‘This is not a science problem anymore': Paths emerge for scaling up rare disease medicine
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.
A similar idea — and other ways to go faster — are now getting closer to reality, pushed forward by new technical, economic and regulatory approaches. The framework is being created by companies like Moderna, by scientists who developed custom therapies that have the potential to benefit a larger population, and by a mother who knows the pain when a medicine doesn’t arrive in time.
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