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    Rights statement: © 2015 Hampson et al. This is an open access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited

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Elicitation of expert prior opinion: application to the MYPAN trial in childhood polyarteritis nodosa

Research output: Contribution to journalJournal article

Published
  • Michael W. Beresford
  • Claudia Bracaglia
  • Alfonso Caldas
  • Rolando Cimaz
  • Joke Dehoorne
  • Pavla Dolezalova
  • Mark Friswell
  • Marija Jelusic
  • Stephen D. Marks
  • Neil Martin
  • Anne-Marie McMahon
  • Joachim Peitz
  • Annet van Royen-Kirkhof
  • Oguz Soylemezoglu
  • Paul Brogan
  • MYPAN Vasculitis Expert Group
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Article numbere0120981
<mark>Journal publication date</mark>30/03/2015
<mark>Journal</mark>PLoS ONE
Issue number3
Volume10
Number of pages14
Publication statusPublished
Original languageEnglish

Abstract

Objectives

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).

Methods

A Bayesian prior elicitation meeting was convened. Opinion was sought on the probability that a patient in the MYPAN trial treated with cyclophosphamide would achieve disease remission within 6-months, and on the relative efficacies of mycophenolate mofetil and cyclophosphamide. Expert opinion was combined with previously unseen data from a recently completed randomised controlled trial in ANCA associated vasculitis.

Results

A pan-European group of fifteen experts participated in the elicitation meeting. Consensus expert prior opinion was that the most likely rates of disease remission within 6 months on cyclophosphamide or mycophenolate mofetil were 74% and 71%, respectively. This prior opinion will now be taken forward and will be modified to formulate a Bayesian posterior opinion once the MYPAN trial data from 40 patients randomised 1:1 to either CYC or MMF become available.

Conclusions

We suggest that the methodological template we propose could be applied to trial design for other rare diseases.

Bibliographic note

© 2015 Hampson et al. This is an open access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited