China halts rare disease trials after deaths, scrutiny — SkimNews
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- Juan Huang founded the Hope for Rare Foundation and helped advance genetic treatment trials in China, aiming to launch two new studies before regulatory changes halted them in 2024.
- Chinese hospitals developed advanced capabilities in diagnosing and treating rare diseases over the past decade, enabling more sophisticated clinical research and lowering treatment costs compared to the U.S.
- Investigator-initiated trials (IIT) in China previously allowed researchers to launch clinical studies without central regulatory approval, accelerating human testing and drawing interest from U.S. biotech firms.
- The Chinese government implemented stricter clinical trial rules in 2024, citing patient deaths and negative press, effectively pausing new investigator-led studies despite prior momentum in rare disease research.
Why it matters: Rare disease patients and researchers lose access to fast-tracked therapies as China’s crackdown adds regulatory delays, reversing a decade of progress that had made the country a low-cost alternative to U.S. trials. The shift raises the bar for trial initiation but slows innovation for untreatable conditions.
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