Project Description

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.

Read our findings here….(Are acute therapies more affordable)