OBJECTIVES : The number of orphan drugs expanding into new orphan indications after first EMA approval has been increasing in Europe throughout the last decade. This study examines the implications of indication expansion for orphan designated drugs on existing prices across EU5 markets as a function of clinical value and level of unmet need.

METHODS : Qualitative in-depth interviews (n=14) with key stakeholders within EU5 markets were conducted, complemented with secondary research of different biomedical and price databases, to gather insights into the impact of expanding onto new orphan labels for products with previously established reimbursed prices.

RESULTS : The likelihood of sustaining list prices is high for orphan drugs in European markets after expanding into a new orphan indication with high unmet need. In France, likelihood of potential list price cut is highest, as demonstrated by eculizumab (2-7%, 2014-17). Net prices are likely to decrease in all countries, with discounts from 5% to 10% depending on patients’ volume increase, perceived additional clinical value and level of unmet need observed in the new indication. Price drops can also vary across countries depending on market archetype. In budget conscious markets, accurately defining the new target population can lead to price maintenance if the drug provides a clear clinical benefit and the manufacturer is able to demonstrate a high unmet need. In HTA-driven and cost-effectiveness markets, price erosion is directly linked to whether the drug fulfils the country-specific HTA requirements or is considered to be cost-effective, respectively.

CONCLUSIONS : Orphan drugs can manage to retain list prices in most EU5 markets after label expansion. However, net price discounts are generally re-negotiated to achieve reimbursement for a new indication depending on the additional clinical benefit and the level of unmet need.