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Orphan Drug Development: Could We Be Seeing The End Of P Values?

 
• By Michael Cipriano

Panel of experts stresses need to move toward innovative approaches for rare disease drug development such as Bayesian methods, but this would require a paradigm shift in US FDA's regulatory structure. 

Letter P
P values for orphan products might never come into focus if FDA moves away from reliance on randomized clinical trials.

Could the scientific community be nearing the end of the end of the P value as an efficacy marker in the rare disease drug development space?

It is certainly possible, a panel of experts says. But the key to shifting away from P values toward Bayesian...

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