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Rare disease families find roadmap to drug development at bootcamps

CNBC · May 7, 2026, 10:48 AM · Also reported by 4 other sources

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  • Developing a new drug can cost a billion dollars and take more than a decade.
  • That's a big reason why 95% of the more than 10,000 rare diseases that exist do not have an FDA-approved treatment.

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Developing a new drug can cost a billion dollars and take more than a decade. That makes investing in new treatments in the rare disease space — where patient populations are small, and the chance of earning a return on that investment even smaller — a risky bet for big pharmaceutical companies.

That's a big reason why 95% of the more than 10,000 rare diseases that exist do not have an FDA-approved treatment.

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