Overview of Trial Design Options: Adults (Challenges in Clinical Trials in People for Whom Available Therapies have Failed )

2/7/01


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Table of Contents

Overview of Trial Design Options: Adults (Challenges in Clinical Trials in People for Whom Available Therapies have Failed )

WARNING

Randomized Controls or Not?

Tuberculous Meningitis

Tuberculous Meningitis

PPT Slide

PPT Slide

Increasing Heterogeneity

Increasing Heterogeneity 2

Increasing Heterogeneity 3

Selected variables predicting decline of pVL < 400 (Multi-drug Rescue Therapy - Montaner et al)

PPT Slide

PPT Slide

PPT Slide

Can we avoid randomized trials?

Gastric Freezing for DU

Gastric Freezing for DU

VA Study - Estrogen & Prostate Ca

Uncontrolled Phase II Cancer studies

Traditional Orthodoxy

Role of Observational Data

Role of Observational Data

Non-randomized concurrent comparisons by post-randomization variables

Analysis by post-randomization adherence in a lipid lowering trial

Analysis by post-randomization adherence in a lipid lowering trial

Salvage Therapy and Non-randomized Controls

Salvage Therapy and Non-randomized Controls

Control of Confounders

Remedies for Confounding in Smaller Sample Sizes

There is no within-study remedy for lack of power in smaller sample size studies!

The Need for Blinding?

The Need for Blinding?

The Need for Blinding?

The Need for Blinding - An example

PPT Slide

PPT Slide

Particular Clinical Trial Challenges

Clinical Trial Challenges

Factorial Designs - Illustration

ACTG 359 (2 x 3 Factorial)

OPTIMA (2 x 2 Factorial)

Randomised trial of effects of vitamin supplements on pregnancy outcomes and T cell counts in HIV-1-infected women in Tanzania Fawzi WW et al. Lancet 1998; 351:1477-82.

Stratified Factorial

Factorial Designs

Latin Squares

Factorial Designs

Factorial Designs

Drug-wise vs. Strategy-wise Evaluation

Drug-wise Evaluation

Strategy-wise Evaluation 1

Strategy-wise Evaluation 2

Miscellaneous Considerations

Cost-Effectiveness Considerations

Some Comparators (US$ per QALY)

Author: liora gelbart