–Document open to public comment until September 29, 2017–
Boston, Mass., September 11, 2017– The Institute for Clinical and Economic Review (ICER) has posted a Draft Scoping Document outlining its planned review of the comparative clinical effectiveness and value of emicizumab for prevention of bleeding in patients with hemophilia A who have inhibitors to coagulation factor VIII. The scoping document will be open to public comment for three weeks until Friday, September 29, 2017 at 5pm ET. It will inform development of a report that will be subject to public deliberation during a March 2018 meeting of the New England Comparative Effectiveness Public Advisory Council (New England CEPAC), one of ICER’s three independent evidence appraisal committees.


ICER’s report will evaluate the comparative clinical effectiveness and value of emicizumab in patients who have hemophilia A with factor VIII inhibitors. Factor VIII is the clotting protein deficient in patients with hemophilia A, and inhibitors interfere with therapies aimed at replacing the protein. ICER’s report will evaluate data on key outcomes measured in clinical trials for emicizumab, such as bleeding, joint damage, and quality of life, compared to data on similar outcomes in patients taking a bypassing agent – the current treatment standard. Bypassing agents can be taken either for the prevention of bleeds, or on an as-needed basis when patients experience a bleed.

The Draft Scoping Document was developed with input from key stakeholders, including patients and patient advocacy groups, who provided important insights into the context of treatment decisions. For example, patients and advocacy groups explained that risks of bleeding and the frequency with which infusions of bypassing agents are needed significantly affect the ability of patients and caregivers to participate in work, school, and recreational activities, and physicians stressed that treatment adherence can be affected when infusions are required frequently. ICER will continue to engage with patients, clinicians, drug manufacturers, and other stakeholders to assess appropriate outcomes and comparators for the review, and to enrich the evidence available from clinical trials.

All interested stakeholders are encouraged to submit comments and suggested refinements to the scope to ensure all perspectives are adequately considered. All comments must be received by September 29th at 5pm ET. Comments can be submitted by email to publiccomments@icer-review.org. More information about submitting a public comment, including formatting specifications, is available on ICER’s website. ICER’s Patient Participation Guide and Manufacturer Engagement Guide provide additional guidance for submitting public comments.

Following the public comment period, a revised scoping document will be posted on or about October 10, 2017.


During the March 2018 public meeting, the New England CEPAC will vote on key questions raised in the report, and a policy roundtable of patient representatives, clinical experts, drug manufacturers, and payers will discuss recommendations for how best to apply the evidence to policy and practice. The New England CEPAC, composed of practicing physicians, methodological experts, and leaders in patient advocacy, provides objective, independent guidance on the application of medical evidence to clinical practice and payer policy decisions.

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.