Data That Changes Lives: How Patient Registries Drive Research

Just answering a few questions about how your bleeding disorder affects your life can make a huge difference in treatment in care was the message of “Change the World with Your Experience: Your Data!” on Saturday, August 23.

The session featured experts from the American Thrombosis and Hemostasis Network (ATHN):  Tammuella Chrisentery-Singleton, MD, chief science officer and Sarah Gonzales, Community Counts Surveillance Project. NBDF was represented by Matt Hartnett, senior community research network manager. Sharing their unique perspectives as patients who have participated in research themselves were Paxon Mills; and Peter Zdziarki, vice president, Glanzmann’s Research Foundation. 

Ms. Mills and Mr. Zdziarki kicked us off with their perspectives, grounding the session in the lived experience. Dr. Singleton followed by explaining the different types of data collection efforts in the bleeding disorders community – including ATHN, Community Counts and Community Voices in Research, a community-powered registry supported by NBDF.

Since its founding in 2006, ATHN has collected clinical data through hemophilia treatment centers (HTCs) and other clinics. For those who participate, healthcare providers submit de-identified information about them such as: diagnosis, how often they have bleeds and where, and other details about their disorder. This data is then combined with information from other participants. Researchers, clinicians and advocacy organizations use it to study patterns, assess treatment effectiveness, and understand  long-term health outcomes. 

ATHN also conducts targeted studies. Currently open for enrollment include ATHN Transcends, which is a natural history study assessing the safety, effectiveness, and practice of treatment in people with inheritable bleeding disorders, ATHN 16, which is studying the safety of SEVENFACT® to treat bleeds in people with hemophilia A and B with inhibitors, and ATHN 10, which is studying people with rare coagulation disorders. 

Sarah Gonzales provided an overview of Community Counts, a public health surveillance program funded by the Centers for Disease Control and Prevention (CDC) to gather and share de-identified medical information about people with bleeding disorders who receive care at US HTCs. Despite the invasive-sounding name, “surveillance” means keeping track of specific health information over a long period of time – in this case, information from people with bleeding disorders. The combined data is then shared with healthcare providers, researchers, public health officials, and the public to guide decision making, improve treatment strategies, and better understand the health trends, and quality of life impacts for people with bleeding disorders. She stressed that patient participation in studies is critical to improve care: “the better the participation, the better the data, and the better the data, the better your care.”

Patient experience is at the center of NBDF’s newly renamed Bleeding Disorders Research Collaborative, explained Matt Hartnett. Formerly the National Research Blueprint, this program seeks to address gaps in knowledge that can improve the lives of people affected by inheritable bleeding disorders, ensure that people with lived experience-- lived experience experts, or LEEs-- are directly involved with the research process, and promotes equity so research findings reflect the entire bleeding disorders community. One of the key ways to participate is through NBDF’s own registry, Community Voices in Research (CVR). CVR gathers data directly from LEEs themselves, rather than through healthcare providers. This patient-reported information complements other research efforts. It also helps identify research priorities important to the community and creates opportunities for participants to take part in additional research studies. By contributing to CVR, LEEs help shape the research that may ultimately improve their care. To read more or enroll, follow this link. 

Hartnett also detailed NBDF’s research ambassador program, which connects LEEs with researchers to co-design more relevant, inclusive and impactful research projects. By bringing the voice of the community directly into the research process, this program ensures that studies address real-world needs and priorities. 

The takeaway of the session was clear: Participation in research, whether through registries or through taking a more active role as a research ambassador, results in better health outcomes for the entire bleeding disorders community. You can get involved with NBDF’s research programs here.