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Re: end2war post# 351410

Wednesday, 02/16/2022 5:00:41 PM

Wednesday, February 16, 2022 5:00:41 PM

Post# of 465622
end2war - for those of us who do not fully understand the math, I felt Dr. Missling at the Rett Ph3 Trial Result conference, gave a nice analogy using a psoriasis trial as example. I am paraphrasing and someone from this board can correct me. But I think Missling was trying to compare RSBQ Total Score versus RSBQ AUC. Missling stated that RSBQ AUC is a higher bar than RSBQ. RSBQ AUC is way to show that the drug is actually helping the patient. RSBQ can show an improvement, but may not really be helping the patient.

Missling's example - a patient has psoriasis covering the arm. The drug is provided to the patient. Under RSBQ the patient could show an improve such as 20% (as and example) which might be consider to some as significantly improved. In other words the patients coverage of psoriasis on the arm improved 20%. But the patient still has 80% psoriasis on the arm.

Under RSBQ AUC, that 20% improvement may show as a significant signal in a trial but you are not really helping the patient. So the 20% would not cut it as success. Missling went on to explain in his psoriasis example; under RSBQ AUC, the patient would have to show 75% removal of the psoriasis to count this as a responder. So he the latter part of the example relates to RSBQ AUC. A much higher bar.

Please let me know if I am misspeaking here. But that is what I believe Missling was trying to state during the Rett Phase 3 Trial Results conference.

Thanks _IGB
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