Professor LJ Wei holds that rules are for lawyers, not (necessarily) clinicians. When designing modern clinical trials, the impetus is often to use “efficient and reliable procedures, to obtain clinically interpretable results with respect to risk-benefit analysis…” Yet these efficient and reliable procedures are often just conventions and rules that provide information that is incomplete or difficult to make clinically interpretable.
In a presentation to the East User Group Meeting, Professor Wei identifies 11 problematic areas that currently challenge trial designers. After giving an overview of the challenges that arise in each, Professor Wei provides a few simple solutions about how to overcome them. All the solutions, however, require moving beyond the comfort zone of conventional procedures.
In the slides attached Wei discusses:
(1) General methodological issues about the clinical usefulness of certain statistical concepts
(2) How to determine a primary endpoint in a multiple outcomes trial
(3) How to handle dropouts & competing risks
(4) Problems with analysis of covariance
(5) Improvements to data monitoring practices
(6) Complications with stratified medicine
(7) Predictive Enrichment (i.e. identifying groups of patients who will benefit from new therapies)
(8) Monitoring safety
(9) Quantifying treatment contrast
(10) Post-marketing safety studies
(11) Meta-Analysis for safety studies
The solutions which Wei provides draw on a number of recent case studies and ground-breaking work in trial design. He also discusses how we might train future biostatisticians to take more innovative and unconventional approaches, without sacrificing reliability.