Biogen, the manufacturer of the controversial drug for Alzheimer’s disease (AD) aducanumab (Aduhelm) needs to cut its $56,000 annual cost and speed the pace of research to determine whether the medication actually keeps the disease at bay.

These are among the key recommendations of a report released today by the Institute for Clinical and Economic Review (ICER). The institute also suggested that Medicare could limit initial coverage of aducanumab to patients enrolled in a randomized clinical trial.

ICER also warned against promotions for aducanumab that would increase public expectations for the medication that exceed what the existing body of evidence shows.  

In June, the US Food and Drug Administration (FDA) granted aducanumab an accelerated approval based on a surrogate endpoint of reduction of amyloid beta plaque.

The agency then set a 2030 deadline for Biogen to deliver proof of a clinical benefit for the medication. Physicians and insurers in the United States are likely to wrestle for years with questions about the use of a drug with its hefty annual cost and unresolved questions about efficacy.  

“Messaging from the manufacturer and patient groups, such as in patient-oriented websites and advertisements, should make it clear both that aducanumab has not been shown to improve cognitive and functional outcomes — rather it may slow decline — and also that removal of amyloid has not been conclusively demonstrated to affect clinical outcomes,” ICER states in its report.

“For this last reason, all stakeholders should avoid using the term ‘amyloid-busting’ in reference to aducanumab since that term would easily be interpreted by patients and families as confirmation that removal of amyloid has demonstrated clinical benefits,” ICER added.

There has been considerable uproar about how the FDA cleared aducanumab for US sales. As reported by Medscape Medical News, on Wednesday the Office of the Inspector General of the Department of Health and Human Services announced a review of FDA procedures triggered by this approval.

Aducanumab is intended for those with mild cognitive impairment or mild dementia stage of disease. Biogen has pressed for patients in the group to begin using the drug while additional scientific data are gathered to try to prove its benefit. Biogen argues that many patients could miss their chance to receive aducanumab if their disease progresses because of delayed access to the treatment.

Patient, Caregiver Toll

In a statement emailed to Medscape Medical News, Biogen said the ICER report did not appropriately consider the burden AD has on society and the strain it places on caregivers.

The report itself also includes comments offered by Maha Radhakrishnan, MD, Biogen’s chief medical officer, who said ICER discounted the “possible holistic value” of the drug.

Patients with AD and their caregivers may be terrified after the condition is diagnosed. They suffer from depression, anxiety, and agitation as they cope with this new reality, Radhakrishnan said.

The ICER report notes the toll AD takes on caregivers. More than 11 million family members and others provide an estimated 15.3 billion hours of unpaid assistance to patients with AD or other dementias, putting them “at risk for negative mental, physical, and emotional outcomes,” ICER adds.

There’s clearly a demand for a drug that would at least hold at bay the effects of AD. However, the significant number of failures of previous drug candidates intended to target amyloid has led some experts to express doubt about whether this is the main or only causal pathway, says ICER.

ICER said its report reflects 8 months of work spent analyzing aducanumab study results and talking with Biogen officials, patient groups, and clinical experts.

“At the conclusion of this effort, despite the tremendous unmet need for new treatments for Alzheimer’s disease, we have judged the current evidence to be insufficient to demonstrate that aducanumab slows cognitive decline, while it is clear that it can harm some patients,” said David Rind, MD, ICER’s chief medical officer, in a statement.

ICER’s research has clout because it helps insurers decide whether and/or how to cover medications. For example, in a June letter to the Centers for Medicare and Medicaid Services (CMS), America’s Health Insurance Plans cited ICER’s work. In the letter, the insurance trade group asked for a national coverage determination on Medicare policy on aducanumab. CMS has since announced that it intends to create such a policy and is accepting comments on this topic through its website.

Other key recommendations from the report include:

• Medical societies and clinicians should advocate for “fair pricing” of aducanumab and for equitable access to AD care in general.

• Medicare should explore how to implement a “rigorous” policy regarding coverage of aducanumab.

• There should be shared decision-making founded upon a robust discussion of the potential harms and benefits of treatment.

The full set of ICER’s recommendations are available here.

Kerry Dooley Young is a freelance journalist based in Washington, DC. She is the core topic leader on patient safety issues for the Association of Health Care Journalists. Young earlier covered health policy and the federal budget for Congressional Quarterly/CQ Roll Call, and the pharmaceutical industry and FDA for Bloomberg. Follow her on Twitter at @kdooleyyoung

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