Assessment of face validity of a disease model of nonsense mutation Duchenne muscular dystrophy: a multi-national Delphi panel study.
Erik LandfeldtRongrong ZhangAnne-Marie ChildsJessika JohannsenDeclan O'RourkeThomas SejersenJurgis StrautmanisUlrike Schara-SchmidtMar TuliniusMaggie C WalterTracey WillisKatharina BueschPublished in: Journal of medical economics (2022)
This study confirms the face validity of key clinical parameters and assumptions underlying the ataluren cost-effectiveness model.