OBJECTIVE: This article aims to calculate the impact of orphan drugs on the Belgian drug budget in 2008 and to forecast its impact over the following 5 years. METHOD: The 2008 budget impact was calculated by triangulating information derived from multiple Belgian data sources. The 2008-2013 budget impact analysis was based on three scenarios reflecting different levels of growth in the number of registered orphan drugs in the European Union, the number of drugs reimbursed in Belgium, and the average annual cost per patient per drug in Belgium. RESULTS: The orphan drug budget impact amounted to €66.2 million (or 5% of the Belgian hospital drug budget) in 2008. The impact would increase to €130-204 million in 2013, depending on the scenario. CONCLUSIONS: This static analysis measured orphan drug costs only, assuming that other components of health expenditure do not change over time. The analysis showed that the budget impact of orphan drugs in Belgium is substantial and rising, thereby putting pressure on total drug expenditure. Policy options to address the rising budget impact include pricing linked to return on investment, risk-sharing arrangements and re-appraisal of orphan drug status if additional indications are approved.
OBJECTIVE: This article aims to calculate the impact of orphan drugs on the Belgian drug budget in 2008 and to forecast its impact over the following 5 years. METHOD: The 2008 budget impact was calculated by triangulating information derived from multiple Belgian data sources. The 2008-2013 budget impact analysis was based on three scenarios reflecting different levels of growth in the number of registered orphan drugs in the European Union, the number of drugs reimbursed in Belgium, and the average annual cost per patient per drug in Belgium. RESULTS: The orphan drug budget impact amounted to €66.2 million (or 5% of the Belgian hospital drug budget) in 2008. The impact would increase to €130-204 million in 2013, depending on the scenario. CONCLUSIONS: This static analysis measured orphan drug costs only, assuming that other components of health expenditure do not change over time. The analysis showed that the budget impact of orphan drugs in Belgium is substantial and rising, thereby putting pressure on total drug expenditure. Policy options to address the rising budget impact include pricing linked to return on investment, risk-sharing arrangements and re-appraisal of orphan drug status if additional indications are approved.