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Institute Report
The Prospects for Biosimilars of Orphan Drugs in Europe
Current Landscape and Challenges Ahead
Jul 21, 2020
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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.

Report Summary

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.

Key Findings


Of the total number of drugs with active orphan status, 39% are biologics, which are medicinal products derived from living organisms.

Current Orphan Designated Drugs Available in the EU by Type

  • This share has increased from 25% five years ago, driven by the effectiveness and specificity of biologic medicines coupled with supportive regulations for orphan medicines
  • The growing pipeline of orphan drugs – both small and large molecule – portends even greater stress on tightly managed drug budgets in the near future.


In Europe, 156 products are approved, registered, and commercially available, treating a variety of rare diseases with over a third available as biologics.

The Landscape of Orphan Drugs in Europe

  • Orphan drugs typically have 10–12 years of market exclusivity before generic or biosimilar competition can enter the market.
  • Forty-eight orphan drugs have already lost market exclusivity, including 14 biologics, and during the period 2020 through 2029, 34 additional biologic orphan drugs are expected to lose exclusivity.
  • The five orphan biologics losing exclusivity by 2024 contributed about €501 million in total spending across Europe in 2019 alone.


Commercial challenges for orphan biosimilar products include a limited market size, with the majority of orphan drugs reaching sales under €100 million per year.

Average Sales for Newly Approved Orphan Medicines in Europe, 2019

  • 80% of orphan drugs had sales of less than €100 million in 2019, with the average biologic sales in 2019 being significantly lower at €61 million.
  • In addition to a small market size, the cost of developing a biosimilar product is typically higher than the cost of developing a generic small molecule product.
  • Despite challenges in clinical development and commercialization, orphan drugs continue to assume a greater role in healthcare, and biosimilars offer a means of reducing costs and increasing access supporting the long-term stability and sustainability of health systems.
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