Alternatives to Randomized Experiments

ABSTRACT— Randomized experiments are preferred for making inferences about causality when they can be implemented and their assumptions are met. Yet assumptions can fail (e.g., attrition, treatment noncompliance) or randomization may be unethical or infeasible. I describe alternative design and statistical approaches that permit testing causal hypotheses and present current empirical evidence related to alternative designs. Alternative designs permit a wider range of research questions to be answered and permit more direct generalization of causal effects; however, when using such designs, estimates of the magnitude of the causal effect may be more uncertain.     

Effects of Participant’s Choice of Different Digital Interventions on Outcomes for Binge-Spectrum Eating Disorders: A Pilot Doubly Randomized Preference Trial

It is unclear whether offering individuals a choice between different digital intervention programs affects treatment outcomes. To generate initial insights, we conducted a pilot doubly randomized preference trial to test whether offering individuals with binge-spectrum eating disorder a choice between two digital interventions is causally linked with superior outcomes than random assignment to these interventions. Participants with recurrent binge eating were randomized to either a choice (n = 77) or no-choice (n = 78) group. Those in the choice group could choose one of the two digital programs, while those in the no-choice group were assigned a program at random. The two digital interventions (a broad and a focused program) took 4 weeks to complete, were based on cognitive-behavioral principles and have demonstrated comparable efficacy, but differ in scope, content, and targeted change mechanisms. Most participants (79%) allocated to the choice condition chose the broad program. While both groups experienced improvements in primary (Eating Disorder Examination Questionnaire global scores and number of binge eating episodes over the past month) and secondary outcomes (dietary restraint, body image concerns, etc.), no significant between-group differences were observed. The two groups did not differ on dropout rates, nor on most indices of intervention engagement. Findings provide preliminary insights towards the role of client preferences in digital mental health interventions for eating disorders. Client preferences may not determine outcomes when digital interventions are based on similar underlying principles, although larger trials are needed to confirm this.     

Process Analyses of Impulsive Behavior in Binge-Eating Disorder During the Randomized Controlled IMPULS Trial

Impulsivity represents a risk factor for patients with binge-eating disorder, and we therefore investigated the treatment process of impulsive behaviors including binge-eating episodes in the randomized controlled IMPULS trial. Using 8 weekly online questionnaires throughout the assessment period, we compared 41 patients participating in the IMPULS program, which emphasized impulsive eating behavior (IG), with 39 control patients who received no intervention (CG). We assessed the frequency of binge eating, other impulsive behaviors, situations in which such behaviors could be inhibited, and the execution of alternative behaviors. Results indicate a stronger binge-eating reduction in the IG compared to the CG at the fifth, seventh, and eighth treatment weeks. Overall, both groups reduced other impulsive behaviors. They did not differ in the amount of inhibited impulsive behaviors and showed similar alternative behaviors, “distraction” most frequently used. IG patients evaluated the IMPULS program as very helpful. The stronger reduction of binge eating in the IG and positive evaluation of the treatment indicate a specific treatment effect regarding impulsive eating behavior. The reduction of other impulsive behaviors across both groups, and the initial reduction of binge eating within the CG, could be explained by an increased degree of self-observation.     

A Randomized Community-Based Trial of Behavior Therapy vs. Usual Care for Adolescent ADHD: Secondary Outcomes and Effects on Comorbidity

Though behavior therapy (BT) for ADHD in adolescence is evidence-based, almost no work examines its implementation and effectiveness in community settings. A recent randomized community-based trial of an evidence-based BT for adolescent ADHD (Supporting Teens’ Autonomy Daily; STAND; N = 278) reported high clinician, parent, and youth acceptability but variable implementation fidelity. Primary outcome analyses suggested no significant differences between STAND and usual care (UC) unless the clinician delivering STAND was licensed. The present study reports secondary outcomes for this trial on indices of comorbidity (anxiety, depression, oppositional defiant disorder, conduct disorder) and ADHD outcomes not targeted by the active treatment (social skills, sluggish cognitive tempo). We also examine whether therapist licensure moderated treatment effects (as in primary outcome analyses). Using intent-to-treat and per protocol linear mixed models, patients randomized to STAND were compared to those randomized to UC over approximately 10 months of follow-up. Group × Time effects revealed that, overall, STAND did not outperform usual care when implemented by community clinicians. However, a Group × Time × Licensure interaction revealed a significant effect on conduct problems when STAND was delivered by licensed clinicians (d = .19–.47). When delivered in community settings, behavior therapy for adolescent ADHD can outperform UC with respect to conduct problems reduction. Community mental health clinics should consider: (1) assigning adolescent ADHD cases to licensed professionals to maximize impact and (2) choosing psychosocial approaches when ADHD presents with comorbid conduct problems. There is also a need to reduce implementation barriers for unlicensed clinicians in community settings.     

Randomized controlled trial of a prenatal focused coparenting consultation for unmarried black fathers and mothers: One-year infant and family outcomes

In the infant mental health field, scant conceptual attention has been given to coparenting and family adaptations of non-white family systems, with no evidence-based, community-informed coparenting interventions responsive to unmarried Black mothers’ and fathers’ life circumstances. This study examined 1-year post-partum child and family outcomes of a novel, modest dosage (six sessions) prenatal focused coparenting consultation (FCC) using randomized controlled trial methodology. One-hundred-thirty-eight expectant families (one or both parents identified as Black/African American) were randomized to an intervention (N = 70) or treatment-as-usual (TAU; control) condition (N = 68). TAU families received navigational support in accessing existing community services for pregnant families. Intervention families received TAU plus 6 dyadic FCC sessions led by a Black male-female Community Mentor team. When infants were three and 12 months old, parents reported on coparenting, father engagement, interparental aggression, depressive symptoms, and infant social and emotional adjustment. Intent-to-treat analyses focusing on 12-month post-partum data indicated significant intervention effects on coparenting, interparental psychological aggression, and infants’ emotional adjustment. Improvement was also seen in depression and father engagement, with gains for both groups. Results suggest FCC delivered by same-race Community Mentors to unmarried Black coparents transitioning to parenthood supports infant and family adaptation during the first year of life.     

Intensive Child-Centered Play Therapy for Children on the Autism Spectrum: A Pilot Study

To investigate the effectiveness of intensive child-centered play therapy (CCPT) for children diagnosed with autism spectrum disorder (ASD), the authors randomly assigned 23 children with diagnosed ASD and symptoms of ASD on the Social Responsiveness Scale–2nd Edition (Constantino, 2012), to an intensive CCPT condition (n = 12) or a no-intervention control group (n = 11). Children who participated in 24 sessions of CCPT showed a statistically significant decrease in ASD core symptoms and behavioral symptoms, such as externalizing problems, attention problems, and aggression, compared with children in the control group. Implications are discussed.     

症状性颈动脉狭窄CEA治疗时机的Meta分析

评估症状性颈动脉狭窄（SCAS）患者颈动脉内膜剥脱术（CEA）手术时机选择及伴发症状对预后的影响.通过相关检索策略查找探讨CEA手术时机选择对于患者预后影响的随机对照试验.所有数据均采用Revman5.0分析软件进行Meta分析.纳入22篇文献,总计9 037名患者.Meta分析结果显示：SCAS患者4w、2w、1w 前后行CEA术后卒中再发率或死亡率组间无统计学差异.合并脑卒中的患者4w、2w 前后及合并短暂性脑缺血发作（TIA）或心肌梗死（MI）的患者4w前后行CEA术后卒中再发率或死亡率无统计学差异.症状稳定的SCAS患者应尽早行CEA术,伴发症状脑卒中、TIA 和MI不影响手术预后.     

Selecting the Right Tool For the Job

There are competing ethical concerns when it comes to designing any clinical research study. Clinical trials of possible treatments for Ebola virus are no exception. If anything, the competing ethical concerns are exacerbated in trying to find answers to a deadly, rapidly spreading, infectious disease. The primary goal of current research is to identify experimental therapies that can cure Ebola or cure it with reasonable probability in infected individuals. Pursuit of that goal must be methodologically sound, practical and consistent with prevailing norms governing human subjects research. Some maintain that only randomized controlled trials (RCTs) with a placebo or standard-of-care arm can meet these conditions. We maintain that there are alternative trial designs that can do so as well and that sometimes these are preferable to RCTs.