Using the Student's T-Test with Extremely Small Sample Sizes
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The Magic of Randomization Versus the Myth of Real-World Evidence
The new england journal of medicine Sounding Board The Magic of Randomization versus the Myth of Real-World Evidence Rory Collins, F.R.S., Louise Bowman, M.D., F.R.C.P., Martin Landray, Ph.D., F.R.C.P., and Richard Peto, F.R.S. Nonrandomized observational analyses of large safety and efficacy because the potential biases electronic patient databases are being promoted with respect to both can be appreciable. For ex- as an alternative to randomized clinical trials as ample, the treatment that is being assessed may a source of “real-world evidence” about the effi- well have been provided more or less often to cacy and safety of new and existing treatments.1-3 patients who had an increased or decreased risk For drugs or procedures that are already being of various health outcomes. Indeed, that is what used widely, such observational studies may in- would be expected in medical practice, since both volve exposure of large numbers of patients. the severity of the disease being treated and the Consequently, they have the potential to detect presence of other conditions may well affect the rare adverse effects that cannot plausibly be at- choice of treatment (often in ways that cannot be tributed to bias, generally because the relative reliably quantified). Even when associations of risk is large (e.g., Reye’s syndrome associated various health outcomes with a particular treat- with the use of aspirin, or rhabdomyolysis as- ment remain statistically significant after adjust- sociated with the use of statin therapy).4 Non- ment for all the known differences between pa- randomized clinical observation may also suf- tients who received it and those who did not fice to detect large beneficial effects when good receive it, these adjusted associations may still outcomes would not otherwise be expected (e.g., reflect residual confounding because of differ- control of diabetic ketoacidosis with insulin treat- ences in factors that were assessed only incom- ment, or the rapid shrinking of tumors with pletely or not at all (and therefore could not be chemotherapy). -
(Meta-Analyses of Observational Studies in Epidemiology) Checklist
MOOSE (Meta-analyses Of Observational Studies in Epidemiology) Checklist A reporting checklist for Authors, Editors, and Reviewers of Meta-analyses of Observational Studies. You must report the page number in your manuscript where you consider each of the items listed in this checklist. If you have not included this information, either revise your manuscript accordingly before submitting or note N/A. Reporting Criteria Reported (Yes/No) Reported on Page No. Reporting of Background Problem definition Hypothesis statement Description of Study Outcome(s) Type of exposure or intervention used Type of study design used Study population Reporting of Search Strategy Qualifications of searchers (eg, librarians and investigators) Search strategy, including time period included in the synthesis and keywords Effort to include all available studies, including contact with authors Databases and registries searched Search software used, name and version, including special features used (eg, explosion) Use of hand searching (eg, reference lists of obtained articles) List of citations located and those excluded, including justification Method for addressing articles published in languages other than English Method of handling abstracts and unpublished studies Description of any contact with authors Reporting of Methods Description of relevance or appropriateness of studies assembled for assessing the hypothesis to be tested Rationale for the selection and coding of data (eg, sound clinical principles or convenience) Documentation of how data were classified -
Chapter 4: Fisher's Exact Test in Completely Randomized Experiments
1 Chapter 4: Fisher’s Exact Test in Completely Randomized Experiments Fisher (1925, 1926) was concerned with testing hypotheses regarding the effect of treat- ments. Specifically, he focused on testing sharp null hypotheses, that is, null hypotheses under which all potential outcomes are known exactly. Under such null hypotheses all un- known quantities in Table 4 in Chapter 1 are known–there are no missing data anymore. As we shall see, this implies that we can figure out the distribution of any statistic generated by the randomization. Fisher’s great insight concerns the value of the physical randomization of the treatments for inference. Fisher’s classic example is that of the tea-drinking lady: “A lady declares that by tasting a cup of tea made with milk she can discriminate whether the milk or the tea infusion was first added to the cup. ... Our experi- ment consists in mixing eight cups of tea, four in one way and four in the other, and presenting them to the subject in random order. ... Her task is to divide the cups into two sets of 4, agreeing, if possible, with the treatments received. ... The element in the experimental procedure which contains the essential safeguard is that the two modifications of the test beverage are to be prepared “in random order.” This is in fact the only point in the experimental procedure in which the laws of chance, which are to be in exclusive control of our frequency distribution, have been explicitly introduced. ... it may be said that the simple precaution of randomisation will suffice to guarantee the validity of the test of significance, by which the result of the experiment is to be judged.” The approach is clear: an experiment is designed to evaluate the lady’s claim to be able to discriminate wether the milk or tea was first poured into the cup. -
Quasi-Experimental Studies in the Fields of Infection Control and Antibiotic Resistance, Ten Years Later: a Systematic Review
HHS Public Access Author manuscript Author ManuscriptAuthor Manuscript Author Infect Control Manuscript Author Hosp Epidemiol Manuscript Author . Author manuscript; available in PMC 2019 November 12. Published in final edited form as: Infect Control Hosp Epidemiol. 2018 February ; 39(2): 170–176. doi:10.1017/ice.2017.296. Quasi-experimental Studies in the Fields of Infection Control and Antibiotic Resistance, Ten Years Later: A Systematic Review Rotana Alsaggaf, MS, Lyndsay M. O’Hara, PhD, MPH, Kristen A. Stafford, PhD, MPH, Surbhi Leekha, MBBS, MPH, Anthony D. Harris, MD, MPH, CDC Prevention Epicenters Program Department of Epidemiology and Public Health, University of Maryland School of Medicine, Baltimore, Maryland. Abstract OBJECTIVE.—A systematic review of quasi-experimental studies in the field of infectious diseases was published in 2005. The aim of this study was to assess improvements in the design and reporting of quasi-experiments 10 years after the initial review. We also aimed to report the statistical methods used to analyze quasi-experimental data. DESIGN.—Systematic review of articles published from January 1, 2013, to December 31, 2014, in 4 major infectious disease journals. METHODS.—Quasi-experimental studies focused on infection control and antibiotic resistance were identified and classified based on 4 criteria: (1) type of quasi-experimental design used, (2) justification of the use of the design, (3) use of correct nomenclature to describe the design, and (4) statistical methods used. RESULTS.—Of 2,600 articles, 173 (7%) featured a quasi-experimental design, compared to 73 of 2,320 articles (3%) in the previous review (P<.01). Moreover, 21 articles (12%) utilized a study design with a control group; 6 (3.5%) justified the use of a quasi-experimental design; and 68 (39%) identified their design using the correct nomenclature. -
Epidemiology and Biostatistics (EPBI) 1
Epidemiology and Biostatistics (EPBI) 1 Epidemiology and Biostatistics (EPBI) Courses EPBI 2219. Biostatistics and Public Health. 3 Credit Hours. This course is designed to provide students with a solid background in applied biostatistics in the field of public health. Specifically, the course includes an introduction to the application of biostatistics and a discussion of key statistical tests. Appropriate techniques to measure the extent of disease, the development of disease, and comparisons between groups in terms of the extent and development of disease are discussed. Techniques for summarizing data collected in samples are presented along with limited discussion of probability theory. Procedures for estimation and hypothesis testing are presented for means, for proportions, and for comparisons of means and proportions in two or more groups. Multivariable statistical methods are introduced but not covered extensively in this undergraduate course. Public Health majors, minors or students studying in the Public Health concentration must complete this course with a C or better. Level Registration Restrictions: May not be enrolled in one of the following Levels: Graduate. Repeatability: This course may not be repeated for additional credits. EPBI 2301. Public Health without Borders. 3 Credit Hours. Public Health without Borders is a course that will introduce you to the world of disease detectives to solve public health challenges in glocal (i.e., global and local) communities. You will learn about conducting disease investigations to support public health actions relevant to affected populations. You will discover what it takes to become a field epidemiologist through hands-on activities focused on promoting health and preventing disease in diverse populations across the globe. -
Pearson-Fisher Chi-Square Statistic Revisited
Information 2011 , 2, 528-545; doi:10.3390/info2030528 OPEN ACCESS information ISSN 2078-2489 www.mdpi.com/journal/information Communication Pearson-Fisher Chi-Square Statistic Revisited Sorana D. Bolboac ă 1, Lorentz Jäntschi 2,*, Adriana F. Sestra ş 2,3 , Radu E. Sestra ş 2 and Doru C. Pamfil 2 1 “Iuliu Ha ţieganu” University of Medicine and Pharmacy Cluj-Napoca, 6 Louis Pasteur, Cluj-Napoca 400349, Romania; E-Mail: [email protected] 2 University of Agricultural Sciences and Veterinary Medicine Cluj-Napoca, 3-5 M ănăş tur, Cluj-Napoca 400372, Romania; E-Mails: [email protected] (A.F.S.); [email protected] (R.E.S.); [email protected] (D.C.P.) 3 Fruit Research Station, 3-5 Horticultorilor, Cluj-Napoca 400454, Romania * Author to whom correspondence should be addressed; E-Mail: [email protected]; Tel: +4-0264-401-775; Fax: +4-0264-401-768. Received: 22 July 2011; in revised form: 20 August 2011 / Accepted: 8 September 2011 / Published: 15 September 2011 Abstract: The Chi-Square test (χ2 test) is a family of tests based on a series of assumptions and is frequently used in the statistical analysis of experimental data. The aim of our paper was to present solutions to common problems when applying the Chi-square tests for testing goodness-of-fit, homogeneity and independence. The main characteristics of these three tests are presented along with various problems related to their application. The main problems identified in the application of the goodness-of-fit test were as follows: defining the frequency classes, calculating the X2 statistic, and applying the χ2 test. -
Guidelines for Reporting Meta-Epidemiological Methodology Research
EBM Primer Evid Based Med: first published as 10.1136/ebmed-2017-110713 on 12 July 2017. Downloaded from Guidelines for reporting meta-epidemiological methodology research Mohammad Hassan Murad, Zhen Wang 10.1136/ebmed-2017-110713 Abstract The goal is generally broad but often focuses on exam- Published research should be reported to evidence users ining the impact of certain characteristics of clinical studies on the observed effect, describing the distribu- Evidence-Based Practice with clarity and transparency that facilitate optimal tion of research evidence in a specific setting, exam- Center, Mayo Clinic, Rochester, appraisal and use of evidence and allow replication Minnesota, USA by other researchers. Guidelines for such reporting ining heterogeneity and exploring its causes, identifying are available for several types of studies but not for and describing plausible biases and providing empirical meta-epidemiological methodology studies. Meta- evidence for hypothesised associations. Unlike classic Correspondence to: epidemiological studies adopt a systematic review epidemiology, the unit of analysis for meta-epidemio- Dr Mohammad Hassan Murad, or meta-analysis approach to examine the impact logical studies is a study, not a patient. The outcomes Evidence-based Practice Center, of certain characteristics of clinical studies on the of meta-epidemiological studies are usually not clinical Mayo Clinic, 200 First Street 6–8 observed effect and provide empirical evidence for outcomes. SW, Rochester, MN 55905, USA; hypothesised associations. The unit of analysis in meta- In this guide, we adapt the items used in the PRISMA murad. mohammad@ mayo. edu 9 epidemiological studies is a study, not a patient. The statement for reporting systematic reviews and outcomes of meta-epidemiological studies are usually meta-analysis to fit the setting of meta- epidemiological not clinical outcomes. -
Statistical Significance Testing in Information Retrieval:An Empirical
Statistical Significance Testing in Information Retrieval: An Empirical Analysis of Type I, Type II and Type III Errors Julián Urbano Harlley Lima Alan Hanjalic Delft University of Technology Delft University of Technology Delft University of Technology The Netherlands The Netherlands The Netherlands [email protected] [email protected] [email protected] ABSTRACT 1 INTRODUCTION Statistical significance testing is widely accepted as a means to In the traditional test collection based evaluation of Information assess how well a difference in effectiveness reflects an actual differ- Retrieval (IR) systems, statistical significance tests are the most ence between systems, as opposed to random noise because of the popular tool to assess how much noise there is in a set of evaluation selection of topics. According to recent surveys on SIGIR, CIKM, results. Random noise in our experiments comes from sampling ECIR and TOIS papers, the t-test is the most popular choice among various sources like document sets [18, 24, 30] or assessors [1, 2, 41], IR researchers. However, previous work has suggested computer but mainly because of topics [6, 28, 36, 38, 43]. Given two systems intensive tests like the bootstrap or the permutation test, based evaluated on the same collection, the question that naturally arises mainly on theoretical arguments. On empirical grounds, others is “how well does the observed difference reflect the real difference have suggested non-parametric alternatives such as the Wilcoxon between the systems and not just noise due to sampling of topics”? test. Indeed, the question of which tests we should use has accom- Our field can only advance if the published retrieval methods truly panied IR and related fields for decades now. -
Simple Linear Regression: Straight Line Regression Between an Outcome Variable (Y ) and a Single Explanatory Or Predictor Variable (X)
1 Introduction to Regression \Regression" is a generic term for statistical methods that attempt to fit a model to data, in order to quantify the relationship between the dependent (outcome) variable and the predictor (independent) variable(s). Assuming it fits the data reasonable well, the estimated model may then be used either to merely describe the relationship between the two groups of variables (explanatory), or to predict new values (prediction). There are many types of regression models, here are a few most common to epidemiology: Simple Linear Regression: Straight line regression between an outcome variable (Y ) and a single explanatory or predictor variable (X). E(Y ) = α + β × X Multiple Linear Regression: Same as Simple Linear Regression, but now with possibly multiple explanatory or predictor variables. E(Y ) = α + β1 × X1 + β2 × X2 + β3 × X3 + ::: A special case is polynomial regression. 2 3 E(Y ) = α + β1 × X + β2 × X + β3 × X + ::: Generalized Linear Model: Same as Multiple Linear Regression, but with a possibly transformed Y variable. This introduces considerable flexibil- ity, as non-linear and non-normal situations can be easily handled. G(E(Y )) = α + β1 × X1 + β2 × X2 + β3 × X3 + ::: In general, the transformation function G(Y ) can take any form, but a few forms are especially common: • Taking G(Y ) = logit(Y ) describes a logistic regression model: E(Y ) log( ) = α + β × X + β × X + β × X + ::: 1 − E(Y ) 1 1 2 2 3 3 2 • Taking G(Y ) = log(Y ) is also very common, leading to Poisson regression for count data, and other so called \log-linear" models. -
Tests of Hypotheses Using Statistics
Tests of Hypotheses Using Statistics Adam Massey¤and Steven J. Millery Mathematics Department Brown University Providence, RI 02912 Abstract We present the various methods of hypothesis testing that one typically encounters in a mathematical statistics course. The focus will be on conditions for using each test, the hypothesis tested by each test, and the appropriate (and inappropriate) ways of using each test. We conclude by summarizing the di®erent tests (what conditions must be met to use them, what the test statistic is, and what the critical region is). Contents 1 Types of Hypotheses and Test Statistics 2 1.1 Introduction . 2 1.2 Types of Hypotheses . 3 1.3 Types of Statistics . 3 2 z-Tests and t-Tests 5 2.1 Testing Means I: Large Sample Size or Known Variance . 5 2.2 Testing Means II: Small Sample Size and Unknown Variance . 9 3 Testing the Variance 12 4 Testing Proportions 13 4.1 Testing Proportions I: One Proportion . 13 4.2 Testing Proportions II: K Proportions . 15 4.3 Testing r £ c Contingency Tables . 17 4.4 Incomplete r £ c Contingency Tables Tables . 18 5 Normal Regression Analysis 19 6 Non-parametric Tests 21 6.1 Tests of Signs . 21 6.2 Tests of Ranked Signs . 22 6.3 Tests Based on Runs . 23 ¤E-mail: [email protected] yE-mail: [email protected] 1 7 Summary 26 7.1 z-tests . 26 7.2 t-tests . 27 7.3 Tests comparing means . 27 7.4 Variance Test . 28 7.5 Proportions . 28 7.6 Contingency Tables . -
Statistical Significance
Statistical significance In statistical hypothesis testing,[1][2] statistical signif- 1.1 Related concepts icance (or a statistically significant result) is at- tained whenever the observed p-value of a test statis- The significance level α is the threshhold for p below tic is less than the significance level defined for the which the experimenter assumes the null hypothesis is study.[3][4][5][6][7][8][9] The p-value is the probability of false, and something else is going on. This means α is obtaining results at least as extreme as those observed, also the probability of mistakenly rejecting the null hy- given that the null hypothesis is true. The significance pothesis, if the null hypothesis is true.[22] level, α, is the probability of rejecting the null hypothe- Sometimes researchers talk about the confidence level γ sis, given that it is true.[10] This statistical technique for = (1 − α) instead. This is the probability of not rejecting testing the significance of results was developed in the the null hypothesis given that it is true. [23][24] Confidence early 20th century. levels and confidence intervals were introduced by Ney- In any experiment or observation that involves drawing man in 1937.[25] a sample from a population, there is always the possibil- ity that an observed effect would have occurred due to sampling error alone.[11][12] But if the p-value of an ob- 2 Role in statistical hypothesis test- served effect is less than the significance level, an inves- tigator may conclude that that effect reflects the charac- ing teristics of the -
Understanding Statistical Hypothesis Testing: the Logic of Statistical Inference
Review Understanding Statistical Hypothesis Testing: The Logic of Statistical Inference Frank Emmert-Streib 1,2,* and Matthias Dehmer 3,4,5 1 Predictive Society and Data Analytics Lab, Faculty of Information Technology and Communication Sciences, Tampere University, 33100 Tampere, Finland 2 Institute of Biosciences and Medical Technology, Tampere University, 33520 Tampere, Finland 3 Institute for Intelligent Production, Faculty for Management, University of Applied Sciences Upper Austria, Steyr Campus, 4040 Steyr, Austria 4 Department of Mechatronics and Biomedical Computer Science, University for Health Sciences, Medical Informatics and Technology (UMIT), 6060 Hall, Tyrol, Austria 5 College of Computer and Control Engineering, Nankai University, Tianjin 300000, China * Correspondence: [email protected]; Tel.: +358-50-301-5353 Received: 27 July 2019; Accepted: 9 August 2019; Published: 12 August 2019 Abstract: Statistical hypothesis testing is among the most misunderstood quantitative analysis methods from data science. Despite its seeming simplicity, it has complex interdependencies between its procedural components. In this paper, we discuss the underlying logic behind statistical hypothesis testing, the formal meaning of its components and their connections. Our presentation is applicable to all statistical hypothesis tests as generic backbone and, hence, useful across all application domains in data science and artificial intelligence. Keywords: hypothesis testing; machine learning; statistics; data science; statistical inference 1. Introduction We are living in an era that is characterized by the availability of big data. In order to emphasize the importance of this, data have been called the ‘oil of the 21st Century’ [1]. However, for dealing with the challenges posed by such data, advanced analysis methods are needed.