BOSTON, March 15, 2018 – The Institute for Clinical and Economic Review (ICER) today released an Evidence Report assessing the comparative clinical effectiveness and value of emicizumab (Hemlibra®, Genentech). The report found that emicizumab offers important improvements in outcomes for people with hemophilia A and inhibitors to Factor VIII, while lowering costs associated with the treatment of this condition.
Emicizumab was approved by the FDA in November of 2017 for prophylaxis (prevention of bleeding) in adults and children who have hemophilia A with coagulation factor VIII inhibitors. Factor VIII is the clotting protein that is deficient in those with hemophilia A, and inhibitors interfere with the factor replacement therapy that would typically be used to treat the condition.
This Evidence Report will be the subject of an upcoming public meeting of the New England Comparative Effectiveness Public Advisory Council (New England CEPAC) in Cambridge, MA on March 29, 2018. The New England CEPAC is one of ICER’s three independent evidence appraisal committees comprising medical evidence experts, practicing clinicians, methodologists, and leaders in patient engagement and advocacy.
“Emicizumab appears to be a very rare win-win-win in treating a small population of patients who have hemophilia and cannot be treated with factor VIII,” noted David Rind, MD, MSc, ICER’s Chief Medical Officer. “Not only does evidence suggest that the therapy improves patient health outcomes, which has a significant impact on patient quality of life, but it also offers substantial cost savings to the health care system, and administration is far less burdensome for patients than the previous standard of care. During our public meeting later this month, we will work with patients and other stakeholders to identify ways in which the evidence can be used to improve patient care. We will also explore ways to increase affordability for individuals and the health care system given the extremely high costs of treating hemophilia A.”
A draft version of this report was previously open for a four-week public comment period. The updated Evidence Report reflects changes made based on comments received from patient groups, clinicians, drug manufacturers, and other stakeholders. Detailed responses to public comments can be found here.
Key Report Findings
ICER’s assessment found that evidence provides high certainty of a substantial net health benefit of emicizumab compared with no prophylaxis for people ages 12 and older for whom treatment with immune tolerance induction has been unsuccessful. For children younger than 12 years of age, there is high certainty that emicizumab provides a small or substantial net health benefit compared with no prophylaxis, and in both adults and children, there is also high certainty of a small or substantial net health benefit compared to prophylaxis with bypassing agents.
Not only does emicizumab provide improved clinical outcomes for patients, but also long-term cost-effectiveness analyses highlighted that it offers overall cost savings, both from a health system perspective considering only direct medical costs, and from a societal perspective considering broader benefits. Importantly, this judgment applies only to the currently indicated population, and would not necessarily apply to broader populations potentially included in any future expanded indications.
ICER’s review also considered additional benefits of the medications not included in clinical trials. When comparing emicizumab to other therapies, caregivers and people living with hemophilia noted that emicizumab offers significantly improved convenience, which may improve adherence. Further, access to a more effective therapy may have important effects on individuals’ career and education choices and may allow children to participate in activities from which they would previously have been restricted. The Evidence Report, accompanying voting questions, all public comments received on the draft report, and ICER’s written response to comments, are available on the ICER website.
During the March public meeting, pre-registered stakeholders will provide brief oral comments on the report and its findings, the New England CEPAC will vote on key questions raised in the report, and a roundtable of experts will discuss recommendations for applying the evidence to policy and practice. Requests to make an oral comment were accepted during the public comment period on the Draft Evidence Report and are now closed.