‘This is not a science problem anymore': Paths emerge for scaling up rare disease medicine
- N1C
- Oct 4, 2023
- 1 min read
Updated: Apr 30, 2024
In 2014, the National Institutes of Health posted an estimate that at the current pace of drug development and approval, it would take 1,000 years to get treatments for the 7,000 known rare diseases.
A paper at the time suggested speeding up that work by grouping together similar diseases, and then treating these patients with the same therapy in a single clinical trial.
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