Definitive sample sizes for clinical trials in rare diseases are usually infeasible. Bayesian methodology can be used to maximise what is learnt from clinical trials in these circumstances. We elicited expert prior opinion for a future Bayesian randomised controlled trial for a rare inflammatory paediatric disease, polyarteritis nodosa (MYPAN, Mycophenolate mofetil for polyarteritis nodosa).
Elicitation of expert prior opinion: application to the MYPAN trial in childhood polyarteritis nodosa / Hampson, Lisa V; Whitehead, John; Eleftheriou, Despina; Tudur-Smith, Catrin; Jones, Rachel; Jayne, David; Hickey, Helen; Beresford, Michael W; Bracaglia, Claudia; Caldas, Afonso; Cimaz, Rolando; Dehoorne, Joke; Dolezalova, Pavla; Friswell, Mark; Jelusic, Marija; Marks, Stephen D; Martin, Neil; Mcmahon, Anne-Marie; Peitz, Joachim; van Royen-Kerkhof, Annet; Soylemezoglu, Oguz; Brogan, Paul A. - In: PLOS ONE. - ISSN 1932-6203. - ELETTRONICO. - 10:(2015), pp. 1-14. [10.1371/journal.pone.0120981]
Elicitation of expert prior opinion: application to the MYPAN trial in childhood polyarteritis nodosa
CIMAZ, ROLANDO;
2015
Abstract
Definitive sample sizes for clinical trials in rare diseases are usually infeasible. Bayesian methodology can be used to maximise what is learnt from clinical trials in these circumstances. We elicited expert prior opinion for a future Bayesian randomised controlled trial for a rare inflammatory paediatric disease, polyarteritis nodosa (MYPAN, Mycophenolate mofetil for polyarteritis nodosa).
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elicitation of 2015.pdf
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