1 March 2018 - Following yesterday’s international Rare Disease Day, here is a snapshot of four new rare disease treatments currently under review by England’s cost-effectiveness watchdog.

The last 20 years have seen a rapid increase in the number of drugs for rare diseases being developed and gaining European approval.

These new therapies can transform the lives of patients, because for many of these conditions there has never been any licensed treatment before. However the high cost and sparse health economic data for orphan and ultra-orphan medicines means patients who could benefit have often come up against barriers to access.

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