About the Report
The landscape for biosimilars is rapidly changing. In Europe, many biologic products with orphan designations are reaching the end of their market exclusivity, allowing for the potential of affordable biosimilars to enter the market to the benefit of healthcare systems and patients alike. This report investigates the challenges biosimilar manufacturers are likely to encounter with clinical development and commercialization of orphan biosimilars as well as the implications of these products to European health systems.
The availability and access to treatments for rare diseases is of critical importance to European patients and healthcare systems. The introduction of biosimilars has the potential to result in savings, sustainably lower costs, and broader patient access to rare disease medicines. For example, five orphan biologics contributing approximately €501 million in total spending are losing market exclusivity by 2024. However, the clinical development of biosimilars brings different challenges for manufacturers than small molecule generics, such as the need to conduct comparative analytical and clinical testing, which can pose recruiting challenges in rare disease patient populations. Specifically, total development costs could run in the range of $40-50 million per orphan biosimilar. Similarly, there are a number of commercialization challenges for orphan drug biosimilars due to their limited potential market size. For example, only about 12% of the 42 currently designated biologic orphan products are likely to have a commercial market size across Europe in excess of €100 million per year, limiting commercial opportunity for biosimilars in this space. Despite limited market size and risks, as orphan drugs assume a greater role in healthcare, the contribution of orphan biosimilars will become more important for the long-term stability and sustainability of health systems.