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ICER Review of Emicizumab for People with Hemophilia A and Inhibitors

April 16, 2018

On April 16th, 2018 the Institute for Clinical Effectiveness and Review (ICER) released its review of Emicizumab for People with Hemophilia A and Inhibitors. ICER is an independent and non-partisan research organization that evaluates the clinical and economic value of prescription drugs, medical tests, and other healthcare and healthcare delivery innovations.

ICER’s review examined evidence for people with hemophilia A and inhibitors. Their review compared prophylaxis with Emicizumab with treating on-demand and prophylaxis with a by-passing agent. In both situations, ICER’s analysis found that prophylaxis with Emicuzmab reduces cost while improving the health of people with hemophilia A and inhibitors.

In the review, ICER creates an economic model of costs and clinical effectiveness based on data reported from clinical trials and studies of the relevant treatments. They also considered “other benefits and contextual considerations” – such as the impact of a treatment on a patient or caregiver’s quality of life or ability to attend school or work – that are important to individuals but for which there may not be rigorous evidence.

ICER sought participation from and comment by NHF throughout its process over the last several months. NHF submitted a number of written comment letters to ICER in conjunction with HFA, and attended and provided testimony at the in-person meeting of the ICER review panel in Boston in March. NHF’s statement was summarized in the final report below.

As NHF’s statement concludes:

The National Hemophilia Foundation (NHF) is the nation’s leading advocacy organization working to ensure that individuals affected by hemophilia and related inheritable bleeding disorders have access to quality medical care, regardless of financial circumstances or place of residence.

Having a bleeding disorder with an inhibitor has a profound impact on individuals and their families – affecting their daily lives and decisions related to work, school, play, physical activity, family life, and access to insurance and related health care costs.  Along with short- and long-term physical effects of internal bleeding and pain that result, psychosocial effects are significant. Affected families face countless sleepless nights, trips to their hemophilia treatment center, emergency rooms, hospital admissions, and a significant disruption to normal family life.

As a result, a number of “other benefits and contextual considerations” are relevant to ICER’s review of Emicizumab, including those related to patient outcomes and quality of life; family and caregiver burden and ability to return to work; reduced complexity of administration; and hemophilia with inhibitors being a condition with a significant effect on quality of life and a high lifetime burden of illness.

NHF is pleased that there has been continued investment and additional treatments approved to treat inhibitors. Emicizumab is a critical new option for individuals with inhibitors, but NHF’s position is that patients together with their doctors must decide what treatment options are best. Every person’s response to treatment is different and a thorough evaluation of risks and benefits and discussion between patient and treater is required. 

Read Emicizumab for People with Hemophilia A and Inhibitors.