By Fiona Soltes
Esmeralda Vázquez is finding herself in places she previously couldn’t have imagined. She’s not only the colleague of scientists, doctors and PhDs. She’s also a subject matter expert, with full understanding that her lived experience can help change the course of research for her community and for generations to come.
Vázquez, diagnosed with Glanzmann’s thrombasthenia as an infant, said for some time, she wasn’t in the habit of disclosing her ultra-rare bleeding disorder. But she has become increasingly involved and outspoken—and is feeling valued as a result.
“If I’m going to be born with this,” she said, “I want to do something good with it.”
Vázquez is a tangible representation of NHF’s progress toward a National Research Blueprint. The community-driven initiative aims to shape the future of research together with the researchers, HTCs, nonprofits, government, industry partners—and those personally affected. Those in that final category, the true subject matter experts (SMEs), can help the others “look at things through our lens,” Vázquez said.
Vázquez, who spoke during the Friday afternoon session, You Can Impact the Future of the Inherited Bleeding Disorders Community: The National Research Blueprint, explained the broad role of the SME. Beyond increasing understanding of the research process, she said, SMEs can contribute to research ideas and planning; support recruitment efforts; assist with dissemination of research study results; educate regarding healthcare inequalities/social determinants of health; and build trust between the community and research.
“You know what you need in order to build your best life, and we need to be able to build that into our research plans,” said Kevin Mills, PhD, NHF chief scientific officer, during the session. He reviewed the goals for the Blueprint, outlined progress, and touched on the effort’s various working groups, ranging from community engagement to health equity, diversity and inclusion (HEDI). The Blueprint, to be launched in 2023, will not just be for the community, Mills said, “but by the community and with the community.”
The session included a brief panel discussion with Mills; Vázquez; Leonard Valentino, MD, NHF president and CEO; and Donna DiMichele, MD, a consultant with decades of experience in academic pediatric hematology practice and clinical hemophilia research.
One audience member with severe hemophilia A noted that he’d had some nontypical symptoms in response to recombinant medications. He wondered about next steps for sharing information with the broader community without, for example, being in a clinical trial. He was given several options, starting with talking to his provider. Beyond that, he—and others—can submit information directly to the FDA about what might be considered an adverse event; participate in the American Thrombosis and Hemostasis Network (ATHN) and the CDC’s Community Counts; and take part in NHF’s Community Voices in Research registry.
“Everybody in our community needs to get on board with this,” Valentino said. “Whether you’re in a chapter, or an HTC, or in industry, this is our future. These are the questions that people identified in the community. These are the gaps in care that were identified by community member, not NHF…. I think it’s incumbent upon all of us in this room now to go recruit everybody else to start participating, to get involved in this. And I’m sure we’ll hear more about ways that people can get involved as we move forward.” ■