The Boston-based Institute for Clinical and Economic Review (ICER) says a “draft scoping document” outlining its planned comparative review of emicizumab‘s clinical effectiveness and value in treating hemophilia A will be available until Sept. 29.
ICER’s report will be subject to a public deliberation in March 2018 by the New England Comparative Effectiveness Public Advisory Council (New England CEPAC), according to a press release.
Emicizumab, an antibody that’s also known as RG6013 or ACE910, replaces the function of clotting factor VIII, which is missing in hemophilia A. The investigative therapy can bridge the gap between factors IXa and X — a required initial step to trigger the natural clotting cascade. Emicizumab was created by Japan’s Chugai Pharmaceutical and is being co-developed with Roche and Genentech.
ICER will evaluate clinical data obtained from the Phase 3 HAVEN 1 study (NCT02622321) and ongoing HAVEN 2 study (NCT02795767). The committee will review overall clinical key outputs such bleeding rates, joint damage and impact on quality of life compared to those seen in patients undergoing current standard care with available bypassing therapeutics.
ICER’s Draft Scoping Document — based on input from patients and advocacy groups — considered both the treatment decision process and the impact of therapy. The review committee will keep meeting patients, caregivers, clinical teams, drug companies and others to ensure an appropriate assessment of all efficacy and safety issues that prove emicizumab is superior to current treatments.
ICER encourages all interested parties to submit comments and suggestions via email to [email protected], until Sept. 29 at 5 p.m. Eastern time. For more information, visit ICER’s website and its Patient Participation Guide. A revised scoping document will be made available by Oct. 10, after the public comment period ends.
The New England CEPAC, made up of clinicians, patient advocacy leaders and methodological experts, will vote on the main questions raised in ICER’s report. A final roundtable with patient representatives, clinical experts, pharmaceutical officials and insurance companies will deliberate how to apply the report’s analysis to policy and practice.