Expanding the evidence base in transplantation: The complementary roles of randomized controlled trials and outcomes research

Steven K. Takemoto, Wolfgang Arns, Suphamai Bunnapradist, Louis P. Garrison, Lluis Guirado, Zoltan Kalo, Gabriel Oniscu, Gerhard Opelz, Maria Piera Scolari, Sergio Stefoni, Magdi Yaqoob, Daniel Brennan

Research output: Contribution to journalArticle

Abstract

Transplantation offers a unique opportunity to demonstrate the complementary roles of randomized controlled trials and outcome research. The surgery and collaboration necessary for the transplant procedure makes randomization and blinding difficult. Because essentially every recipient is included in a transplant registry, sampling bias is minimized. Regulatory agencies generally do not consider outcomes research when assessing efficacy of new drugs or medical interventions. This workgroup summary examines the suitability of outcomes research to complement results of randomized controlled trials and related issues: efficacy versus effectiveness, internal versus external validity, data types, limitations, and analysis methodologies. Many advances in outcomes research have been pioneered in transplantation. A case is made for regulatory and reimbursement authorities to use outcomes research when making efficacy, effectiveness, and coverage decisions in transplantation.

Original languageEnglish (US)
Pages (from-to)18-25
Number of pages8
JournalTransplantation
Volume86
Issue number1
DOIs
Publication statusPublished - Jul 15 2008
Externally publishedYes

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