Methods for Equivalence and Noninferiority Testing

Classical hypothesis testing focuses on testing whether treatments have differential effects on outcome. However, sometimes clinicians may be more interested in determining whether treatments are equivalent or whether one has noninferior outcomes. We review the hypotheses for these noninferiority and equivalence research questions, consider power and sample size issues, and discuss how to perform such a test for both binary and survival outcomes. The methods are illustrated on 2 recent studies in hematopoietic cell transplantation.

A NOVEL AND SIMPLE RULE OF THUMB FOR MULTIPLICITY CONTROL IN EQUIVALENCE TESTING USING TWO ONE-SIDED TESTS

Equivalence testing is growing in use in scientific research outside of its traditional role in the drug approval process. Largely due to its ease of use and recommendation from the United States Food and Drug Administration guidance, the most common statistical method for testing (bio)equivalence is the two one-sided tests procedure (TOST). Like classical point-null hypothesis testing, TOST is subject to multiplicity concerns as more comparisons are made. In this manuscript, a condition that bounds the family-wise error rate (FWER) using TOST is given. This condition then leads to a simple solution for controlling the FWER. Specifically, we demonstrate that if all pairwise comparisons of k independent groups are being evaluated for equivalence, then simply scaling the nominal Type I error rate down by (k - 1) is sufficient to maintain the family-wise error rate at the desired value or less. The resulting rule is much less conservative than the equally simple Bonferroni correction. An example of equivalence testing in a non drug-development setting is given.

Analytical statistics : statistical methods, testing for significance /

Mode of access: Internet.

Comparative phylogeographic summary statistics for testing simultaneous vicariance

Testing for simultaneous vicariance across comparative phylogeographic data sets is a notoriously difficult problem hindered by mutational variance, the coalescent variance, and variability across pairs of sister taxa in parameters that affect genetic divergence. We simulate vicariance to characterize the behaviour of several commonly used summary statistics across a range of divergence times, and to characterize this behaviour in comparative phylogeographic datasets having multiple taxon-pairs. We found Tajima's D to be relatively uncorrelated with other summary statistics across divergence times, and using simple hypothesis testing of simultaneous vicariance given variable population sizes, we counter-intuitively found that the variance across taxon pairs in Nei and Li's net nucleotide divergence (pi(net)), a common measure of population divergence, is often inferior to using the variance in Tajima's D across taxon pairs as a test statistic to distinguish ancient simultaneous vicariance from variable vicariance histories. The opposite and more intuitive pattern is found for testing more recent simultaneous vicariance, and overall we found that depending on the timing of vicariance, one of these two test statistics can achieve high statistical power for rejecting simultaneous vicariance, given a reasonable number of intron loci (> 5 loci, 400 bp) and a range of conditions. These results suggest that components of these two composite summary statistics should be used in future simulation-based methods which can simultaneously use a pool of summary statistics to test comparative the phylogeographic hypotheses we consider here.

Essays on mixed-frequency data : forecasting and unit root testing

Doutoramento em Economia

An investigation into the barriers to the implementation of automation and robotics technologies in the construction industry

The rising problems associated with construction such as decreasing quality and productivity, labour shortages, occupational safety, and inferior working conditions have opened the possibility of more revolutionary solutions within the industry. One prospective option is in the implementation of innovative technologies such as automation and robotics, which has the potential to improve the industry in terms of productivity, safety and quality. The construction work site could, theoretically, be contained in a safer environment, with more efficient execution of the work, greater consistency of the outcome and higher level of control over the production process. By identifying the barriers to construction automation and robotics implementation in construction, and investigating ways in which to overcome them, contributions could be made in terms of better understanding and facilitating, where relevant, greater use of these technologies in the construction industry so as to promote its efficiency. This research aims to ascertain and explain the barriers to construction automation and robotics implementation by exploring and establishing the relationship between characteristics of the construction industry and attributes of existing construction automation and robotics technologies to level of usage and implementation in three selected countries; Japan, Australia and Malaysia. These three countries were chosen as their construction industry characteristics provide contrast in terms of culture, gross domestic product, technology application, organisational structure and labour policies. This research uses a mixed method approach of gathering data, both quantitative and qualitative, by employing a questionnaire survey and an interview schedule; using a wide range of sample from management through to on-site users, working in a range of small (less than AUD0.2million) to large companies (more than AUD500million), and involved in a broad range of business types and construction sectors. Detailed quantitative (statistical) and qualitative (content) data analysis is performed to provide a set of descriptions, relationships, and differences. The statistical tests selected for use include cross-tabulations, bivariate and multivariate analysis for investigating possible relationships between variables; and Kruskal-Wallis and Mann Whitney U test of independent samples for hypothesis testing and inferring the research sample to the construction industry population. Findings and conclusions arising from the research work which include the ranking schemes produced for four key areas of, the construction attributes on level of usage; barrier variables; differing levels of usage between countries; and future trends, have established a number of potential areas that could impact the level of implementation both globally and for individual countries.

Conducting statistical tests of hypotheses : five common misconceptions found in transportation research

Most statistical methods use hypothesis testing. Analysis of variance, regression, discrete choice models, contingency tables, and other analysis methods commonly used in transportation research share hypothesis testing as the means of making inferences about the population of interest. Despite the fact that hypothesis testing has been a cornerstone of empirical research for many years, various aspects of hypothesis tests commonly are incorrectly applied, misinterpreted, and ignored—by novices and expert researchers alike. On initial glance, hypothesis testing appears straightforward: develop the null and alternative hypotheses, compute the test statistic to compare to a standard distribution, estimate the probability of rejecting the null hypothesis, and then make claims about the importance of the finding. This is an oversimplification of the process of hypothesis testing. Hypothesis testing as applied in empirical research is examined here. The reader is assumed to have a basic knowledge of the role of hypothesis testing in various statistical methods. Through the use of an example, the mechanics of hypothesis testing is first reviewed. Then, five precautions surrounding the use and interpretation of hypothesis tests are developed; examples of each are provided to demonstrate how errors are made, and solutions are identified so similar errors can be avoided. Remedies are provided for common errors, and conclusions are drawn on how to use the results of this paper to improve the conduct of empirical research in transportation.

Mean and variance of estimates of the bispectrum of a harmonic random process-an analysis including leakage effects

Analytical expressions are derived for the mean and variance, of estimates of the bispectrum of a real-time series assuming a cosinusoidal model. The effects of spectral leakage, inherent in discrete Fourier transform operation when the modes present in the signal have a nonintegral number of wavelengths in the record, are included in the analysis. A single phase-coupled triad of modes can cause the bispectrum to have a nonzero mean value over the entire region of computation owing to leakage. The variance of bispectral estimates in the presence of leakage has contributions from individual modes and from triads of phase-coupled modes. Time-domain windowing reduces the leakage. The theoretical expressions for the mean and variance of bispectral estimates are derived in terms of a function dependent on an arbitrary symmetric time-domain window applied to the record. the number of data, and the statistics of the phase coupling among triads of modes. The theoretical results are verified by numerical simulations for simple test cases and applied to laboratory data to examine phase coupling in a hypothesis testing framework

Proactive detection of high collision concentration locations on highways

In previous research (Chung et al., 2009), the potential of the continuous risk profile (CRP) to proactively detect the systematic deterioration of freeway safety levels was presented. In this paper, this potential is investigated further, and an algorithm is proposed for proactively detecting sites where the collision rate is not sufficiently high to be classified as a high collision concentration location but where a systematic deterioration of safety level is observed. The approach proposed compares the weighted CRP across different years and uses the cumulative sum (CUSUM) algorithm to detect the sites where changes in collision rate are observed. The CRPs of the detected sites are then compared for reproducibility. When high reproducibility is observed, a growth factor is used for sequential hypothesis testing to determine if the collision profiles are increasing over time. Findings from applying the proposed method using empirical data are documented in the paper together with a detailed description of the method.

Toward resolving deep neoaves phylogeny : data, signal enhancement, and priors

We report three developments toward resolving the challenge of the apparent basal polytomy of neoavian birds. First, we describe improved conditional down-weighting techniques to reduce noise relative to signal for deeper divergences and find increased agreement between data sets. Second, we present formulae for calculating the probabilities of finding predefined groupings in the optimal tree. Finally, we report a significant increase in data: nine new mitochondrial (mt) genomes (the dollarbird, New Zealand kingfisher, great potoo, Australian owlet-nightjar, white-tailed trogon, barn owl, a roadrunner [a ground cuckoo], New Zealand long-tailed cuckoo, and the peach-faced lovebird) and together they provide data for each of the six main groups of Neoaves proposed by Cracraft J (2001). We use his six main groups of modern birds as priors for evaluation of results. These include passerines, cuckoos, parrots, and three other groups termed “WoodKing” (woodpeckers/rollers/kingfishers), “SCA” (owls/potoos/owlet-nightjars/hummingbirds/swifts), and “Conglomerati.” In general, the support is highly significant with just two exceptions, the owls move from the “SCA” group to the raptors, particularly accipitrids (buzzards/eagles) and the osprey, and the shorebirds may be an independent group from the rest of the “Conglomerati”. Molecular dating mt genomes support a major diversification of at least 12 neoavian lineages in the Late Cretaceous. Our results form a basis for further testing with both nuclear-coding sequences and rare genomic changes.

Revealing performance heterogeneity through knowledge management maturity evaluation : a capability-based approach

The dynamic capabilities view (DCV) focuses on renewal of firms’ strategic knowledge resources so as to sustain competitive advantage within turbulent markets. Within the context of the DCV, the focus of knowledge management (KM) is to develop the KMC through deploying knowledge governance mechanisms that are conducive to facilitating knowledge processes so as to produce superior business performance over time. The essence of KM performance evaluation is to assess how well the KMC is configured with knowledge governance mechanisms and processes that enable a firm to achieve superior performance through matching its knowledge base with market needs. However, little research has been undertaken to evaluate KM performance from the DCV perspective. This study employed a survey study design and adopted hypothesis-testing approaches to develop a capability-based KM evaluation framework (CKMEF) that upholds the basic assertions of the DCV. Under the governance of the framework, a KM index (KMI) and a KM maturity model (KMMM) were derived not only to indicate the extent to which a firm’s KM implementations fulfill its strategic objectives, and to identify the evolutionary phase of its KMC, but also to bench-mark the KMC in the research population. The research design ensured that the evaluation framework and instruments have statistical significance and good generalizabilty to be applied in the research population, namely construction firms operating in the dynamic Hong Kong construction market. The study demonstrated the feasibility of quantitatively evaluating the development of the KMC and revealing the performance heterogeneity associated with the development.

A pilot randomised trial of Medilixir cream shows some promising results, but control treatment may have been inappropriate

Aim To test the efficacy of Medilixir [cream] against the standard treatment of aqueous cream in the provision of relief from the symptoms of postburn itch. Design RCT with two parallel arms. Setting Professor Stuart Pegg Adult Burns Centre, Royal Brisbane and Women's Hospital, Brisbane, Australia. Participants Fifty-two patients aged between 18 and 80 years, admitted directly to the burns centre between 10 March and 22 July 2008, were able to provide informed consent, and had shown no allergic reaction to a patch test with the study medication, were randomised. Patients admitted from intensive care or high dependency were excluded. Main results Effect estimates and confidence intervals were not reported for any of the outcomes; only group means/proportions and P-values from hypothesis testing were provided. More patients in the intervention group reported itch reduction compared to comparison treatment (91 vs. 82%, P=0.001). Itch recurrence after cream application occurred later in the intervention group compared to the control group (P<0.001). Use of antipruritic medication was significantly greater in the control group (P=0.023). There was no difference in sleep disturbance between groups (not quantified). On average, Medilixir took longer to apply than aqueous cream (157s for Medilixir vs. 139s for aqueous cream; mean difference 17s), but authors noted that the groups did not differ significantly (CI for mean difference and P-values were not reported).

Damping structure selection in nonlinear ship manoeuvring models

This paper presents the application of a statistical method for model structure selection of lift-drag and viscous damping components in ship manoeuvring models. The damping model is posed as a family of linear stochastic models, which is postulated based on previous work in the literature. Then a nested test of hypothesis problem is considered. The testing reduces to a recursive comparison of two competing models, for which optimal tests in the Neyman sense exist. The method yields a preferred model structure and its initial parameter estimates. Alternatively, the method can give a reduced set of likely models. Using simulated data we study how the selection method performs when there is both uncorrelated and correlated noise in the measurements. The first case is related to instrumentation noise, whereas the second case is related to spurious wave-induced motion often present during sea trials. We then consider the model structure selection of a modern high-speed trimaran ferry from full scale trial data.

Adoption of accountable-eHealth systems by future healthcare professionals: An empirical research model based on the Australian context

This paper provides a first look at the acceptance of Accountable-eHealth systems, a new genre of eHealth systems, designed to manage information privacy concerns that hinder the proliferation of eHealth. The underlying concept of AeH systems is appropriate use of information through after-the-fact accountability for intentional misuse of information by healthcare professionals. An online questionnaire survey was utilised for data collection from three educational institutions in Queensland, Australia. A total of 23 hypothesis relating to 9 constructs were tested using a structural equation modelling technique. A total of 334 valid responses were received. The cohort consisted of medical, nursing and other health related students studying at various levels in both undergraduate and postgraduate courses. The hypothesis testing disproved 7 hypotheses. The empirical research model developed was capable of predicting 47.3% of healthcare professionals’ perceived intention to use AeH systems. A validation of the model with a wider survey cohort would be useful to confirm the current findings.