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Pink Sheet – US Medicare Agency Clarifies View Of Accelerated Approval

Pink Sheet – US Medicare Agency Clarifies View Of Accelerated Approval

The US Centers for Medicare & Medicaid Services is reassuring oncology patient advocates that it is not changing its historic approach to coverage of medicines approved via the Accelerated Approval pathway.

CMS is facing bipartisan criticism for its restrictive coverage policy for beta amyloid targeted therapies to treat Alzheimer’s, which deems drugs approved via the Food & Drug Administration’s AA process to be eligible for coverage only in the context of clinical trials – a policy that essentially means there is no meaningful access for Medicare beneficiaries to Eisai Co., Ltd./Biogen, Inc.’s Leqembi (lecanemab), or the more controversial predecessor product Aduhelm (aducanumab). (Also see “Medicare And Alzheimer’s Drugs: Pressure To Relax Coverage Restrictions Rising Wtih Donanemab Data” – Pink Sheet, 7 May, 2023.)

In defending the policy during a House hearing at the end of April, CMS Administrator Chiquita Brooks-LaSure suggested that the agency does not accept FDA’s off-stated position that drugs granted Accelerated Approval are still “fully approved.” (Also see “Leqembi Coverage Expansion: US Medicare Head Promises Light Touch On Registry” – Pink Sheet, 1 May, 2023.)

That prompted a letter from Friends of Cancer Research seeking clarification on whether the agency has changed its view on the status of Accelerated Approval drugs. “We respectfully ask that CMS clarify its position on accelerated approvals, particularly how it applies to reimbursement of cancer drugs approved by FDA and/or included in clinical guidelines developed by subject matter experts,” the 2 May letter said.

“In your letter, you asked whether CMS has changed its approach to drugs and biologics approved by the FDA under the Accelerated Approval Program,” Brooks-LaSure responded May 9. “We have not,” she declared (with the emphasis included).

However, she went on to suggest an approach to Accelerated Approval that seems to imply that some uses of the pathway will indeed face more scrutiny than others.

“For many accelerated approval drugs for diseases like cancer or human immunodeficiency virus (HIV), there is an established scientific consensus that validated surrogate outcomes are indicators of health outcomes,” she wrote. “For example, with respect to cancer, there is scientific evidence as well as wide acceptance by clinicians of the association between shrinking tumors (surrogate outcome) and symptoms or overall patient survival in patients receiving such cancer drugs (health outcome).”

As a practical matter, FDA now views the surrogate endpoints for HIV as sufficient for “full” traditional approval; Accelerated Approval is not generally an option in that class any longer. (Also see “Accelerated Approval In The US: It Really Is Rare Outside Of Oncology” – Pink Sheet, 8 Sep, 2022.)

In oncology, “surrogates” like Progression Free Survival or response rates have also been used as the basis for “full” approval in some contexts.

Thus, while Brooks-LaSure’s message may be comforting in the field of oncology, it leaves open the prospect that future uses of novel surrogates like beta amyloid could trigger similar scrutiny from the payor agency.