title: Expanding the evidence base in transplantation: the complementary roles of randomized controlled trials and outcomes research.
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.
authors.affiliation: National Institute of Transplantation, S. Mark Taper Foundation Transplant Center, Los Angeles, CA 90057, USA. sktakemoto@hotmail.com
authors.forename: Steven K
authors.initials: SK
authors.lastname: Takemoto
authors.forename: Wolfgang
authors.initials: W
authors.lastname: Arns
authors.forename: Suphamai
authors.initials: S
authors.lastname: Bunnapradist
authors.forename: Louis P
authors.initials: LP
authors.lastname: Garrison
authors.forename: Lluis
authors.initials: L
authors.lastname: Guirado
authors.forename: Zoltan
authors.initials: Z
authors.lastname: Kalo
authors.forename: Gabriel
authors.initials: G
authors.lastname: Oniscu
authors.forename: Gerhard
authors.initials: G
authors.lastname: Opelz
authors.forename: Maria Piera
authors.initials: MP
authors.lastname: Scolari
authors.forename: Sergio
authors.initials: S
authors.lastname: Stefoni
authors.forename: Magdi
authors.initials: M
authors.lastname: Yaqoob
authors.forename: Daniel C
authors.initials: DC
authors.lastname: Brennan
mesh_terms: D015982:Bias; D019317:Evidence-Based Medicine; D006801:Humans; D019370:Observation; D016377:Organ Transplantation; D016032:Randomized Controlled Trials as Topic; D012042:Registries; D015203:Reproducibility of Results; D012107:Research Design; D016896:Treatment Outcome