This is a medical website developed by healthcare professionals at Genentech/Roche and is intended to provide US healthcare professionals with information regarding emicizumab from available clinical research data, real-world data and data from registries. Our aim is to share new data and findings from ongoing studies and real world use with emicizumab, to give an up-to-date, full and comprehensive summary of emicizumab’s long-term safety and efficacy outcomes, providing valuable information that we hope will allow more informed treatment decision making.
You may read our letter to the hemophilia community from September 2020 about this website here.
A website has been developed for US patients and caregivers and may be accessed at www.emipatientinfo.com. The patient and caregiver website is intended to provide patients and caregivers with information on topics of interest for emicizumab, including patient-friendly summaries of these information. The information provided is only for educational purposes and is intended to support informed and meaningful treatment discussions between patients and their healthcare providers.
Further, we will ensure patient organizations receive the appropriate information regarding the safety and efficacy of emicizumab in a timely manner, so they may inform the patient communities that they serve. Patients and their caregivers should speak with their treating physician or local patient organizations, if they have any questions in respect to their hemophilia management. The Hemlibra Prescribing Information, as approved by the US Food and Drug Administration (FDA), should always be the primary source of information on the safety and efficacy of the medicine.
Updates on the number of fatalities in people treated with emicizumab, whether known to be related to emicizumab or not, are and will continue to be included in public safety registries and databases, such as FAERS (FDA Adverse Event Reporting System).
In regards to providing case details or causality, we are not able to provide extensive information related to serious adverse events or fatalities reported in the post-approval setting because the level of detail available and Genentech/Roche's ability to confirm individual details is variable. In addition, the information on adverse events provided to Genentech/Roche from consumers or physicians after approval is confidential, and detailed information cannot be shared without the consent of the reporting person, the patient or patient’s family. Patient privacy is very important to Genentech/Roche, so we are also careful not to disclose specific details about an adverse event that could jeopardize the privacy of either the patient or their family, or breach patient confidentiality.
We have worked with experts in the field to develop a series of manuscripts reviewing mortality in hemophilia A and using this to establish a framework to further assess the reported fatalities in people receiving emicizumab. These manuscripts were published in the Journal of Thrombosis and Hemostasis in December 2020 and can be found on this website under the Publications and Scientific Presentations/Hemophilia A and Mortality section.
Patient safety is of the highest importance to us. Information on any safety events that impact the overall benefit/risk profile of emicizumab will be shared as quickly as possible with regulatory bodies and the hemophilia community. Updates on the number of severe adverse events in people treated with emicizumab, whether known to be related to emicizumab or not, will be shared in safety registries and databases, as well as through peer-reviewed publications and conference abstracts that are planned across 2020 and beyond.
As of February 2021, more than 10,000 patients worldwide have been treated with emicizumab (Source: Genentech/Roche Data on File 2021). The overall benefit-risk for emicizumab remains favorable.
The last updates, published with a cutoff date of June 30, 2020, can be found on the “Safety” page of this website. These have been archived and will not be further updated. Moving forward, we will share data and findings on the safety and effectiveness of emicizumab from peer-reviewed publications and presentations to the scientific community.
The quarterly updates were first established after the initial approval of emicizumab by the FDA in November 2017. The transition from clinical development to approved access for patients in many countries has meant a move from single patient reports to large databases of information on the use of emicizumab. Having a much broader availability of information from databases and individual experiences, means we can see early signals, identify trends and improve our overall understanding of the safety and efficacy profile of emicizumab. During this transition, the source of safety information has also expanded from controlled clinical trial reporting to ongoing voluntary adverse event (AE) reporting by healthcare professionals, patients and their caregivers, changing the level of detail of information about individual safety cases that we receive. In light of this transition, and so that we continue to ensure transparency, and aim to meet the community’s expectations and needs for information on hemophilia treatments, we have evolved our communication of post-approval information on emicizumab.