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Redefining Statistical Significance For Rare Disease Research

 
• By Christian Schneider

Biopharma Excellence recently convened three experts in the world of rare disease in a roundtable to discuss the emerging approaches to data collection when patient populations are very small. Their discussion has been summarized for In Vivo.    

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• Source: Shutterstock

A recent panel discussion hosted by Biopharma Excellence featured experts in biotech and the evolving regulatory environment to talk about the challenge of achieving statistical significance with novel and highly targeted therapies, especially for rare/ultra-rare diseases.

Daniel O’Connor of the UK’s MHRA noted that the challenge of achieving statistical significance when target populations are tiny is nothing new, but conceded that randomized trials aren’t always viable...

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