Top 20 most expensive drugs in US

ISPOR 2019 Are acute therapies and curative drugs more affordable than chronic treatments in rare diseases? An analysis of the top 20 most expensive drugs in the US


The cost of innovative drugs for rare diseases has continually pushed the upper pricing thresholds of payers in recent years.
Recent drug launches have included acute use gene-therapies and life-saving treatments, which promise a ‘cure’ or at a minimum, a life-altering impact.
Previous publications have assessed annual costs only and group together both gene/acute therapies and chronic treatments which may not provide a complete picture and places a focus on acute treatments being ‘the most expensive drugs in the World’.

This study investigates costs of gene/acute therapies versus chronic treatments for rare diseases, on an annual and lifetime basis to provide a more balanced comparison.

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