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TRIALS, ISSN 1745-6215, 11/2018, Volume 19
BackgroundA key step in the design of a RCT is the estimation of the number of participants needed in the study. The most common approach is to specify a... 
MEDICINE, RESEARCH & EXPERIMENTAL | CLINICALLY IMPORTANT DIFFERENCE | ESTIMANDS
Journal Article
BMJ-BRITISH MEDICAL JOURNAL, ISSN 1756-1833, 11/2018, Volume 363
Randomised controlled trials are considered to be the best method to assess comparative clinical efficacy and effectiveness, and can be a key source of data... 
MEDICINE, GENERAL & INTERNAL | CLINICALLY IMPORTANT DIFFERENCE | ESTIMANDS
Journal Article
Pharmaceutical Statistics, ISSN 1539-1604, 01/2017, Volume 16, Issue 1, pp. 29 - 36
Recent research has fostered new guidance on preventing and treating missing data. Consensus exists that clear objectives should be defined along with the... 
estimands | clinical trials | missing data | DEPRESSION | DULOXETINE | PREVENTION | MULTIPLE-IMPUTATION | PAROXETINE | PHARMACOLOGY & PHARMACY | STATISTICS & PROBABILITY | Data Interpretation, Statistical | Sample Size | Humans | Clinical Trials as Topic - methods | Clinical Trials as Topic - standards | Models, Statistical | Research Design | Clinical trials | Index Medicus
Journal Article
BMJ-BRITISH MEDICAL JOURNAL, ISSN 1756-1833, 11/2018, Volume 363
Randomised controlled trials are considered to be the best method to assess comparative clinical efficacy and effectiveness, and can be a key source of data... 
MEDICINE, GENERAL & INTERNAL | CLINICALLY IMPORTANT DIFFERENCE | ESTIMANDS
Journal Article
Statistics in Medicine, ISSN 0277-6715, 10/2019, Volume 38, Issue 23, pp. 4761 - 4771
The treatment effect in subgroups of patients is often of interest in randomized controlled clinical trials, as this may provide useful information on how to... 
clinical trial | identification | subgroup | causal inference | Bayesian analysis | estimand | Index Medicus
Journal Article
Pharmaceutical Statistics, ISSN 1539-1604, 01/2017, Volume 16, Issue 1, pp. 6 - 11
ICH E9 Statistical Principles for Clinical Trials was issued in 1998. In October 2014, an addendum to ICH E9 was proposed relating to estimands and sensitivity... 
ICH E9 | sensitivity analyses | estimands | CLINICAL-TRIALS | MISSING DATA | PHARMACOLOGY & PHARMACY | STATISTICS & PROBABILITY | Data Interpretation, Statistical | Drug Industry - methods | Humans | Drug Design | Clinical Trials as Topic - methods | Models, Statistical | Research Design | Index Medicus
Journal Article
Statistics in Biopharmaceutical Research, ISSN 1946-6315, 07/2018, Volume 10, Issue 3, pp. 233 - 235
Dropouts confound the treatment effect when the outcome and the dropout process both depend on subject characteristics. If dropout is unrelated to treatment,... 
Causal estimand | Effect among the treated | Principal stratification | Missing data | De jure effect | CLINICAL-TRIALS | MATHEMATICAL & COMPUTATIONAL BIOLOGY | STATISTICS & PROBABILITY | ESTIMANDS
Journal Article
Pharmaceutical Statistics, ISSN 1539-1604, 09/2017, Volume 16, Issue 5, pp. 378 - 392
In some randomized (drug versus placebo) clinical trials, the estimand of interest is the between‐treatment difference in population means of a clinical... 
quantile regression | missing at random | trimmed mean | sensitivity analysis | estimand | dropout | CHOOSING ESTIMANDS | TO-TREAT ANALYSIS | MULTIPLE-IMPUTATION | PHARMACOLOGY & PHARMACY | STATISTICS & PROBABILITY | INFERENCE | Data Interpretation, Statistical | Clinical Trials as Topic | Patient Dropouts | Clinical trials | Index Medicus
Journal Article
Statistics in Biopharmaceutical Research, ISSN 1946-6315, 01/2017, Volume 9, Issue 1, pp. 126 - 134
In linear regression models, covariate-adjusted analysis is not expected to change the estimates of the treatment effect in the clinical trials with randomized... 
Estimands | Type I error | Biased estimates | Power | BASE-LINE | RANDOMIZATION | MODELS | MATHEMATICAL & COMPUTATIONAL BIOLOGY | STATISTICS & PROBABILITY | OUTCOMES | LIKELIHOOD
Journal Article
Pharmaceutical Statistics, ISSN 1539-1604, 11/2018, Volume 17, Issue 6, pp. 685 - 700
Journal Article
Clinical Trials, ISSN 1740-7745, 8/2019, Volume 16, Issue 4, pp. 345 - 349
The proposed addendum to the International Conference on Harmonization document, Statistical Principles for Clinical Trials, can be read in two ways. There is... 
intent to treat | MEDICINE, RESEARCH & EXPERIMENTAL | TRIALS | principal stratification | Estimand | Clinical trials | Medical research | Index Medicus
Journal Article
Pharmaceutical Statistics, ISSN 1539-1604, 03/2019, Volume 18, Issue 2, pp. 166 - 183
The analysis of adverse events (AEs) is a key component in the assessment of a drug's safety profile. Inappropriate analysis methods may result in misleading... 
estimands | safety data | clinical trials | adverse events | benefit assessment | STATISTICAL-METHODS | TRIALS | COST-EFFECTIVENESS | METAANALYSIS | STATE | STATISTICS & PROBABILITY | COMPETING RISKS | MODEL | ISSUES | PHARMACOLOGY & PHARMACY | Clinical trials | Patient safety | Side effects | Drug therapy | Index Medicus | Main Papers | Main Paper
Journal Article
Journal of Drugs in Dermatology, ISSN 1545-9616, 08/2017, Volume 16, Issue 8, pp. 734 - 742
Background: An issue in long-term clinical trials of biologics in psoriasis is how to handle missing efficacy data. This methodological challenge may not be... 
IMPUTATION | PERSPECTIVE | USERS GUIDES | MEDICAL LITERATURE | ESTIMANDS | ETANERCEPT | DERMATOLOGY | Antibodies, Monoclonal - therapeutic use | Data Interpretation, Statistical | Psoriasis - drug therapy | Humans | Biological Products - therapeutic use | Randomized Controlled Trials as Topic
Journal Article
Statistics in Biopharmaceutical Research, ISSN 1946-6315, 01/2019, Volume 11, Issue 1, pp. 1 - 12
A year after joining the FDA in 2011 as Director of the Office of Biostatistics in the Center for Drug Evaluation and Research (CDER), I prepared a talk, and... 
Estimands | Statistical policy | Master protocols | Bayesian methods | Regulatory guidance | DESIGN | COMBINING MULTIPLE COMPARISONS | CLINICAL-TRIALS | MATHEMATICAL & COMPUTATIONAL BIOLOGY | MISSING DATA | STATISTICS & PROBABILITY | THERAPIES
Journal Article
Therapeutic Innovation & Regulatory Science, ISSN 2168-4790, 7/2015, Volume 49, Issue 4, pp. 584 - 592
Lack of adherence to study protocol and missing data are often unavoidable in clinical trials, and both increase the need to differentiate between the ideal... 
effectiveness | nonadherence | estimands | clinical trials | efficacy | missing data | ASSUMPTIONS | MEDICAL INFORMATICS | SENSITIVITY | PREVENTION | FRAMEWORK | PHARMACOLOGY & PHARMACY | Objectives | Clinical trials | Validity | Clinical medicine | Authorship
Journal Article