title: Comparative effectiveness research using matching-adjusted indirect comparison: an application to treatment with guanfacine extended release or atomoxetine in children with attention-deficit/hyperactivity disorder and comorbid oppositional defiant disorder.
abstract: OBJECTIVE
  To illustrate a matching-adjusted indirect comparison by comparing the efficacy of guanfacine extended release (GXR) and atomoxetine (ATX) in reducing oppositional symptoms in children with attention-deficit/hyperactivity disorder and comorbid oppositional defiant disorder.
  
  
  METHODS
  Individual patient data were used from a GXR trial; only published summary data were used from ATX trials. In a matching-adjusted indirect comparison, individual patients from the GXR trial were weighted such that their mean baseline characteristics matched those published for ATX trials. Placebo-arm outcomes were then compared to further assess balance between the matched populations. Changes in the Conners' Parent Rating Scale-Revised Short Form Oppositional Subscale from baseline to endpoint among GXR-treated and ATX-treated patients were then compared.
  
  
  RESULTS
  Before matching, the GXR (n = 143) and ATX (n = 98) trial populations had significant differences in baseline characteristics and placebo-arm outcomes. After matching, baseline characteristics were well balanced across trials, and placebo-arm outcomes became nearly identical. Comparing active treatment arms across the matched populations, GXR was associated with a significantly greater reduction in mean Conners' Parent Rating Scale-Revised Short form oppositional subscale compared with ATX {-5.0 [95% confidence interval (CI): -6.6 to -3.4] vs. -2.4 [CI: -3.7 to -1.1], p = 0.01, effect size = 0.58}.
  
  
  CONCLUSIONS
  In the absence of head-to-head randomized trials, matching-adjusted indirect comparisons can provide timely and reliable comparative evidence for decision makers and can be applied even when very few trials are available for the treatments of interest.
authors.affiliation: Analysis Group, Inc., Boston, MA, United States. jsignorovitch@analysisgroup.com
authors.forename: James
authors.initials: J
authors.lastname: Signorovitch
authors.forename: M Haim
authors.initials: MH
authors.lastname: Erder
authors.forename: Jipan
authors.initials: J
authors.lastname: Xie
authors.forename: Vanja
authors.initials: V
authors.lastname: Sikirica
authors.forename: Mei
authors.initials: M
authors.lastname: Lu
authors.forename: Paul S
authors.initials: PS
authors.lastname: Hodgkins
authors.forename: Eric Q
authors.initials: EQ
authors.lastname: Wu
mesh_terms: D000293:Adolescent; D000069445:Atomoxetine Hydrochloride; D001289:Attention Deficit Disorder with Hyperactivity / Q000150:complications / Q000188:drug therapy / Q000453:epidemiology; D019958:Attention Deficit and Disruptive Behavior Disorders / Q000150:complications / Q000188:drug therapy* / Q000453:epidemiology; D002648:Child; D057186:Comparative Effectiveness Research; D003692:Delayed-Action Preparations; D016316:Guanfacine / Q000008:administration & dosage / Q000627:therapeutic use*; D006801:Humans; D010043:Outcome and Process Assessment, Health Care / Q000706:statistics & numerical data; D017891:Pharmacoepidemiology / Q000379:methods* / Q000706:statistics & numerical data; D011437:Propylamines / Q000008:administration & dosage / Q000627:therapeutic use*; D016032:Randomized Controlled Trials as Topic / Q000592:standards* / Q000706:statistics & numerical data
chemical_list: D003692:Delayed-Action Preparations; D011437:Propylamines; D016316:Guanfacine; D000069445:Atomoxetine Hydrochloride