Orphan biosimilars are biologic medicines that are similar to original biologics that have orphan designations, meaning they are intended to treat rare diseases or conditions². They offer an opportunity to reduce the high costs of biologic treatments and improve patient access to essential therapies²⁴. However, they also pose some challenges for regulators, such as the need to conduct comparative analytical and clinical testing, which can be difficult in rare disease patient populations²,³. Additionally, there are commercialization challenges for orphan drug biosimilars due to their limited potential market size and the complexity of reimbursement policies²,³. Therefore, orphan biosimilars are both an opportunity and a challenge for regulators, depending on the specific product and the regulatory framework.

(1) The Prospects for Biosimilars of Orphan Drugs in Europe. https://www.iqvia.com/insights/the-iqvia-institute/reports/the-prospects-for-biosimilars-of-orphan-drugs-in-europe.

(2) Orphan Biosimilars: an Untapped Opportunity? - The Center For Biosimilars. https://www.centerforbiosimilars.com/view/orphan-biosimilars-an-untapped-opportunity.

(3) The opportunities and challenges of biosimilar orphans. https://www.tandfonline.com/doi/full/10.1517/21678707.2016.1171142.