The National Bleeding Disorders Foundation (NBDF) has been nominated for the Inaugural Prix Galien Patient First Award in Honor of Michael J. Fox by The Galien Foundation, a global institution recognizing groundbreaking achievements in the life sciences. The newly introduced award recognizes excellence in clinical research, clinical trials, and patient-centered care improvement initiatives that embody the principle "No decision for me without me."
The nomination is recognition of NBDF’s Bleeding Disorders Research Collaborative (BDRC), a patient-centered research initiative that brings together people affected by bleeding disorders, caregivers, health care professionals, researchers, advocates, government partners, and industry leaders to identify and address the community's most important research priorities. By placing lived experience experts (LEEs) at the center of the research process, BDRC helps ensure that scientific advances reflect the real-world needs of patients and families and are guided by principles of health equity, diversity, and inclusion.
“When patients define priorities, research targets what is most important to them,” said Maria Santaella, senior vice president of research strategy at NBDF. “And when they help design the studies, the studies are stronger, the resulting evidence is more relevant, and the path to implementation gets shorter.”
The collaborative effort began in 2020 with the launch of NBDF’s National Research Blueprint (NRB), which convened working groups—nearly half comprised of lived experience experts-- to identify key challenges and gaps in bleeding disorders research. The findings and recommendations of those working groups were recently published in the journal Expert Review of Hematology. The NRB was rebranded to the Bleeding Disorders Research Collaborative in 2025 to reflect the diversity of voices included in the initiative.
“For decades, patients entered research at the end of the pipeline, as subjects in studies whose questions others had already chosen,” said Santaella. “The Bleeding Disorders Research Collaborative rejects that: our lived experience experts are equal partners in prioritization, design, implementation, and analysis. And because this work was funded entirely by NBDF, without industry sponsorship, our priorities answer to the community, not to commercial interests. That changes the science.”
The BDRC has already produced results. In 2025, NBDF’s Research Roundtable confronted the underrepresentation of females in clinical research, now advancing through a PCORI-funded project to develop female-specific outcome measures. NBDF’s LEE Research Ambassador Program will provide LEEs with education and training on the important aspects of research and how they can meaningfully contribute their lived experiences to support the research goals.
The nomination recognizes the success of NBDF's patient-centered research model, which empowers people with bleeding disorders to actively participate in research study design, implementation, and evaluation. Through this collaborative approach, NBDF is helping advance more relevant, inclusive, and meaningful research for the bleeding disorders community.
“We are very proud of this nomination, which is a reflection of the hard work of our working group members, clinicians, researchers, and partners who contribute to the BDRC,” said Santaella. “But most importantly, it recognizes one of the central tenets of NBDF: lived experience experts are not passive bystanders to science; they are essential partners in it.”
