Czabotar PE, Garcia-Saez AJ
Symptoms of high cortisol may include unexplained weight gain (especially around the abdomen), purple stretch marks, muscle weakness, and mood changes
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Others have specific frameworks for research chemicals that impose requirements around documentation, end-use certification, or import licensing
While Alnylams Phase III APOLLO-B study hit both key primary and secondary endpoints, the FDA stated in the documents that the effects of patisiran compared to placebo on both endpoints were small, of questionable clinical meaningfulness, and may not be detectable by patients. Major discussion points during the adcomm meeting included whether these key endpoints were appropriate to ascertain clinical meaningfulness in the ATTR-CM population, and panelists expressed varying opinions on whether the magnitude of the benefit seen in trial data was clinically meaningful
The key is to design coverage that promotes appropriate use while encouraging lifestyle improvements
