessful research attempts, pharmaceutical giant Eli Lilly’s CEO David Ricks said that after a series of positive phase two results for its Donanemab offering, his company is “getting closer and closer to the goal”.

Speaking at CNBC’s Healthy Returns Summit in May, a month before the FDA approved Aduhelm’s rival Biogen, he said his team felt “good about the probability of success” and said he wanted an “accelerated” route too explore what he called “adaptative avenues for the FDA to consider earlier study of data” that “should be used in a serious and widespread disease like Alzheimer’s”.

However, he conceded that recruiting for the next phase of the studies would require a significantly larger cohort of participants, and since it would take 18 months, he did not expect a new approved product until late 2023 at the earliest.

Several experts told CNBC that the Biogen drug’s unique threshold for regulatory approval, with the treatment potential appearing to trump uncertain real-world benefits, efforts of competitors like Lilly, who are focused on drug development on relatively based on similar techniques.

Aduhelm’s own clinical study data had shown that the drug successfully attacked and cleared clusters of a certain type of protein that many researchers believe may be responsible for Alzheimer’s disease. But it didn’t offer enough evidence to prove that the drug provided cognitive benefits to patients.

Debate on targeting amyloid beta formations

Known by scientists as aducanumab, it works by offering a set of identical antibodies that are cloned from white blood cells. These antibodies are chosen for their targeting abilities, as they can identify specific proteins called beta-amyloids that have built up certain formations in the body.

There is ample evidence that these beta-amyloid formations, also known as “pathological aggregates” or “plaques,” are a major cause of Alzheimer’s disease, although the exact causal mechanisms are not yet fully understood, according to Christian Pike von USC’s Leonard Davis School of Gerontology. Nonetheless, he says the antibodies can help prevent these plaques from forming before other particles are caused to break them apart, a process that is clearly identifiable in before and after neural imaging.

As an analogy, it may be helpful to think of the amyloid beta proteins as young people walking through a city during the day, where the city is the human body and the day is a human lifespan. In certain cities, when afternoon turns into evening, individual young people gather, and some of these gatherings can become toxic and begin to cause problems. The antibodies supplied by Aduhelm act like police officers arriving at the scene, identifying disruptive gatherings, surrounding them, separating them, and then instructing bystanders to disperse the young people.

“When you say, ‘Well, hey, the FDA is buying in that general concept,'” Pike said on a phone call, “if we can remove beta-amyloid from the brains of people with the disease, even if we can there is limited evidence of cognitive benefits, “he continued,” there could be a variety of different therapies that would qualify under these criteria.

The long string of past failures within the Alzheimer’s pipeline that targeted beta-amyloid will continue to weigh on optimism until conclusive evidence is produced – something this week’s controversy over the first new approved Alzheimer’s drug in decades shows has not yet been done.

“What we’re going to find out by using this drug one way or another is whether or not the amyloid clearing hypothesis is correct,” says USC health economist Darius Lakdawalla, who argues that Biogens will continue to test it Drug will prove useful to this confirmatory experiment.

“If it’s right, then I think it opens the door to a lot of innovation, a lot of drug candidates that will try to remove amyloid in the pursuit of that hypothesis in the future.”