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.
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.
The benefit/risk profile of emicizumab, as outlined in the prescribing information has not changed since it was approved in people with hemophilia A with and without factor VIII inhibitors.
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.