Biosimilars and Future Biotherapeutics

Biosimilars in Rare and Orphan Diseases

The emergence of biosimilars in rare and orphan diseases marks a critical turning point in the accessibility and affordability of advanced biologic therapies for small and underserved patient populations. Rare diseases, defined in many regions as conditions affecting a small percentage of the population, often lack effective treatments due to limited commercial incentives and high development costs. When therapies do exist—typically in the form of biologics such as monoclonal antibodies, enzyme replacement therapies, or fusion proteins—they are frequently priced at a premium, placing a substantial burden on healthcare systems and making access difficult for patients, particularly in low- and middle-income countries. The introduction of biosimilars offers a potential solution to this challenge by increasing treatment options and lowering costs, thereby expanding access to life-altering or life-saving therapies for patients with rare conditions. Despite the enormous potential, the development and adoption of biosimilars in the context of rare diseases face unique scientific, regulatory, and market-related challenges. From a scientific perspective, rare diseases often involve highly complex biologics and a limited understanding of disease mechanisms, which makes demonstrating biosimilarity more difficult.