When the Rare Disease Diversity Coalition (RDDC) was first created, few could have imagined the global influence it would wield or the extent to which communities outside the United States would engage with its scientific data and observations of the lived experiences of rare disease patients from historically underserved populations. In Q4 of 2024, when I had the opportunity to speak on two continents outside of North America, to share the work RDDC had recently created I knew I had to say yes. I knew this would be a chance to amplify the coalition’s mission to address inequities in rare disease communities and to foster global collaboration. These speaking engagements would provide the platform to exemplify our growing international impact, to embark on a transformative journey across two different countries to share the coalition’s work and cultivate international partnerships.

A Voice at the IndoUSRare Summit in India

The trip began in New Delhi, India, where I was invited to speak on a distinguished panel at the IndoUSRare Summit. This summit brought together experts, patient advocates, and policymakers to address pressing challenges in the rare disease landscape. Thanks to our partnership with IndoUSRare, we were selected as one of the speakers for the Global Inclusion and Access to Orphan Drugs panel. It was an honor to engage as a panelist alongside esteemed representatives from Sanofi, International Rare Disease Research Consortium, Sickle Cell Thalassemia Patient Network, Global Alliance for Rare Disorders Access Access, and the National Insurance Corporation of India. 

I shared with the panel and the audience the US’s perspective on health equity as it impacts rare disease patients from historically marginalized populations, highlighting the RDDC’s groundbreaking efforts to improve access to diagnostics, treatments, and clinical trials for underserved populations. In addition, I presented some key insights from a recent literature review that will be sent to a publisher this year for consideration – the review focuses on the unique challenges faced by historically medically underrepresented populations who are inadequately represented in clinical research – and shared some strategies to improve the systemic inequities in our healthcare infrastructure. During the panel discussion, I learned about reimbursements and pricing for orphan drugs in India, and we discussed the barriers that rare disease patients face for access to therapeutics.

Beyond the summit, I engaged in extraordinary conversations with rare disease patients and advocacy organizations in both India and the US. These discussions revealed the shared struggles and unique cultural contexts shaping the rare disease experience in different regions. They underscore the global resonance of the RDDC’s work, as patients and advocates expressed deep interest in strategies to improve health outcomes, build trust, and address systemic inequities.

A European Stop in Brussels

The journey progressed to Brussels, Belgium, where I had the distinct privilege of delivering a presentation at the inaugural Clinical Trials in Rare Disease Conference in Europe, hosted by GlobalData. The event emphasized the complexities of conducting clinical trials for rare conditions, including the urgent need for diversity and inclusivity to ensure equitable outcomes.

In Brussels, I met with European-based patient advocacy organizations, who were captivated by the findings of the Inequities in Rare Disease Community Report, a groundbreaking resource created by RDDC in partnership with the National Organization for Rare Disorders (NORD). The report sheds light on critical disparities in rare disease diagnosis, care, and research access, particularly among historically underserved and marginalized communities. It emphasizes the urgent need to address social determinants of health, improve diagnostic equity, and promote cultural competence within healthcare systems.

During these conversations, European advocates expressed a keen interest in several key insights from the report, including the disproportionate diagnostic odyssey faced by patients of diverse racial and ethnic backgrounds, the role of systemic bias in healthcare delivery, and the lack of diversity in clinical trial participation. They were particularly inspired by RDDC’s actionable recommendations, such as fostering trust through community engagement, establishing tailored patient support programs, and advocating for policy reforms that prioritize equity in rare disease funding and resource allocation.

These discussions reinforced the universal relevance of the coalition’s work, as European advocates sought to learn from RDDC’s strategies and adapt them to their own regional contexts. The exchange emphasized the shared challenges and opportunities across borders, reinforcing the need for global collaboration to eliminate inequities in rare disease care and research. It was energizing to witness how the findings of the report resonated deeply with advocates striving to create more inclusive systems for rare disease patients worldwide.

A Global Vision for Rare Disease Advocacy

This journey across India and Europe marked a milestone in RDDC’s evolution from a national initiative to a global force for change. What began as a mission to address health disparities in the US has blossomed into a movement with worldwide implications. It was a profound privilege to share RDDC’s scientific data and lived-experience insights on a global stage, reflecting the coalition's growing relevance and influence.

The insights and partnerships cultivated during these trips have already sparked exciting new collaborations:

• European advocacy groups have expressed interest in co-developing frameworks for more inclusive clinical trials, incorporating considerations by RDDC’s actionable recommendations.
• Discussions with Indian patient networks are designing, tailored, culturally nuanced, support programs, bridging the gap between diagnosis and care for underserved populations.
• The coalition is now better equipped to integrate global best practices into our U.S.-based efforts, ensuring that the lessons learned abroad fuel progress at home.

This is only the beginning of RDDC’s global vision. Some of the coalition’s founders may not have envisioned this level of global engagement, but the journey to India and Europe proved that rare disease advocacy knows no borders—and neither does the RDDC’s commitment to driving meaningful change.The RDDC is proving that the fight for health equity in rare disease transcends borders—and that our investment in this mission is driving meaningful change worldwide.