EBCare Registry, developed and funded by the biotechnology company Lotus Tissue Repair in collaboration with DebRA of America, DEBRA International and experts in the field of EB medical research and advocacy. Recently, we connected with Jennifer Gorzelany, an advisor to Lotus Tissue Repair, to discuss the Registry.
In this first part of the two-part interview, Gorzelany shares the history behind EBCare and attempts to clarify misconceptions about the Registry and its mission.
EB Resource (EBR): How were you first introduced to epidermolysis bullosa? What has been your own personal experience with EB?
Jennifer Gorzelany (Jennifer): I have worked at two biotechnology companies that have developed and launched therapies for rare genetic diseases, and I currently work with several companies in similar disease areas. So I have a longstanding interest in rare diseases. Before becoming involved with EBCare and Lotus Tissue Repair, I had never heard of EB. Over the last year, I have had the opportunity to meet many patients and families and have been touched by their stories. I am proud to be associated with the EB community and inspired to do all I can to help.
EBR: And what is the history behind the Registry? When did it officially launch and what led to its development?
Jennifer: DebRA of America, DEBRA International and Lotus Tissue Repair wanted to develop additional ways to help the EB community. Many other rare disease patient organizations and companies have built registries that played a key role in advancing better care and treatments. The success of other registries led to the formation of EBCare and the EBCare Registry. The EBCare Registry was launched in February 2012.
EBR: What have you found to be some of the biggest misconceptions about the EBCare Registry?
Jennifer: A common concern among patients and families is confidentiality. The information a patient or family provides to the Registry is strictly confidential and the Registry is compliant with U.S. and international privacy and data security regulations. Physicians or researchers who want to use the registry information in their work must submit a written request to the EBCare Board of Managers, describing why they would like to use the data. The Board reviews these requests, and Registry information is only shared with the Board’s approval. The information provided to physicians and researchers is “de-identified.” This means that no information that would make it possible to identify a patient or family is revealed.
EBR: This interview is a great opportunity to clarify what the EBCare Registry is all about. Can you tell our readers what EBCare is and isn’t, especially for those who are unfamiliar with it?
Jennifer: The EBCare Registry is the first self-reported registry for EB. Patients or their caregivers can enter much needed information about their diagnosis, symptoms, wound care, medical treatment and quality of life into the Registry. By participating in the Registry, patients supply information that can be used by EB advocacy groups, physicians treating EB, and researchers studying EB — potentially leading to better care and new treatments for the disease. Registries like the EBCare Registry are especially important in the case of rare diseases, because with so few patients, these diseases are often not well characterized or understood. By providing information through the Registry, patients can play a key role in advancing better care and treatments.
The fact that the EBCare Registry includes ‘self-reported’ data is very important, because patient- or caregiver-reported information offers different and potentially more intimate insights about the condition than physician-reported information. For example, an organization like DebRA of America could use the Registry data collected regarding the overall cost of EB to make a more compelling case to insurance companies and healthcare payers as to why the cost of wound care supplies should be covered. Also, companies developing new EB treatments, including Lotus Tissue Repair, are particularly interested in understanding patient-reported symptoms and quality of life information, to ensure that during clinical trials, we more accurately determine what a clinically meaningful benefit is. The answers to these questions are more comprehensive and complete when reported by patients or their caregivers.
As for what the Registry is not, it is not a marketing tool, and participants’ names are never shared without their consent. While DEBRA International, DebRA of America and Lotus Tissue Repair developed the Registry, any qualified researcher whose goal is to help the EB community can request the de-identified Registry data.
EBR: What are the overall objectives of having a registry like this in place?
Jennifer: The goal of the EBCare Registry is to securely and confidentially collect as much self-reported information about the experiences of EB patients and their families as possible, and to provide de-identified disease information to qualified doctors and researchers. By gathering information on how EB impacts the lives of those with the disease and their families, we hope to support advocacy, improve care and encourage the development of new treatments.