EXPRESS: Rare Diseases Are Not Rare: Challenges and Opportunities for OM Research

There are more than 7,000 known rare diseases, yet around 95% of them lack effective treatments. This paper explores how Operations Management (OM) research can help improve patient access to rare disease treatments. We first examine key challenges and opportunities from the perspectives of governments, industry, insurers, and patients. We then outline future research directions, focusing on regulatory frameworks, subsidy and incentive schemes, pricing and coverage decisions, (bio)pharmaceutical manufacturing, and challenges in developing economies. The application of OM methodologies to the rare disease landscape is still limited, but offers significant potential to advance new drug development, increase patient access to correct diagnosis and treatment, and reduce healthcare inequalities for patients with rare diseases.