TY - JOUR
T1 - Advances in clinical trials methodology
T2 - Intervention optimization approaches in emergency medicine
AU - Bernstein, Steven L.
AU - Carter, Patrick M.
AU - Meurer, William
AU - Walton, Maureen A.
AU - Kidwell, Kelly M.
AU - Cunningham, Rebecca M.
AU - Dziura, James
AU - Collins, Linda M.
N1 - Funding Information:
Several of this report's co-authors (MW, PC, RC, KK) are developing an ED-based adaptive intervention to reduce risky drinking and violent behavior among adolescents. The study, called the SafERteens M-Coach study and funded by the National Institute on Alcohol Abuse and Alcoholism (R01AA024755), builds on prior work from our research group demonstrating that a selective 30-min brief in-person therapy intervention (SafERteens intervention) for alcohol and violence delivered during emergency care reduces severe peer violence, dating violence, and alcohol consequences [ 16-18 ]. A post hoc analysis identified that response and non-response at 3 months post-intervention depended on several factors, including greater problem severity (e.g., alcohol use, violence), delinquent behaviors, mental health concerns, and negative peer influences, suggesting that although a single brief in-ED intervention session may be sufficient for many youth, a subset of youth require a more intensive and longer-term intervention [ 19 ]. Pilot studies indicated that participants are amenable to extended interventions delivered via text messages and/or remote coaching [ 19 , 20 ]. These findings suggested that a productive direction would be development and optimization of a time-varying adaptive intervention.
Funding Information:
One of us (SLB) is currently conducting a study to optimize a fixed tobacco treatment regimen for adult smokers seen in the ED. The study protocol has been previously reported [9]. The study extends work previously done by our group and others demonstrating the efficacy of ED-initiated treatment for tobacco dependence [10,11], and is supported by the National Cancer Institute (R01CA201873). In this optimization trial, four intervention components are examined: (1) a Brief Negotiated Interview (BNI), which is a shortened version of a motivational interview; (2) 6 weeks of nicotine replacement therapy (NRT, nicotine patches and gum), with the first patch applied in the ED; (3) an active referral to the state smokers' quitline, made by the research assistant faxing a consultation form; and (4) enrollment in a 6-week texting program. Participants are contacted by telephone at 1 and 3 months. Participants who self-report abstinence from smoking at 3 months are asked to return to the ED for biochemical validation, via measurement of exhaled carbon monoxide. Study enrollment and follow-up are completed.Supported by grants R01CA201873, R01MD011516, K23DA039341, R01AA022931, P01CA180945, P50DA039838, R01DA040480, and R01AA024755 from the National Institutes of Health, and R49CE002099 from the Centers for Disease Control and Prevention.
Funding Information:
Supported by grants R01CA201873 , R01MD011516 , K23DA039341 , R01AA022931 , P01CA180945 , P50DA039838 , R01DA040480 , and R01AA024755 from the National Institutes of Health , and R49CE002099 from the Centers for Disease Control and Prevention.
Publisher Copyright:
© 2021 Elsevier Inc.
PY - 2022/3
Y1 - 2022/3
N2 - The classical two-arm randomized clinical trial (RCT) is designed to test the efficacy or effectiveness of an intervention, which may consist of one or more components. However, this approach does not enable the investigator to obtain information that is important in intervention development, such as which individual components of the intervention are efficacious, which are not and possibly should be removed, and whether any components interact. The Multiphase Optimization Strategy (MOST) is a new framework for development, optimization, and evaluation of interventions. MOST includes the RCT for purposes of evaluation, but inserts a phase of research before the RCT aimed at intervention optimization. The optimization phase requires one or more separate trials similar in scope to an RCT, but employing a different experimental design. The design of the optimization trial is selected strategically so as to maximize the amount of scientific information gained using the available resources. One consideration in selecting this experimental design is the type of intervention to be optimized. If a fixed intervention, i.e. one in which the same intervention content and intensity is provided to all participants, is to be optimized, a factorial experiment is often appropriate. If an adaptive intervention, i.e. one in which intervention content or intensity is varied in a principled manner, is to be optimized, a sequential multiple-assignment randomized trial (SMART) is often a good choice. The objective of this article is to describe MOST and the scientific rationale for its use; describe two current applications of MOST in emergency medicine research, one using a factorial experiment and the other using a SMART; and discuss funding strategies and potential future applications in studying the care of individuals with acute illness, injury, or behavioral disorders.
AB - The classical two-arm randomized clinical trial (RCT) is designed to test the efficacy or effectiveness of an intervention, which may consist of one or more components. However, this approach does not enable the investigator to obtain information that is important in intervention development, such as which individual components of the intervention are efficacious, which are not and possibly should be removed, and whether any components interact. The Multiphase Optimization Strategy (MOST) is a new framework for development, optimization, and evaluation of interventions. MOST includes the RCT for purposes of evaluation, but inserts a phase of research before the RCT aimed at intervention optimization. The optimization phase requires one or more separate trials similar in scope to an RCT, but employing a different experimental design. The design of the optimization trial is selected strategically so as to maximize the amount of scientific information gained using the available resources. One consideration in selecting this experimental design is the type of intervention to be optimized. If a fixed intervention, i.e. one in which the same intervention content and intensity is provided to all participants, is to be optimized, a factorial experiment is often appropriate. If an adaptive intervention, i.e. one in which intervention content or intensity is varied in a principled manner, is to be optimized, a sequential multiple-assignment randomized trial (SMART) is often a good choice. The objective of this article is to describe MOST and the scientific rationale for its use; describe two current applications of MOST in emergency medicine research, one using a factorial experiment and the other using a SMART; and discuss funding strategies and potential future applications in studying the care of individuals with acute illness, injury, or behavioral disorders.
KW - Clinical trials
KW - Research methodology
UR - http://www.scopus.com/inward/record.url?scp=85121786173&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85121786173&partnerID=8YFLogxK
U2 - 10.1016/j.ajem.2021.12.028
DO - 10.1016/j.ajem.2021.12.028
M3 - Article
C2 - 34968972
AN - SCOPUS:85121786173
SN - 0735-6757
VL - 53
SP - 6
EP - 11
JO - American Journal of Emergency Medicine
JF - American Journal of Emergency Medicine
ER -