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While the long-range goal is looking for cures for all bleeding disorders, she said, “in the meantime, it means ensuring that families affected by the disorder can thrive. A world without inheritable blood disorders really begins with research.”
The lived experience expert (LEE) has an important seat at the table, she said. Despite the disruption of the COVID-19 pandemic, focus groups and community interviews were held as part of Phase One of the NRB. “We involved the whole community,” Santaella said. “When we talk about the community, we’re talking about the whole community, industry partners, the government, researchers, members of the multidisciplinary team.”
One of the earliest outputs was to rename the patient and family groups. Formerly known as subject matter experts (SMEs), that placed an unnecessary burden to “explain themselves and what they were experts in,” Santaella said. The working group came up with the term lived experience expert and defined it as “individuals, caregivers and family members directly impacted by inheritable bleeding disorders. Their diverse and public knowledge gives them the unique ability to translate lived experiences into meaningful system change.”
The NRB is in Phase Two of its development, where priorities – outlined in Phase One – are defined. “Now we know what we need to study,” Santaella said. “What we need to figure out is how we do it in a way that involves the whole community.”
While Phase Two is not complete, Phase Three is in its initial stages, where planning and fundraising is taking place with a goal of “figuring out how to implement those research plans.”
The overview of the NRB was followed by a panel discussion that included Donna DiMichele, MD, Sammie Valadez, co-chair of the National Research Blueprint Lived Experience Expert (LEE) Working Group, and Len Valentino, MD, National Bleeding Disorders Foundation president and CEO. The discussion was moderated by Michael Recht, MD, PhD.
“This is a top priority,” Dr. Valentino said. “It came from the board of directors. It goes hand-in-glove with access to care. We need to have innovation to get better, safer, more effective products and better care. The whole NRB is aimed at doing exactly that: How can we drive innovation in the community to drive your health and well-being going forward?”
The knowledge of LEEs is crucial to the success of research, Valadez said. “Research was not at the top of my priority list to begin with. The more I’ve learned about how much we need, it has grown substantially. When we came together in April, from those focus groups, I went knee-deep that day and have been over my head since then.”
In January 2024, an NRB Summit will be held, where a comprehensive research plan will be unveiled. “The goal is simple in theory, but not so simple in practicality: markedly accelerate real progress that makes a real difference to patients and families, and to do it soon,” Santaella said.
