Abstract
BackgroundTo examine the value of a Sequential Multiple Assignment Randomized Trial (SMART) design compared to a conventional randomized control trial (RCT) for telemedicine strategies to support titration of insulin therapy for Type 2 Diabetes Mellitus (T2DM) patients new to insulin.MethodsMicrosimulation models were created in R using a synthetic sample based on primary data from 63 subjects enrolled in a pilot study of a smartphone application (App), Diabetes Pal compared to a nurse-based telemedicine strategy (Nurse). For comparability, the SMART and an RCT design were constructed to allow comparison of four (embedded) adaptive interventions (AIs).ResultsIn the base case scenario, the SMART has similar overall mean expected HbA1c and cost per subject compared with RCT, for sample size of n = 100 over 10,000 simulations. SMART has lower (better) standard deviations of the mean expected HbA1c per AI, and higher efficiency of choosing the correct AI across various sample sizes. The differences between SMART and RCT become apparent as sample size decreases. For both trial designs, the threshold value at which a subject was deemed to have been responsive at an intermediate point in the trial had an optimal choice (i.e., the sensitivity curve had a U-shape). SMART design dominates the RCT, in the overall mean HbA1c (lower value) when the threshold value is close to optimal.ConclusionsSMART is suited to evaluating the efficacy of different sequences of treatment options, in addition to the advantage of providing information on optimal treatment sequences.
Highlights
A major objective of clinical trials, randomized controlled trials (RCTs) is to identify which of two or more therapies is most effective
Full list of author information is available at the end of the article
(a) SingHealth Polyclinics’ Insulin Initiation Telecare Program (Nurse): The program was designed to support insulin initiation for patients with Type 2 Diabetes Mellitus (T2DM) at Operationalization of Competing Trial Designs To compare a traditional RCT and Sequential Multiple Assignment Randomized Trial (SMART) for evaluation of effectiveness and cost in the trial context, we implemented a microsimulation of these two trial designs run over a 12-week “study” period
Summary
A major objective of clinical trials, randomized controlled trials (RCTs) is to identify which of two or more therapies is most effective. When a treatment that works for most people based on an RCT is not effective for a particular patient, in clinical. Clinical trials in which individuals are randomized to sequences of treatment strategies are seldom used [1]. An alternative to an idiosyncratic series of choices are decision rules such as those embodied in guidelines developed by medical professional organizations: a combination of expert opinion, behavioral, psychosocial and biological theories, and observational studies to formulate adaptive treatment algorithms, or adaptive interventions (AIs) [2, 3]. To examine the value of a Sequential Multiple Assignment Randomized Trial (SMART) design compared to a conventional randomized control trial (RCT) for telemedicine strategies to support titration of insulin therapy for Type 2 Diabetes Mellitus (T2DM) patients new to insulin
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