In 2004, I was cured of Plaque Psoriasis that covered 95% of my body. The medication was Enbrel which was first approved in 1999 for arthritis. I found it curious that Missling used this condition as a trial example when explaining the anchored RSBQ. I recall my doctor telling me that patients responding to the medication saw plaque clearance rates of 75%, so clearly Amgen had established an endpoint that was a dramatic outcome for the patient. Though even a 50% clearance would have been clinically meaningful, $35,000 per year in cost would not have been worth the benefit, especially with moderately effective topicals available at a fraction of the cost.
I don’t quite understand the concerns I am reading regarding Trofinetide. We all know it is inferior in effectiveness and has some, well rather uncomfortable side effects for wheel chair bound patients. I wouldn’t be so certain that the fda approves their drug, in light of the fact it has no effect on adult patients, and used for a primary endpoint what we all now realize is an error prone RSBQ score comparison between 2 points in time. Caregiver bias is a given. Even if approved and on the market before Blarcamesine, if you were the caregiver of the patient, would you not switch to the more effective, fewer side effect option; and alternatively if you were a physician recommend your patient switch meds; and if you were an insurance company manager, steer payment to the better indication.
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