Rare diseases, comprised of more than 7,000 conditions, are a major global health challenge, affecting over 300 million people worldwide.

In low- and middle-income countries (LMICs), the burden is substantial and likely underestimated due to underdiagnosis, weak surveillance, and limited access to early diagnosis and preventive care. Even where treatments exist, access to orphan medicines remains highly unequal, shaped by low political priority, regulatory and value assessment gaps, constrained funding, and limited health-system capacity.

This Dolon Institute report, supported by IFPMA, analyzes the barriers to accessing orphan medicines in LMICs and identifies practical, system-level solutions across patient and product pathways. It reviews current initiatives and argues that sustainable progress requires embedded approaches to strengthen diagnosis, regulatory routes, financing, and distribution. With new momentum from the WHO Rare Disease Resolution (May 2025) and the forthcoming 10-year Global Action Plan (2028–2038), it sets out a timely agenda for coordinated action to deliver equitable access.

This report was originally published on 27 February 2026 by Dolon Institute.