Rare Diseases and the WHO Model List of Essential Medicines - Report of the RDI Working Group

Access to essential medicines is fundamental to achieving Universal Health Coverage (UHC) and health equity, particularly for People Living With Rare Disease (PLWRD). Rare diseases (RDs) collectively affect over 300 million people worldwide, presenting unique challenges such as prolonged diagnostic journeys, limited treatment options, and high costs. Recognizing these challenges, Rare Diseases International (RDI) established the Essential Medicines Working Group (EMWG) in June 2024 to improve understanding of the World Health Organization Essential Medicines List (WHO EML) as a tool to enhance access to medicines for rare diseases. Composed of representatives from RDI member organizations and their members, the EMWG analyzed successful and unsuccessful applications for rare disease medicines to the EML, identifying challenges, success factors, and opportunity to strengthen the evaluation process for essential RD medicines. This report describes the results of the research carried out by the working group.