Paula Greciano Calero, Silvia Escribá Bori, Juan Antonio Costa Orvay, Nina González Pons, María Del Carmen Martín Pérez, Dolores Cardona Alfonseca, Cristina Nogales Velázquez, Sergi Verd Vallespir, Alicia Esther Tur Salom, Antonella Chiandetti, Marcos Navarro Noguera, Anna Grau Blanch, María Magdalena Rotger Genestar, Marianna Mambié Meléndez, Mercedes Fernández Hidalgo, Juana María Seguí Llinas, Laura Martorell Bon, Patricia Arestuche Aguilar, Beatriz Garrido Conde, María Del Valle Sánchez Grao, Katia Sarraff Trujillo, Antoni Muntaner Alonso, Catalina Grimalt Ferragut, Andrea Soriano Marco, Viviana Gómez Rojas, Juan Pol Serra
Can we screen for heart disease in children at public health centres? A multicentre observational study of screening for heart disease with a risk of sudden death in children
European journal of pediatrics
multistate models used decades analyze economics expensive longlasting treatments recently also served address questions clinical drug development seems timely introduce broader pharmacometrics community technical aspects problemsolving capabilities models minimal model introduced answer questions interest drug developers regulatory agencies patients carers payers clinical study simulated 1000 patients randomly allocated 11 placebo active treatment recruitment phase deaths counted administrative data cutoff occurs 858 days first patient randomized minimal model one initial state two transient states two absorbing states fully parameterized semimarkov processes govern unidirectional transitions states simulations explore influence parameter uncertainty sample size validity statistical inferences questions interest stakeholders addressed predominantly graphic displays programming codes made available drug developers regulators invited reevaluate methods currently use assess benefits risks new treatments