Access to innovative treatment for paroxysmal nocturnal hemoglobinuria in low- and middle-income countries

Abstract Background: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, chronic, and life-threatening blood disorder characterized by uncontrolled complement activation and hemolysis. Without treatment, patients face severe complications including thrombosis, fatigue, organ damage, and early mortality. In low- and middle-income countries (LMICs), access to complement inhibitors remains virtually nonexistent. In response, The Max Foundation, in collaboration with the International PNH Interest Group (IPIG), has launched the Humanitarian PACT for PNH to increase access to PNH treatment globally. The Humanitarian PACT aims to promote equitable access to treatment and care for PNH through a collaboration model supporting local providers with strategic implementation planning and targeted investments in clinical capacity and health systems strengthening. Other members of the Humanitarian PACT for PNH include hematologists treating PNH patients in targeted countries, and Novartis who will make iptacopan, a first-in-class oral complement factor B inhibitor, available to The Max Foundation for eligible patients in select LMICs. Methods: The implementation phase of the Humanitarian PACT for PNH focused on readiness planning to ensure safe and effective delivery of iptacopan in LMIC settings. Key activities include: 1) stakeholder engagement with in-country healthcare professionals (HCP), 2) mapping diagnostic capacity for PNH confirmation, including flow cytometry, 3) assessing and strengthening clinical site readiness, including access to required vaccinations and provision of clinical training, 4) developing country-specific implementation plans and importation pathways, and 5) establishing extensive patient support strategies to ensure adherence and quality of care. Results: From January to June 2025, implementation planning was underway in 26 countries to identify feasibility and timeline of program launch by country. Site assessments were conducted in 4 institutions from 3 countries, highlighting readiness and key areas for capacity building, including number of hematologists, availability and accessibility of PNH diagnostic tests, vaccines, and antibiotic prophylaxis. Clinical training led by IPIG, covering diagnosis, pathology, clinical decision-making, medication management, and disease monitoring were provided to 2 partner HCPs from 1 country. Logistics planning included forecasting demand, development of program-specific inventory and supply guidelines, and validation of importation pathway and requirements. Due to the specialized nature of the disease and treatment, strategies focused on extensive patient support were established, including a program-specific accompaniment plan, integration of supportive programs for transportation, education, and adherence, and medical training for the Max team. Conclusions: The introduction of iptacopan through the Humanitarian PACT represents a critical step toward addressing inequities in access to PNH treatment in LMICs. Strategic implementation planning enables country partners to integrate novel therapies into existing health systems while building capacity in diagnostics and clinical management. By addressing both access and system readiness, this model demonstrates how equitable access to innovative medicines may serve as a foundation for strengthening rare disease care in resource-limited settings.