Estimating efficacy in a proposed randomized trial with initial and later non-compliance

Research output: Contribution to journalArticle

Abstract

A controversial topic in obstetrics is the effect of walking on the probability of Caesarean section among women in labour. A major reason for the controversy is the presence of non-compliance that complicates the estimation of efficacy, the effect of treatment received on outcome. The intent-to-treat method does not estimate efficacy, and estimates of efficacy that are based directly on treatment received may be biased because they are not protected by randomization. However, when non-compliance occurs immediately after randomization, the use of a potential outcomes model with reasonable assumptions has made it possible to estimate efficacy and still to retain the benefits of randomization to avoid selection bias. In this obstetrics application, non-compliance occurs initially and later in one arm. Consequently some parameters cannot be uniquely estimated without making strong assumptions. This difficulty is circumvented by a new study design involving an additional randomization group and a novel potential outcomes model (principal stratification).

Original languageEnglish (US)
Pages (from-to)211-221
Number of pages11
JournalJournal of the Royal Statistical Society. Series C: Applied Statistics
Volume56
Issue number2
DOIs
StatePublished - Mar 2007

Fingerprint

Noncompliance
Randomized Trial
Randomisation
Efficacy
Potential Outcomes
Principal Stratification
Estimate
Selection Bias
Biased
Immediately
Randomization
Randomized trial
Model
Obstetrics
Potential outcomes

Keywords

  • Causal inference
  • Potential outcomes
  • Principal stratification

ASJC Scopus subject areas

  • Mathematics(all)
  • Statistics and Probability

Cite this

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abstract = "A controversial topic in obstetrics is the effect of walking on the probability of Caesarean section among women in labour. A major reason for the controversy is the presence of non-compliance that complicates the estimation of efficacy, the effect of treatment received on outcome. The intent-to-treat method does not estimate efficacy, and estimates of efficacy that are based directly on treatment received may be biased because they are not protected by randomization. However, when non-compliance occurs immediately after randomization, the use of a potential outcomes model with reasonable assumptions has made it possible to estimate efficacy and still to retain the benefits of randomization to avoid selection bias. In this obstetrics application, non-compliance occurs initially and later in one arm. Consequently some parameters cannot be uniquely estimated without making strong assumptions. This difficulty is circumvented by a new study design involving an additional randomization group and a novel potential outcomes model (principal stratification).",
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