BRIEF—ICER to assess gene therapy for beta thalassemia

US health technology assessor the Institute for Clinical and Economic Review (ICER) announced yesterday that it plans to assess the comparative clinical effectiveness and value of LentiGlobin from US biotech bluebird bio 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.

In Europe, bluebird has priced Zynteglo, offered as a one-time infusion, at nearly $1.8 million, with the cost spread over five years.

The ICER’s Evidence Report on beta thalassemia will be reviewed during a public meeting of the New England Comparative Effectiveness Public Advisory Council ( New England CEPAC) in July 2020.

An Open Input period began Thursday and is intended to allow stakeholders to share key information relevant to the development of the evidence report.

Comments will be accepted from all interested stakeholders until December 20, 2019 at 5pm ET. During this time, the ICER will also contact key patient groups and clinical experts to gain further insights on the patient perspective and clinical context of treating beta thalassemia.