Beta Thalassemia

New England CEPAC

ICER plans to assess the comparative clinical effectiveness and value of LentiGlobin (Bluebird Bio) and luspatercept-aamt (Reblozyl®, Bristol Myers Squib and Celgene) for the treatment of beta thalassemia. LentiGlobin is an investigational gene therapy currently marketed in Europe under the brand name Zynteglo®; an FDA decision regarding potential US approval is expected by late 2020. Luspatercept-aamt was approved by the FDA in November 2019.

Date of Review: July 2020

For questions, please contact Catherine Koola, Program Manager, at ckoola@icer-review.org.

Associated Meetings

July 30, 2020 10am-4pm ET

TBA


Key Dates

Associated Materials

12/05/2019 – 12/20/2019

01/06/2020

01/06/2020 – 01/27/2020
Public Comments

01/06/2020

02/04/2020
Revised Scoping Document

03/11/2020
Research Protocol

04/06/2020
Model Analysis Plan

05/27/2020
Draft Evidence Report

05/27/2020
Draft Voting Questions

05/27/2020 – 06/23/2020
Public Comments

07/16/2020
Evidence Report

07/16/2020
Revised Voting Questions

07/16/2020
Response to Public Comments

07/30/2020
Meeting Agenda

07/30/2020
Evidence Presentation

08/20/2020
Final Evidence Report and Meeting Summary

08/20/2020
Report-at-a-Glance