Rare DIEM Virtual Summit 2025: Latin America’s Rare-Disease Clinical Trials Advantage

By Elmira Safarova, PhD | Founder & CEO, Rarus Health

Setting the Stage — Only 5.4 % of Rare-Disease Clinical Trials Happen in Latin America. That Must Change.

Last week I had the honor of speaking at the Rare DIEM (Diseases In Emerging Markets) Virtual Summit 2025 by Farmacon Global, where researchers, patient advocates, regulators, and industry leaders came together to confront a stubborn paradox:

• Latin America (LATAM) possesses every ingredient for high-impact clinical research—diverse genetics, centralized public-health systems, motivated investigators, and recruitment costs up to 40 % lower than in the US or EU.
• Yet barely one in twenty clinical trials takes place in the region, and even fewer focus on orphan and ultra-rare diseases.

Our panel—“Data Sovereignty & AI in LATAM Clinical Trials: Protecting Patients and Institutions”—looked at how we can unlock that potential without repeating the extractive models of the past. Below are my main take-aways and the concrete steps Rarus Health is taking to accelerate the shift.

1. LATAM’s Competitive Edge Is Real—and Urgent

Advantage	What It Means for Sponsors
Cost efficiency	Up to 40 % lower per-patient cost while meeting ICH-GCP standards.
Fast site activation	Countries such as Mexico and Chile routinely green-light trials in <120 days, shaving months off time-to-market.
Rapid recruitment	Treatment-naïve populations and national health registries make it easier to identify eligible patients.
High-value therapeutic gaps	Neuromuscular and neurodevelopmental disorders remain under-represented, offering “blue-ocean” space for sponsors willing to innovate.

Yet these advantages stall when regulatory fragmentation, limited natural-history data, and a lingering trust gap keep sponsors on the sidelines.

2. Data Sovereignty + AI: From Buzzwords to Blueprint

During the panel we agreed on three principles:

1. Federated data modelsKeep data where it is generated; move only encrypted insights for analysis. This respects national sovereignty and avoids mass data exports that erode trust.
2. AI-driven patient matchingModern machine-learning models can scan electronic records and patient-reported outcomes to identify trial-ready cohorts in weeks rather than months—if we have structured, consented data to feed them.
3. Recruitment-enabling educationFamilies are willing partners when they understand how their data translates to faster diagnoses, trials, and therapies. Education must be built into every protocol, not bolted on later.

“Trust emerges when patients see their data as a bridge to better care—not an asset extracted from them.” —My closing line at the summit

3. What the Ecosystem Still Lacks

• Regional harmonization. Separate ethics boards and import rules force sponsors to run country-by-country gauntlets.
• Longitudinal access. Advocacy groups feel betrayed when a study drug never makes it to local formularies. Compassionate-use and early-access programs must be planned up-front.
• Shared infrastructure. Only a handful of high-income metros have the biobanks, genetic testing, or telehealth reach needed for rare-disease trials.

4. How Rarus Health Is Closing the Gaps

Challenge	Rarus Health Solution	Impact
Fragmented patient data	Project REALES—a patient-driven neuromuscular registry that standardizes clinical, genomic, and real-world data across LATAM in Spanish.	Provides the structured, consented datasets AI needs for rapid cohort discovery.
Trust & transparency	Dynamic, GDPR-style consent delivered via web app, plus plain-language dashboards so families see how their data is used.	Converts participants into long-term partners and ambassadors.
Natural-history evidence	Longitudinal follow-up modules co-designed with caregivers and clinicians.	Generates endpoints and PROs that regulators recognize, de-risking trial design.
Recruitment bottlenecks	AI-enabled matching engine that sits atop our registry and hospital EHR integrations.	Sponsors receive pre-qualified cohorts within a single IRB framework.
Education gap	Webinars, micro-courses, and local ambassador programs (e.g., Moebius Chile, Club Pompe International, Dravet LATAM Foundation).	Builds “recruitment-enabling education” into community culture and speeds enrollment.

Call to Action—Let’s Build the Next 1,000 Rare-Disease Trials in LATAM

If you are:

• A pharma or biotech seeking faster, more diverse recruitment
• A CRO looking to streamline multi-country operations
• A patient organization ready to turn lived experience into scientific power

…then I invite you to partner with us.

Book a meeting with me to explore how we can accelerate your next rare-disease study

Together we can transform Latin America from a 5 % footnote into a global powerhouse for ethical, patient-centered innovation—while bringing life-changing therapies closer to the people who need them most.

Con corazón y ciencia,

Elmira Safarova, PhD