MMRM vs. LOCF: A Comprehensive Comparison Based on Simulation Study and 25 NDA Datasets

Siddiqui, Ohidul; Hung, H. M. James; O'Neill, Robert

doi:10.1080/10543400802609797

Cited by 301Statistical Methods and Bayesian InferenceStatistical Methods in Clinical TrialsPharmaceutical studies and practices

MMRM vs. LOCF: A Comprehensive Comparison Based on Simulation Study and 25 NDA Datasets

Siddiqui, Ohidul, Hung, H. M. James, O'Neill, Robert·2009·Journal of Biopharmaceutical Statistics

DOI 10.1080/10543400802609797

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Abstract

In recent years, the use of the last observation carried forward (LOCF) approach in imputing missing data in clinical trials has been greatly criticized, and several likelihood-based modeling approaches are proposed to analyze such incomplete data. One of the proposed likelihood-based methods is the Mixed-Effect Model Repeated Measure (MMRM) model. To compare the performance of LOCF and MMRM approaches in analyzing incomplete data, two extensive simulation studies are conducted, and the empirical bias and Type I error rates associated with estimators and tests of treatment effects under three missing data paradigms are evaluated. The simulation studies demonstrate that LOCF analysis can lead to substantial biases in estimators of treatment effects and can greatly inflate Type I error rates of the statistical tests, whereas MMRM analysis on the available data leads to estimators with comparatively small bias, and controls Type I error rates at a nominal level in the presence of missing completely at random (MCAR) or missing at random (MAR) and some possibility of missing not at random (MNAR) data. In a sensitivity analysis of 48 clinical trial datasets obtained from 25 New Drug Applications (NDA) submissions of neurological and psychiatric drug products, MMRM analysis appears to be a superior approach in controlling Type I error rates and minimizing biases, as compared to LOCF ANCOVA analysis. In the exploratory analyses of the datasets, no clear evidence of the presence of MNAR missingness is found.

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Missing data were managed using LMM, which is based on all observed data and accounts for data missing at random.
“Missing data were managed using LMM, which is based on all observed data and accounts for data missing at random [108-110].”
From Development and Evaluation of a Smartphone-Based Chatbot Coach to Facilitate a Balanced Lifestyle in Individuals With Headaches (BalanceUP App): Randomized Controlled Trial (2024) · cites this paper as [109]

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Authors: Siddiqui, O., Hung, H. M. J., & O'Neill, R.
Affiliations: United States Food and Drug Administration (US)
a Office of Biostatistics, Office of Translational Sciences, Center for Drug Evaluation and Research, Food and Drug Administration , Silver Spring, Maryland, USA
Corresponding author: Siddiqui, Ohidul
ORCID: Hung, H. M. James
Venue: Journal of Biopharmaceutical Statistics
Publisher: Informa UK Limited
Published: 2009-02-12
Volume: 19
Issue: 2
Pages: 227-246
ISSN: 1054-3406 (ISSN-L)
1520-5711
DOI: 10.1080/10543400802609797
OpenAlex: W2129152730
Cited by: 301
References: 32
Citations by year: 2026: 5 · 2025: 8 · 2024: 7 · 2023: 15 · 2022: 10 · 2021: 12 · 2020: 18 · 2019: 14 · 2018: 16 · 2017: 23 · 2016: 22 · 2015: 25 · 2014: 32 · 2013: 31 · 2012: 20
FWCI: 17.15
Type: journal article
Language: en
Open access: closed
Locations: Journal of Biopharmaceutical Statistics (publishedVersion)
PubMed (publishedVersion)
Topics: Physical Sciences › Mathematics › Statistics and Probability › Statistical Methods and Bayesian Inference
Physical Sciences › Mathematics › Statistics and Probability › Statistical Methods in Clinical Trials
Health Sciences › Medicine › Pediatrics, Perinatology and Child Health › Pharmaceutical studies and practices
Keywords: last observation carried forward, missing data imputation, clinical trials, type I error rate, estimation bias, missing completely at random, missing at random, missing not at random, new drug applications, neurological drugs, psychiatric medication, covariance analysis
MeSH terms: Algorithms, Clinical Trials as Topic, Computer Simulation, Data Interpretation, Statistical, Humans, Longitudinal Studies, Psychotropic Drugs, Regression Analysis, United States, United States Food and Drug Administration, Reproducibility of Results, Models, Statistical, Bias, Likelihood Functions, Least-Squares Analysis, Databases, Factual

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MMRM vs. LOCF: A Comprehensive Comparison Based on Simulation Study and 25 NDA Datasets

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