Why Orphan Medicines Remain Out of Reach in Low- and Middle-income Countries – and What Can Be Done
Elisa Garau, Becky Neil, Katarina Kitarovic, Elena Nicod
February 2026
Rare diseases are a major global health challenge. More than 7,000 conditions affect an estimated 300 million people worldwide, though definitions vary (in the EU, a disease is rare if it affects fewer than 5 in 10,000 people, and related therapies are designated as orphan medicines). 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 report analyses 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.
