OBJECTIVES: In 2019, we compared the costs of the most expensive gene/acute therapies and chronic treatments for rare diseases in the US. This is an updated analysis which compares the annual and lifetime treatment costs of the 10 most expensive drugs in the US with Germany.

METHODS: The most expensive drugs were identified from a literature review of PubMed and grey databases. The ex-factory prices and dosing regimens of these treatments were identified and analysed to calculate costs for the first year of treatment. Lifetime treatment costs were calculated by applying life expectancy data for each indication. A similar analysis was performed for Germany using the treatments identified from the US analysis.

RESULTS: The top 10 most expensive drugs in the first year are a combination of gene/acute therapies and chronic treatments, compared with 2019 where they were all gene/acute therapies. Zolgensma, Luxturna and Folotyn remain in the top 10, with the additional gene/acute therapy, Spinraza, since 2019. Zolgensma has the highest costs in the first year in the US and Germany, while Takhzyro accrues far higher lifetime costs in both countries (~$20m in the US compared with ~$2m for Zolgensma). When considering the lifetime costs of these products, the acute treatments are ranked #7-10 in the US. Based on the German ex-factory price, Zolgensma ranks as the most expensive gene/acute therapy on a lifetime basis at #4. Annual and lifetime costs of the top 10 most expensive drugs have increased since 2019 and are generally far higher in the US than in Germany.

CONCLUSIONS: Overall, the research suggests that gene/acute therapies may in fact be much more affordable than chronic treatments over a patient’s lifetime. Innovative assessment frameworks need to focus on the long-term game, to assist payer decision-making and mitigate concerns relating to high ‘one-off’ prices.