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Compliance-adjusted intervention effects in survival data

Author

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  • Lois G. Kim

    (MRC Biostatistics Unit, Cambridge)

  • Ian R. White

    (MRC Biostatistics Unit, Cambridge)

Abstract

Time-to-event endpoints are a common outcome of interest in randomised clinical trials. The primary analysis should usually be by intention-to-treat, giving an indication of the effectiveness of the intervention in a population as a whole. However, the benefit specifically for an individual receiving the intervention is becoming increasingly important as patient decisions become more evidence-based. Effectiveness is defined as the benefit of intervention as actually applied, and may be estimated from simple all-or-nothing compliance data. Efficacy, on the other hand, is the benefit of intervention under ideal circumstances, and requires more complex compliance data. Intervention effectiveness and efficacy after accounting for non-compliance can be estimated in various ways, some of which have already been implemented in Stata (e.g. Author-Email: strbee). Recently, Loeys and Goetghebeur (2003) provided new methodology using proportional-hazards techniques in survival data where compliance is all-or-nothing in the intervention arm and perfect in the control arm. Here, their method is implemented in Stata. The output is a hazard ratio for the effectiveness of intervention, adjusted for observed adherence to intervention in the treated group. An example application is discussed for a subset of a large, randomised trial of screening where the average benefit of 26% risk reduction becomes a 34% risk reduction for individuals attending screening.

Suggested Citation

  • Lois G. Kim & Ian R. White, 2004. "Compliance-adjusted intervention effects in survival data," United Kingdom Stata Users' Group Meetings 2004 15, Stata Users Group.
    • Handle: RePEc:boc:usug04:15
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    References listed on IDEAS

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    1. T. Loeys & E. Goetghebeur, 2003. "A Causal Proportional Hazards Estimator for the Effect of Treatment Actually Received in a Randomized Trial with All-or-Nothing Compliance," Biometrics, The International Biometric Society, vol. 59(1), pages 100-105, March.
    2. Ian R. White & Sarah Walker & Abdel Babiker, 2002. "strbee: Randomization-based efficacy estimator," Stata Journal, StataCorp LP, vol. 2(2), pages 140-150, May.
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