The ABCs of RCTs

The main thrust of Dr. Allen’s talk was that HTE is real and widely prevalent. Thus, researchers must be creative in accounting for this heterogeneity.

Multiphase Optimization Strategy

Sandra H. Soto, PhD, MPH, BSN

The second speaker was Sandra Soto, PhD, MPH, BSN, assistant professor in the School of Nursing and Thurston Arthritis Research Center at the University of North Carolina at Chapel Hill. Dr. Soto began by describing the classical approach for developing interventions, in which randomized clinical trials test multiple intervention components simultaneously in one package and investigators piece together their best intervention based on evidence from existing literature, theory and experimentation.

Dr. Soto provided the example of modifying an existing program meant to enhance physical activity among patients with arthritis. Using the classical approach, researchers may choose to add multiple interventions, such as use of pedometers, encouragement through social networking, phone calls from community health workers and creation of an educational video series. Ultimately, these interventions may be found to be effective, but how can a researcher know which components were most or least helpful? For this, the Multiphase Optimization Strategy (MOST) comes in handy.

The first step in MOST is preparation, which refers to laying the groundwork for optimization by deriving a conceptual model, selecting candidate intervention components, conducting pilot work and identifying optimization objectives. The next step is optimization, during which such approaches as factorial design can allow for individual intervention components to be turned on or off, such as only giving pedometers or using calls from community health workers for some, but not all, participants, and comparing the main effects of each component, as well as understanding interactions between components. The third step is to ask: Is the optimized intervention expected to be sufficiently effective? This can be evaluated by looking at P values, effect size, cost, time or other measures of relevance.

The final phase is evaluation, in which researchers can confirm the effectiveness of the optimized intervention. MOST is meant to allow researchers to be methodical in how they assess and optimize interventions, thereby being able to separate the wheat from the chaff.

More & more, clinicians have a growing number of possible treatments available to choose from, but lack evidence to guide those decisions.

Sequential Steps

Laura Schanberg, MD, MACR

The session’s final speaker was Laura Schanberg, MD, MACR, professor of pediatrics, Division of Pediatric Rheumatology, Duke University School of Medicine, Durham, N.C. Dr. Schanberg described how clinicians routinely make a series of treatment decisions with patients over the course of their disease with the goal of achieving the most beneficial expected outcome. Along the way are key decision points, milestones in the disease process, events that prompt the need for specific decisions and several feasible treatment options at each decision point.