Scientific advances have transformed the outlook for many people living with rare diseases, yet access to orphan medicines remains highly unequal around the world. For patients in many low- and middle-income countries (LMICs), effective treatments remain unavailable, not simply because of affordability, but because of wider systemic barriers affecting diagnosis, regulation, financing and healthcare delivery.

This report explores why these inequalities persist and examines what governments, industry, international organisations and patient groups can do to improve access. Rather than focusing on pricing alone, it takes a whole-system perspective, mapping the barriers that exist across both patient and product pathways and identifying practical solutions capable of supporting sustainable access over the long term. 

The paper highlights how limited diagnostic infrastructure, fragmented regulatory systems, constrained healthcare capacity and weak incentives for market entry combine to restrict access to orphan medicines in many countries. It also showcases examples of initiatives already making a difference while recognising that lasting progress will require coordinated action across governments, healthcare systems, industry and global health organisations. 

Published against the backdrop of growing international momentum on rare diseases, including the adoption of the WHA Rare Disease Resolution, the report provides a timely framework for understanding one of the most pressing global health equity challenges facing the rare disease community.

Whether you work in global health, market access, policy or rare disease strategy, this paper offers practical insights into how sustainable access can be expanded beyond high-income countries.

Download the full report to explore the barriers, opportunities and policy solutions that could help improve access to orphan medicines for patients in low- and middle-income countries.