— Report will be subject of New England CEPAC meeting in June 2022; Draft Scoping Document open to public comment until December 16, 2021  —

BOSTON, November 24, 2021 – 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.  

The assessment will be publicly discussed during a meeting of the New England CEPAC (New England CEPAC) in June 2022, where the independent evidence review panel will deliberate and vote on evidence presented in ICER’s report.

ICER’s website provides timelines of key posting dates and public comment periods for this assessment.

Consistent with ICER’s process for announcing new assessments, we have spent the past five weeks engaging with targeted stakeholders, including relevant patient groups, the manufacturer of the treatment being assessed, clinical specialists, as well as payers. Based on this preliminary cross-stakeholder engagement, today ICER has posted a Draft Scoping Document outlining how we 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 publiccomments@icer.org 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 in the scoping document. Following the public comment period, a revised scoping document will be posted on or about December 23, 2021.

About ICER

The Institute for Clinical and Economic Review (ICER) is an independent non-profit research institute that produces reports analyzing the evidence on the effectiveness and value of drugs and other medical services. ICER’s reports include evidence-based calculations of prices for new drugs that accurately reflect the degree of improvement expected in long-term patient outcomes, while also highlighting price levels that might contribute to unaffordable short-term cost growth for the overall health care system. For more information about ICER, please visit ICER’s website.