ICER to Assess Gene Therapy for Hemophilia A

— Report will be subject of New England CEPAC meeting in August 2020; Open Input now being accepted until January 22, 2020 —

BOSTON, January 3, 2020 – The Institute for Clinical and Economic Review (ICER) announced today that it plans to assess the comparative clinical effectiveness and value of valoctocogene roxaparvovec (BioMarin Pharmaceutical, Inc.) for the treatment of hemophilia. Valoctocogene roxaparvovec is an investigational gene therapy, and a decision regarding FDA approval is expected by mid-2020. ICER’s analysis will likely compare valoctocogene roxaparvovec to factor VIII replacement therapy and emicizumab (Hemlibra®, Genentech).

ICER’s Evidence Report on hemophilia A will be reviewed during a public meeting of the New England Comparative Effectiveness Public Advisory Council (New England CEPAC) in August 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 January 22, 2020 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 hemophilia A.

For more information about the Open Input period, visit ICER’s website. ICER’s Manufacturer Engagement Guide, Patient 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.org and must be received by 5 PM ET on January 22, 2020 to be considered.

A draft scoping document, which will provide more detail on ICER’s planned analysis, will be available on January 24, 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 patients, clinicians, insurers, and policymakers can improve the quality and value of health care. For more information about ICER, please visit ICER’s website.