Frequently Asked Questions

 

Here you will find frequently asked questions about this website. For other questions or more detailed answers, please contact your local Roche representative.

This website has been created to provide 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.

This website includes links to published materials with information on emicizumab, including safety and efficacy, quality of life and real-world data. These materials may include scientific congress presentations and peer-reviewed manuscripts. In addition to emicizumab-specific information, a series of educational materials related to the reporting of safety data and pharmacovigilance practices are available.

Materials on this website only include emicizumab related peer-reviewed and published information and exclude opinion or editorial pieces (such as Letters to Editor, news articles, testimonials or commentaries), as well as articles referring to emicizumab use in off-label indications.

Once you confirm that you are a healthcare professional, you may ask to be kept informed about updates to the website by contacting your local Roche representative.

We will ensure patient organisations 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 organisation, if they have any questions in respect to their haemophilia management. Emicizumab’s label, as approved for use by health authorities, including the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA), 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, including EUHASS (European Haemophilia Safety Surveillance), FAERS (FDA Adverse Event Reporting System), and UKHCDO (United Kingdom Haemophilia Centre Doctors' Organisation). We have also worked with experts in the field to develop a series of manuscripts reviewing mortality in haemophilia A and using this to establish a framework to further assess the reported fatalities in people receiving emicizumab.

These manuscripts have been published in the Journal of Thrombosis and Hemostasis in December 2020 and are available on this website under the Manuscripts/Safety section.

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 Roche's ability to confirm individual details is variable. In addition, the information on adverse events provided to Roche from consumers or physicians after approval is confidential, and detailed information can not be shared without the consent of the reporting person, the patient or patient’s family. Patient privacy is very important to Roche, so we are also careful not to disclose specific details about an adverse event that could jeopardise the privacy of either the patient or their family, or breach patient confidentiality.

We also continue to work with experts in the field to collect, analyse, and publish relevant data and information related to the safety and efficacy of emicizumab. We will share links to these published materials on this website as they become available.

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 haemophilia 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 in countries where emicizumab is approved for use, has not changed since it was approved in people with haemophilia A with and without factor VIII inhibitors.

As of February 2021, over 10,000 people have been treated with emicizumab (Source: F. Hoffmann-La Roche, 2021. Data on file.).

Should any adverse event or side effect be experienced please refer to https://medinfo.roche.com/.