ICER to Assess Gene Therapy for Beta Thalassemia

— Report will be subject of New England CEPAC meeting in July 2020; Open Input now being accepted until December 20, 2019 —

BOSTON, December 5, 2019 –The Institute for Clinical and Economic Review (ICER) announced today that it plans to assess the comparative clinical effectiveness and value of LentiGlobin (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.

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.

ICER’s website provides timelines of key posting dates and public comment periods for this assessment. An Open Input period begins today 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, 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.

For more information about the Open Input period, visit ICER’s website. ICER’s Manufacturer Engagement GuidePatient Participation Guide, and Patient Guide to Open Input provide additional information for manufacturers and patient groups, including an explanation of what types of information may be most informative. There are no page limits to Open Input submissions, and input received will be incorporated throughout report development. All input can be emailed to publiccomments@icer-review.org and must be received by 5 PM ET on December 20, 2019 to be considered.

A draft scoping document, which will provide more detail on ICER’s planned analysis, will be available on January 6, 2020. That document will be open to public comment for three weeks.

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.

ICER’s reports incorporate extensive input from all stakeholders and are the subject of public hearings through three core programs: the California Technology Assessment Forum (CTAF), the Midwest Comparative Effectiveness Public Advisory Council (Midwest CEPAC), and the New England Comparative Effectiveness Public Advisory Council (New England CEPAC). These independent panels review ICER’s reports at public meetings to deliberate on the evidence and develop recommendations for how