Challenges faced by biosimilar orphan drugs in European health systems

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As treatments for rare diseases become a greater healthcare priority, the contribution of biosimilars as a means of reducing costs and increasing patient access will become more important for the long-term sustainability of health systems [1]. While the current impact of non-orphan biosimilars suggests this contribution could be significant, uptake of biosimilars across Europe remains variable and there is a risk that sustainable competitive markets are not yet established. A recent review of biosimilar policies in Europe found that key principles for a sustainable biosimilar market include supporting innovation, physician prescribing freedom, and allowing for multiple suppliers [2].

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Developers of orphan drug biosimilars in Europe face a number of clinical development and commercialization challenges due to the current landscape for biosimilars and the special characteristics of orphan drugs. The implications for European health systems are discussed in this article.

Targeted financial incentives may be required to attract multiple biosimilar manufacturers to invest in the development of orphan drug biosimilars. One possible incentive is priority review vouchers issued by the European Medicines Agency that can be sold to another company based on their value. This would accelerate the commercialization process of a drug with a large potential market. Financial incentives may also be employed by payers and providers, alongside awareness building, to encourage investigators and patients to participate in biosimilar clinical trials.

Educational outreach that gives all stakeholders confidence in biosimilar versions of orphan drugs is crucial to driving uptake. Patient advocacy groups could play an important role in championing biosimilars as an essential means to extend patient access and free up resources for investment in innovative treatments.

Procurement processes – particularly tender design and execution – may require a different approach for orphan drug biosimilars due to their potentially very small volumes, the use of very few distribution centres and the potentially very high costs. Procurement mechanisms that allow clinicians to select biosimilars of orphan drugs, while maintaining their prescribing freedom, may be required to overcome long-term loyalties to originator products. Such mechanisms might include sharing savings from biosimilar use with the prescriber’s centre.

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Clinical development challenges faced by orphan drug biosimilar developers

The landscape for orphan drugs in Europe

1. GaBI Online - Generics and Biosimilars Initiative. Country scorecards show biosimilar sustainability []. Mol, Belgium: Pro Pharma Communications International; [cited 2020 Oct 30]. Available from:  
2. GaBI Online - Generics and Biosimilars Initiative. Sustainable biosimilar policies in Europe []. Mol, Belgium: Pro Pharma Communications International; [cited 2020 Oct 30]. Available from: 

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