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Pink Sheet — Medicare’s ‘Aduhelm’ Policy A Precedent For Accelerated Approvals But Not Cancer Drugs – Gottlieb

Pink Sheet — Medicare’s ‘Aduhelm’ Policy A Precedent For Accelerated Approvals But Not Cancer Drugs – Gottlieb

The Centers for Medicare and Medicaid Services has established a precedent regarding Medicare Part B coverage for accelerated approvals with its decision triggered by Biogen, Inc.’s Aduhelm to only cover that class of Alzheimer’s treatments when patients are enrolled in a clinical trial, according to former US Food and Drug Administration Commissioner Scott Gottlieb.

But the agency is likely to steer clear of applying the policy in the future to oncology drugs because that would upend Medicare’s current reliance on medical compendia to determine its coverage of uses that are not traditionally approved, he added during a meeting sponsored by the Partnership to Fight Chronic Disease 1 August.

Gottlieb was also a senior advisor at CMS during the George W. Bush Administration and is now a resident fellow at the American Enterprise Institute and partner at the venture capital firm New Enterprise Associates.

Aduhelm was granted a controversial accelerated approval by FDA in June 2021 and CMS released a draft national policy the following January conditioning coverage for Aduhelm and upcoming drugs in the same class on further evidence development, a policy known as CED that had very rarely been applied to drugs but has often been used for medical devices.

The final version of the policy limited coverage of drugs with accelerated approvals to use in clinical trials while drugs with traditional approvals would be covered when patients are enrolled in a registry. (Also see “Medicare Alzheimer’s Decision Varies Evidence Mandate For Accelerated vs. Traditional Approvals” – Pink Sheet, 7 Apr, 2022.)

The decision has raised alarms among stakeholders that CMS will take a similar approach for Medicare coverage for other accelerated approvals.

CMS has tried to tamp down concerns by maintaining the policy addressed a unique situation.  (Also see “Medicare Restrictions On Aduhelm Do Not Signal Broad Concern With Accelerated Approvals – CMS” – Pink Sheet, 13 Apr, 2022.) But Gottlieb agreed there is cause for concern.

“I don’t think that this is a one off. I think they have established a doctrine and they’re going to use it for Part B drugs” in the future, Gottlieb said. “This was a long-sought policy,” he continued. “I was there when the CED [policy] was put into place,” and “this was a very big discussion.”

There were “instances where CMS staff were kind of flirting with the idea of trying to make a coverage decision on the basis that the drug was approved under an accelerated approval. We pushed back on that” but “that whole debate has come up time and time again.”

However, CMS “may be reluctant to use it in the oncology setting and that’s why the first setting they used it in was in Alzheimer’s,” Gottlieb suggested. “I think [oncology] would be a heavier political lift.” Medicare will cover off-label uses of cancer drugs if the indication is supported by National Comprehensive Cancer Network (NCCN) medical compendium.

“Even though [CMS] will say publicly and privately they are not bound by the compendia, everyone thinks they are bound by the … NCCN compendium, so they would face a lot of push back from Capitol Hill if they tried to do something like this in the setting of oncology,” Gottlieb continued.

A recent letter from CMS Administrator Chiquita Brooks-LaSure to Friends of Cancer Research would seem to support that perspective.  (Also see “US Medicare Agency Clarifies View Of Accelerated Approval” – Pink Sheet, 16 May, 2023.)

CMS ‘Wrong On The Science’ With CED Policy

Gottlieb also described the CMS decision for Aduhelm as in line with previous important CED policies for medical devices or procedures where the agency was “fundamentally wrong on the science.”

The Aduhelm decision “felt like a policy in search of a drug target” and “like CMS wanted to implement a policy where they could demonstrate that they could apply sort of a medical device reimbursement paradigm to a Part B drug and say, ‘We’re not going to pay for it. We’re going to make our own independent judgement and come up with criteria by which we’re going to decide we’re not going to pay for something,’” Gottlieb said.

But CMS “probably chose the wrong drug, because their initial basis for it was just that they fundamentally didn’t believe that removing amyloid plaque was going to deliver a therapeutic benefit. They didn’t believe the clinical trial data that FDA had signed off on and approved the drug on the basis of, and they were just completely wrong on the science.”

Gottlieb also pointed to past decisions that restricted Medicare coverage for implantable defibrillators and transaortic valve replacement surgery as other examples. “Every time CMS has tried to establish a clinical criteria that I can think of in a big coverage decision, where they try to use a clinical criteria to determine who should and should not be eligible for the [treatment] that was their own criteria … they’ve been wrong,” he maintained.–Gottlieb