Intellectual property and rare diseases : Addressing market failures through strategic frameworks

Rare diseases, also known as “orphan diseases”, collectively affect millions worldwide but remain overlooked in research and funding due to their low prevalence. Treatments for rare conditions have historically received less attention from biomedical innovation researchers and intellectual property scholars, largely due to systemic economic barriers such as small patient populations, high research and development costs, and limited commercial incentives. These challenges often suggest that intellectual property plays a minimal role in addressing the needs of rare disease treatments. However, this paper challenges that assumption by demonstrating the critical role of intellectual property in incentivising viable innovations and fostering sustainable advancements in this field. Using Australia’s National Strategic Action Plan for Rare Diseases as a case study, this paper examines how intellectual property can be effectively leveraged within public-private partnerships to address the market failures associated with the research and development of drugs for rare conditions. Drawing on successful strategies from initiatives like the Drugs for Neglected Diseases initiative (DNDi) and the Medicines for Malaria Venture (MMV), the paper highlights how tailored intellectual property frameworks can drive sustainable innovation while enhancing access to rare disease therapies.

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