Case Study – Rare-Disease: ODC Implemented a Three-Pillar Strategy to Unlock LATAM for a Global Ultra-Rare Kidney Disease Trial
Challenge The sponsor needed to recruit patients with C3G and IC-MPGN — two ultra-rare complement-mediated kidney diseases — for the Phase III VALIANT trial evaluating pegcetacoplan. With an estimated incidence of 0.2–3 cases per million people and diagnosis requiring specialized renal biopsy with electron microscopy, identifying eligible patients in LATAM represented a major operational and clinical challenge.
Strategy
- Omnichannel Physician Engagement: Presented at national nephrology congresses, disseminated via SBN mailing lists and WhatsApp networks, and launched a national rare disease registry with COMDORA.
- Centralized Nephropathology Partnership: Partnered with Instituto de Nefropatologia (Brazil) — led by Dr. Stanley Araújo — conducting a nationwide retrospective review of renal biopsy archives and EMRs, identifying 230+ potential patients across Brazil and Argentina.
- Senior Medical Advisor Network: Engaged Dr. Lilian Palma (Pediatric Nephrologist) and Dr. Stanley Araújo to activate investigator networks, support physician education, and build referral pathways across both countries.
Results & Why This Matters
- ~20% of global VALIANT enrollment came from LATAM.
- Brazil ranked #2 recruiting country worldwide, behind only the United States.
- 230+ potential patients identified retrospectively across Brazil and Argentina and received a correct diagnosis.
VALIANT Trial Details The New England Journal of Medicine – Original Article Trial of Pegcetacoplan in C3 Glomerulopathy and Immune-Complex MPGN F. Fakhouri, A.S. Bomback, et al. for the VALIANT Trial Investigators Group* Pegcetacoplan was FDA-approved in 2025 for pediatric (first-ever) & adult patients with C3G/IC-MPGN.
