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Generalizability of findings from randomized controlled trials: application to the National Institute of Drug Abuse Clinical Trials Network.

ABSTRACT: To compare randomized controlled trial (RCT) sample treatment effects with the population effects of substance use disorder (SUD) treatment.Statistical weighting was used to re-compute the effects from 10 RCTs such that the participants in the trials had characteristics that resembled those of patients in the target populations.Multi-site RCTs and usual SUD treatment settings in the United States.A total of 3592 patients in 10 RCTs and 1?602?226 patients from usual SUD treatment settings between 2001 and 2009.Three outcomes of SUD treatment were examined: retention, urine toxicology and abstinence. We weighted the RCT sample treatment effects using propensity scores representing the conditional probability of participating in RCTs.Weighting the samples changed the significance of estimated sample treatment effects. Most commonly, positive effects of trials became statistically non-significant after weighting (three trials for retention and urine toxicology and one trial for abstinence); also, non-significant effects became significantly positive (one trial for abstinence) and significantly negative effects became non-significant (two trials for abstinence). There was suggestive evidence of treatment effect heterogeneity in subgroups that are under- or over-represented in the trials, some of which were consistent with the differences in average treatment effects between weighted and unweighted results.The findings of randomized controlled trials (RCTs) for substance use disorder treatment do not appear to be directly generalizable to target populations when the RCT samples do not reflect adequately the target populations and there is treatment effect heterogeneity across patient subgroups.

SUBMITTER: Susukida R

PROVIDER: S-EPMC5461185 | biostudies-literature | 2017 Jul

REPOSITORIES: biostudies-literature

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Generalizability of findings from randomized controlled trials: application to the National Institute of Drug Abuse Clinical Trials Network.

Susukida Ryoko R Crum Rosa M RM Ebnesajjad Cyrus C Stuart Elizabeth A EA Mojtabai Ramin R

Addiction (Abingdon, England) 20170316 7

<h4>Aims</h4>To compare randomized controlled trial (RCT) sample treatment effects with the population effects of substance use disorder (SUD) treatment.<h4>Design</h4>Statistical weighting was used to re-compute the effects from 10 RCTs such that the participants in the trials had characteristics that resembled those of patients in the target populations.<h4>Settings</h4>Multi-site RCTs and usual SUD treatment settings in the United States.<h4>Participants</h4>A total of 3592 patients in 10 RCT ...[more]

PMID: 28191694

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Project description:BackgroundAdolescent and young adult (AYA) patients with cancer have not seen the same improvements in survival as younger (pediatric) patients and older patients (adults 40 years old or older). This may be related to their lower participation in clinical trials.MethodsThis study examined AYA patient accrual to SWOG Cancer Research Network phase 1 to 3 clinical treatment trials for 29 cancers over 25 years (January 1996 to December 2020). Trial enrollments for AYA patients (15-39 years old) were compared with trial enrollments for older patients (40 years old or older) in SWOG and with US AYA cancer population rates derived from US Census and National Cancer Institute/Surveillance, Epidemiology, and End Results data.ResultsIn total, 84,219 patients were enrolled in SWOG treatment trials, including 7109 AYA patients (8.4%); in contrast, AYAs constituted 3.8% of the US cancer population. By histology, the highest proportions of AYA patients were in trials for Hodgkin disease (825 of 1220; 67.6%) and acute lymphocytic leukemia (350 of 678; 51.6%), whereas breast cancer trials had the greatest number of AYA patients (3032 of 32,693; 9.3%). SWOG AYA patients were more often female (68.8% vs 58.7%; P < .001), Black (10.1% vs 8.2%; P < .001), and Hispanic (10.6% vs 5.6%; P < .001) than SWOG patients who were 40 years old or older, and they were more often female (68.8% vs 65.1%; P < .001) but less often Black (10.1% vs 11.8%; P < .001) or Hispanic (10.6% vs 12.8%; P < .001) than AYA patients in the US cancer population.ConclusionsAYA patients with cancer were well represented in SWOG clinical trials in comparison with US cancer population patients with the same cancers. The SWOG AYA population was more racially/ethnically diverse than older SWOG patients, although it was less diverse than the US AYA cancer population.Lay summaryAdolescent and young adult (AYA) patients with cancer (aged 15-39 years) have not seen the same improvements in survival as younger (pediatric) patients and older patients (adults 40 years old or older). This may be related to their lower participation in clinical trials. This study evaluated the extent to which AYA patients were enrolled in a large, National Cancer Institute-sponsored network group over 25 years (1996-2020). Overall, 8.4% of the enrolled patients (7109 of 84,219) were AYAs; this was twice the corresponding rate of 3.8% in the US cancer population. AYA patients were also more racially/ethnically diverse than older trial patients, although they were less racially/ethnically diverse than the US AYA cancer population.

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Generalizability of findings from randomized controlled trials: application to the National Institute of Drug Abuse Clinical Trials Network.

Publications

Generalizability of findings from randomized controlled trials: application to the National Institute of Drug Abuse Clinical Trials Network.

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