ICER To Look at Gene Therapy for Thalassemia

The Institute for Clinical and Economic Review (ICER) announced today that it will assess the comparative clinical effectiveness and value of betibeglogene autotemcel (Zynteglo/LentiGlobin, bluebird bio) for the treatment of beta thalassemia. An FDA decision on betibeglogene autotemcel is expected in mid-2022.  ICER is inviting public comment on its initial Draft Scoping Document, which details how they plan to conduct this assessment.  

All interested stakeholders are encouraged to submit comments and suggested refinements to the scope to ensure all perspectives are adequately considered.

Comments can be submitted by email to [email protected] and must be received by 5 PM ET on December 16, 2021. Please submit public comments as a Word document in the following format:

  • 12-point Times New Roman font
  • Three-page maximum (not including references or appendices)

ICER’s Patient Portal and Manufacturer Engagement Guide provide additional guidance for submitting public comments, including suggestions for what types of information may be most useful.

In addition to comments on the scope, ICER also welcomes submissions from stakeholders on examples of low-value care practices within this clinical area. These submissions will inform a report section focused on strategies to reduce waste and preserve resources for high-value, potentially higher-cost treatments.

More information is available at https://icer.org/news-insights/press-releases/icer-to-assess-gene-therapy-for-beta-thalassemia/.

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