The NBDF Research Roundtable brings together key stakeholders to address current challenges in clinical trial design for inheritable bleeding disorders (BDs). In a pre-competitive product agnostic environment, the Research Roundtable convenes lived experience experts (LEEs, people directly impacted by BDs), researchers, non-profit organizations, and industry and government partners, including the Food and Drug Administration (FDA). It fosters focused discussions aimed at advancing clinical trial design, accelerating drug development, and enhancing patient care across the bleeding disorders community. NBDF plans to host this meeting on an annual basis, each time with a different foci.
The inaugural 2025 NBDF Research Roundtable will center on the inclusion of women, girls, and people who have or have had the potential to menstruate (WGPPM) in clinical trials, an essential step toward advancing equitable care. Historically, WGPPM have been underrepresented in trials due to concerns around reproductive health, the perceived complexity of hormonal cycles, and outdated assumptions that WGPPM are not significantly affected by BDs and results in men automatically apply to them. These barriers have limited our understanding of treatment effects across sexes, ultimately affecting WGPPM’s health outcomes. NBD’s Research Roundtable will address these issues by uniting LEEs, researchers, industry and regulatory experts to develop practical strategies for designing trials that fully include and represent WGPPM, ensuring more comprehensive data to improve care for all.
Goals
- Identify barriers to the inclusion of WGPPM in clinical trials for inheritable BDs, including those tied to reproductive health, hormonal cycles, and historical biases.
- Convene LEEs, researchers, clinicians, industry and government partners to address regulatory considerations and explore LEE-centered approaches for inclusive trial designs.
- Develop strategies to overcome identified barriers and establish shared guidelines and best practices to enhance diversity in clinical trials.
- Identify actionable steps, timelines, specific and measurable goals, and collaborative opportunities to implement strategies.
Statement of Need
Although the inheritable BDs community has seen significant advancements in recent years thanks to new and innovative therapies and improved diagnostics. However, there are still persistent gaps in care today, particularly for WGPPM and those with ultra-rare BDs, that, if addressed, could dramatically improve lives. NBDF fully understands the potential associated with bridging these gaps and is committed to taking the lead in a community-driven journey, convening stakeholders to discuss and create actionable changes to mitigate challenges and barriers to care for the WGPPM population.