Significant concerns discussed in the report re. clinical value of Trofinetide, clinical trial methodology, endpoints chosen, side effect burden and cost to the taxpayer in socially funded health care system.
I found the most concerning line to be:
"A price reduction of more than 98% would be
required for trofinetide plus BSC to be considered cost-effective at a willingness-to-pay threshold of $100,000 per QALY gained."
A jargon heavy public health way to say based on the panel's economic analysis, the cost of the drug isn't justified by the assessed clinical benefit.
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neuren pharmaceuticals limited
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