For years, researchers studying the bleeding disorders community have sought answers to questions about people’s experiences that only community voices can answer.
Now thanks to My Bleeding Disorders Community (MyBDC), a new community-powered registry from the National Hemophilia Foundation (NHF), these questions can finally begin to be answered. Through a series of surveys, the online platform collects data that will make a difference in research, education and ultimately people’s quality of life, says Michelle Witkop, DNP, FNP-BC, NHF’s head of research.
Toward better data
In the past several years, NHF’s Medical and Scientific Advisory Council (MASAC) recognized that the voices of people with bleeding disorders and their family members are key to better research. Others are looking for more community input too, including the Food and Drug Administration (FDA) and pharmaceutical companies. NHF’s public policy team has wanted data about people’s experiences to use in advocacy initiatives.
Bleeding disorders community members share details of their lives during focus groups, conferences and events at hemophilia treatment centers (HTCs). But NHF needed a more consistent way to collect and organize those experiences to turn them into useful data. “Singular voices are important,” Witkop says. “But to be able to put them all together and have a database of experiences that have happened will be very powerful.”
A veteran researcher and former lead clinician at the Northern Regional Bleeding Disorder Center in Michigan, Witkop joined NHF in 2017 and was tasked with building this new data collection program. She immediately thought of replicating the questionnaire style of data collection employed by the Nurses’ Health Study, one of the largest investigations of risk factors of major chronic disease in women.
“MyBDC is all survey-based and different from other bleeding disorders-related registries,” Witkop says. MyBDC surveys collect data on a wide range of topics, including diagnosis, inhibitor history, pain intensity, treatment, anxiety, depression, gene therapy and medical marijuana use. Female participants receive additional questions relating to women’s issues.
It is designed to complement information gathered by other registries, like the Centers for Disease Control and Prevention’s Community Counts monitoring program and the American Thrombosis & Hemostasis Network’s ATHNdataset, which collects population data about bleeds, treatment and other factors. MyBDC will provide additional insights to enhance research.
MyBDC is also open to parents, spouses, siblings, grandparents and children of people with bleeding disorders. “With this data, we’ll be able to understand what it really means to live with a bleeding disorder for all of those who are affected,” says NHF research nurse specialist Maria Santaella, MSN, RN-BC, CPHON, who manages MyBDC along with Witkop.
MyBDC is not just a tool to collect information to hand over to researchers. It will allow the community to have a say in shaping research. “Through this platform, we can prioritize gaps in education, care and resources as identified by the community. Looking at grouped responses, we can identify areas that could benefit from additional research, such as joint health, anxiety and depression. Then we will be able to funnel our research dollars into those areas as well as do more in-depth surveys to gather more information,” Witkop says.
How it works
MyBDC is accessible by smartphone, tablet or computer. The first step is filling out a short enrollment survey. Next is a longer baseline survey, which takes about 30 minutes. Information gathered in the baseline survey allows for individual tailoring of additional surveys. For example, a woman with a bleeding disorder might receive a quick survey on managing her condition during her menstrual cycle.
Going forward, participants will complete an annual “core survey,” which will repeat some questions year after year. “This is very important because this is how we’ll identify trends and figure out health changes within the community over time,” Santaella says. To protect users’ privacy, all participant data is de-identified (details such as name and date of birth are removed) and aggregated (responses are combined). “It’s not someone looking at your individual surveys,” Santaella says. “We take anonymity and security of the information very seriously.”
Beyond the core survey, people may take as many surveys as they like. The more surveys completed, the more data NHF can gather—and the more participants can learn about themselves, since they have access to their own data, Witkop says.
When the database is robust enough, researchers can submit applications to use MyBDC data in research projects, Witkop says. A committee will review all requests. For-profit entities, such as pharmaceutical companies, will be charged a fee to access the data on a revenue-based scale. “NHF will give relevant, de-identified, aggregate data to answer specific research questions,” Witkop says. “We may submit a survey developed by a company through MyBDC, but the responses provided will never be patient-level. They will always be grouped and de-identified.”
Community involvement, community benefits
At the start of planning for MyBDC, NHF conducted focus groups to learn what community members wanted out of a community-powered registry.
One topic that kept coming up, Witkop says, was people’s frustration that they never know what happens to the information they provide to researchers. This led to the creation of MyBDC’s personalized dashboard, a secured page where participants can view their survey responses and compare them with others’ anonymous responses.
“The dashboard is unique in bleeding disorders registries,” Witkop says. “And the more surveys people fill out, the more information they will be able to monitor over time and compare.” Santaella says community members get excited when she explains the dashboard to them. “It’s not a one-way thing. It’s a two-way communication so that participants can learn about themselves and learn how they relate to others. And they can follow trends over time,” she says.
Community members also were concerned about funding, wary of outside influence and their personal information being sold. NHF has committed to funding MyBDC for five years, says Witkop, but she hopes and anticipates it will go beyond that time frame. NHF received two grants from industry partners: one to develop the strategic plan, and one to embed education within the platform. “The industry partners don’t receive anything in return for their grants,” Witkop says. “The grants don’t allow any access to the data. All data requests will be treated the same, regardless of whether the requester provided funding.”
Highlighting all voices
MyBDC will amplify voices of groups that historically haven’t received as much attention, such as those with rare bleeding disorders, siblings and LGBTQ+ community members. “We only have a handful of people with factor XIII deficiency in the United States. If we can gather 10 of them in MyBDC and understand their experiences, their barriers to accessing care, we’ll be able to better help them,” Santaella says.
Amber Federizo, DNP, APRN, FNP-BC, director of research at the Hemostasis & Thrombosis Center of Nevada, is excited to use future data from MyBDC to advocate for women with bleeding disorders.
“Sometimes these women suffer in silence and they aren’t taken seriously. MyBDC is a chance for women to have an impact with their numbers, not just an anecdote in one center or another. This is an opportunity to bring them together nationally and say, ‘This is happening, and we have to address it,’” Federizo says. For example, women’s sexual health, such as bleeding during intercourse, can be glossed over for lack of strong data, says Federizo, who urges all women with bleeding disorders to participate in MyBDC. “If we don’t have the data, if people don’t get involved, we won’t know what’s going on and, unfortunately, much of the suffering will continue,” she says.
Improved education and advocacy
Another goal of MyBDC is to spot trends in the community and offer educational resources to help people better manage their health.
The dual survey and education components of MyBDC have already been put to use, Santaella says. When news broke of hospitalizations and deaths from vaping, NHF wanted to learn how MyBDC participants were affected. A short survey asked whether they had vaped or used e-cigarettes within the past year.
“If they replied yes, we asked whether they had any symptoms and we provided them with education around the issue in case they hadn’t seen the coverage in the news,” Santaella says. “It’s a perfect example of what we can do with MyBDC to benefit our community.”
MyBDC enrollees also have the opportunity to participate in voluntary and anonymous virtual advisory boards. These allow members to provide input to industry and the FDA on clinical trial design.
MyBDC will also play a role in political advocacy. NHF’s Chief Officer of External Affairs Michelle Rice offers this example: “If a state enacts a law that restricts access to a specific therapy, we can survey people and ask if and how they’re impacted. Then, when we are advocating to lawmakers, we can say, ‘This law is affecting X number of people.’ It gives real weight to our advocacy messages.”
Educating government and industry on the experiences and needs of people within the bleeding disorders community is another significant use of MyBDC’s robust data. “Personal stories are important to highlight, but nowadays strong data makes the difference,” Santaella says. “It’s going to help NHF do what it does well even better.”
MyBDC at a Glance
What is it?
MyBDC is a data collection platform and partnership between the bleeding disorders community and the National Hemophilia Foundation. MyBDC doesn’t conduct research; it collects data to be used in research and for enrollees to use to better their health.
Who can participate?
People with bleeding disorders and their family members, including parents, siblings, spouses and grandparents.
What do participants get in return?
MyBDC’s personalized dashboard allows users to compare their information with de-identified, aggregated data from other community members. Educational tools and resources help improve health. Virtual advisory boards allow participants to offer preclinical input on clinical trial design.
Who can apply to use the data?
Researchers, including students, industry partners and hemophilia treatment centers, can submit requests, which will be reviewed by a research committee.
Is the personal information collected safe?
Data is confidential and secure. Researchers will never have access to personal information or patient-level data.