Estimation of treatment effects and model diagnostics with two-way time-varying treatment switching: an application to a head and neck study

Treatment switching frequently occurs in clinical trials due to ethical reasons. Intent-to-treat analysis without adjusting for switching yields biased and inefficient estimates of the treatment effects. In this paper, we propose a class of semiparametric semi-competing risks transition survival mod...

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Bibliographic Details
Published in:Lifetime data analysis 2020-10, Vol.26 (4), p.685-707
Main Authors: Chen, Qingxia, Zhang, Fan, Chen, Ming-Hui, Cong, Xiuyu Julie
Format: Article
Language:English
Subjects:
Algorithms
Clinical trials
Computer Simulation
Diagnostic software
Diagnostic systems
Economics
Estimates
Ethical standards
Finance
Head & neck cancer
Head and Neck Neoplasms
Health Sciences
Humans
Insurance
Likelihood Functions
Management
Mathematics and Statistics
Maximum likelihood estimates
Medical prognosis
Medicine
Methods
Operations Research/Decision Theory
Parameter estimation
Quality Control
Randomized Controlled Trials as Topic - methods
Reliability
Safety and Risk
Software
Statistics
Statistics for Business
Statistics for Life Sciences
Survival Analysis
Switching
Treatment Switching
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Summary:Treatment switching frequently occurs in clinical trials due to ethical reasons. Intent-to-treat analysis without adjusting for switching yields biased and inefficient estimates of the treatment effects. In this paper, we propose a class of semiparametric semi-competing risks transition survival models to accommodate two-way time-varying switching. Theoretical properties of the proposed method are examined. An efficient expectation–maximization algorithm is derived to obtain maximum likelihood estimates and model diagnostic tools. Existing software is used to implement the algorithm. Simulation studies are conducted to demonstrate the validity of the model. The proposed method is further applied to data from a clinical trial with patients having recurrent or metastatic squamous-cell carcinoma of head and neck.
ISSN:1380-7870
1572-9249
DOI:10.1007/s10985-020-09495-0