Learning : Short Courses

 
 


Adaptive Randomization Strategies for Phase III Studies

Scott S. Emerson, MD, PhD

March, 2010

Abstract: Recently much attention has been devoted to the re-design of ongoing randomized clinical trials based on interim results. One such area of interest is the use of interim trial results to determine the randomization of future subjects. In this short course we will review such methods as “covariate adaptive” randomization, “play-the-winner” randomization schemes, and adaptively dropping treatment arms or subgroups. We will focus on the ability of these methods to better address the efficiency and ethical issues inherent in randomized clinical trials. We will also discuss the special considerations that must be made at the time of study design, during conduct of the clinical trial, and when reporting results, and how those special considerations might impinge on gaining regulatory approval for a new drug, biologic, or device.

Course Materials (pdf):   Presentation Handouts

Video Slideshows:  

  1. •1. Overview:

  2. Scientific and clinical setting

  3. (Slides 1-26) (26:11)


  1. •2. Reporting Inference:

  2. Target of inference; role of precision

  3. (Slides 27-43) (11:54)


  1. •3. Phases of Investigation:

  2. Phase I, II, III clinical trials

  3. (Slides 44-58) (11:41)


  1. •4. Statistical Hypotheses:

  2. Summary measures, controls

  3. (Slides 59-81) (10:55)


  1. •5. Statistical Role of Variables:

  2. Review of confounding, precision,

  3. variance inflation, effect modification

  4. (Slides 82-110) (19:35)


  1. •6. Common Statistical Models:

  2. Linear, logistic, Poisson, proportional hazards regression

  3. (Slides 111-128) (08:37)


  1. •7. Important Questions of Statistical Modeling:

  2. Unadjusted vs adjusted analyses

  3. (Slides 129-172) (37:59)


  1. •8. Randomization:

  2. (Slides 173-189) (16:33)

  1. •9. Analytic Models:

  2. Population model; Randomization model

  3. (Slides 190-197) (34:02)


  1. •10. Complete Randomization:

  2. (Slides 198-220) (15:51)


  1. •11. Stratified Randomization:

  2. Blocking; Stratification on covariates

  3. (Slides 221-235) (15:30 )


  1. •12. Covariate Adaptive Allocation:

  2. (Slides 236-247) (23:45)


  1. •13. Adaptive Randomization Ratios:

  2. (Slides 248-255) (04:05)


Learning Material:


  1. •Short Courses

  2. •Research Talks

  3. •Case Studies