Moving Beyond Aduhelm: Cell/Gene Therapy May Be ‘Poster Child’ For What Comes Next

Former FDA principal deputy commissioner Amy Abernethy says cell and gene therapy is an area primed for improved ‘parallel conversation’ between FDA and CMS on data requirements.

Is gene therapy where FDA and CMS will be able to connect? • Source: Nielsen Hobbs; the Pink Sheet | Shutterstock image

In the wake of the US Centers for Medicare & Medicaid Services’ limited coverage decision for Biogen, Inc./Eisai Co., Ltd.’s Aduhelm, sponsors have been seeking assurances that CMS’ policy not to cover the Alzheimer’s therapy outside of clinical trials – in essence nullifying the US Food & Drug Administration’s Accelerated Approval action in 2021 – was a rare, one-off decision.

That is certainly the message that CMS has been sending ever since that controversial decision was made, most recently at the Duke-Margolis health policy conference, where both CMS and FDA officials strove to present the Aduhelm debate as an outlier – and a learning opportunity for both agencies on the benefits of a “parallel conversation” on data requirements

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