Tutorial- Monday at 1:45 PM EDT
Title: Statistical Advancement in the Area of Rare Disease Development - SMART Opportunities in Rare Disease Research
Presenters: Kelley Kidwell, University of Michigan

Abstract:
Sequential, multiple assignment, randomized trial (SMART) designs are often motivated to identify tailored sequences of treatments or dynamic treatment regimens (DTRs) in larger samples. SMARTs employ at least two randomizations in sequence where only some groups may be re-randomized based on response or other characteristics related to previous treatment. More recently, instead of focusing on DTRs, the SMART design has been applied to the setting of rare diseases to obtain more information from a small sample of individuals. This talk will provide an overview of small sample SMART (snSMART) designs and primarily Bayesian methods for analyses. The differences between snSMART and SMART designs will be highlighted and methods to analyze snSMART data, calculate sample size, and add adaptive components will be presented.

Bio:
Kelley Kidwell is associate professor of Biostatistics at the University of Michigan School of Public Health and is an expert in large and small sample sequential, multiple assignment, randomized trial (SMART) design and analysis. She is the primary investigator of current FDA and PCORI contracts, also had a previous PCORI methods contract, all related to SMART design, and has been a co-investigator on many NIH and industry funded grants, of which 7 currently funded NIH grants implement SMART design.