A likelihoodist trial procedure

TL;DR

This paper introduces a new likelihood-based sequential testing procedure for medical treatment comparisons, offering improved evidence assessment and early stopping capabilities, validated through simulations on real-world data.

Contribution

It presents a novel likelihood ratio-based trial method that addresses limitations of traditional null hypothesis testing in treatment comparisons.

Findings

Misleading evidence rate was 5%

Early stopping was frequent and misleading evidence occurred in only 0.4% of cases

Procedure performed well on real-world treatment effect data

Abstract

A simple and common type of medical research involves the comparison of one treatment against another. The logical aim should be both to establish which treatment is superior and the strength of evidence supporting this conclusion, a task for which null hypothesis significance testing is particularly ill-suited. This paper describes and evaluates a novel sequential inferential procedure based on the likelihood evidential paradigm with the likelihood ratio as its salient statistic. The real-world performance of the procedure as applied to the distribution of treatment effects seen in the Cochrane Database of Systematic Reviews is simulated. The misleading evidence rate was 5% and mostly this evidence was only weakly misleading. Early stopping occurred frequently and was associated with misleading evidence in only 0.4% of cases.