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Here is a good example of FDA guidance on the issue.[1]

FDA acknowledges that FVIII levels can serve as a proxy for clinical outcome (reduced bleeding episodes) in hemophilia A.

Our understanding of what is "normal" in FVIII blood levels is such that a single arm, surrogate biomarker study is sufficient for approval.

[1]https://www.fda.gov/downloads/BiologicsBloodVaccines/Guidanc...




Could you help by pointing out where they talk about not obtaining baseline/control data? After a quick glance, they only seem to mention using a within-subject design.


Sorry, misread your reply. Yes, they do an internal baseline/control, but don't have a control arm (either placebo or active comparator) in the trial.

That was the focus of the JAMA article.




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