BRIEF—ICER to review treatments for beta thalassemia

US health technology assessor The Institute for Clinical and Economic Review (ICER) has posted a  Draft Scoping Document outlining a planned review of the comparative clinical effectiveness and value treatments for beta thalassemia.

The products are: US biotech firm bluebird bio’s LentiGlobin; and luspatercept-aamt (Reblozyl, from Acceleron Pharma and Bristol-Myers Squibb/Celgene, for the treatment of beta thalassemia.

LentiGlobin is an investigational gene therapy currently marketed in Europe under the brand name Zynteglo; a US Food and Drug Administration decision regarding potential US approval is expected by late 2020.

Luspatercept-aamt was approved by the FDA in November 2019.

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-review.org and must be received by 5 PM ET on January 27, 2020.

All comments submitted must meet the ICER’s formatting specifications.