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Cost effective assay choice for rare disease study designs

  • Desmond D. Campbell*
  • , Robert M. Porsch
  • , Stacey S. Cherny
  • , Valeria Capra
  • , Elisa Merello
  • , Patrizia De Marco
  • , Pak C. Sham
  • , Maria Mercê Garcia-Barceló
  • *Corresponding author for this work
  • The University of Hong Kong
  • IRCCS Istituto Giannina Gaslini - Genova

Research output: Contribution to journalArticlepeer-review

2 Scopus citations

Abstract

High throughput assays tend to be expensive per subject. Often studies are limited not so much by the number of subjects available as by assay costs, making assay choice a critical issue. We have developed a framework for assay choice that maximises the number of true disease causing mechanisms 'seen', given limited resources. Although straightforward, some of the ramifications of our methodology run counter to received wisdom on study design. We illustrate our methodology with examples, and have built a website allowing calculation of quantities of interest to those designing rare disease studies.

Original languageEnglish
Article number13023
JournalOrphanet Journal of Rare Diseases
Volume10
Issue number1
DOIs
StatePublished - 2015
Externally publishedYes

Funding

FundersFunder number
University of Hong Kong
European Commission
Research Grants Council, University Grants Committee201209176012, 201010159012, HKU 7666/11 M
Seventh Framework ProgrammeHEALTH-F2-2010-241909, 241909

    Keywords

    • High-throughput assay
    • Rare disease
    • Study design
    • WES
    • WGS

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