Events

Targeted Maximum Likelihood Estimation (TMLE) provides an approach for estimating the causal effects of longitudinal interventions with several attractive properties. TMLE uses estimates of both the propensity score (as used in inverse probability weighting) and of a series of outcome regressions (as can be used...

In this talk, we will consider two innovative trial designs relevant to the modern field of mobile health (mHealth), namely, the sequential multiple-assignment randomized trial (SMART), and the more recently developed micro-randomized trial (MRT). Both designs involve sequential, within-individual randomizations, but are different in their...

We recently fit a series of models to account for uncertainty and variation in coronavirus tests. I will talk about the background of this problem and our analysis, and then we will expand into a general discussion of Bayesian workflow.

Andrew is a professor...

Many epidemiological questions concern potential interventions to alter the pathways presumed to mediate an association.

Individualised treatment, which relies on the ability to identify and prescribe subject-specific treatments, is an important application of using data in the context of decision-making.

Phase III randomised controlled trials (RCTs) are typically long and expensive, restricting their use and resulting in long lead times to answer important clinical questions. Researchers and funders have recognised the need for trials to become more efficient, yet the overwhelming majority of trials continue...

The Victorian Centre for Biostatistics (ViCBiostat) will offer two exciting new courses in the 2020 edition of its Summer School.

In this era of “data science” it is vitally important to clearly articulate the...

Effective treatment of chronic diseases and disorders typically requires ongoing interventions where clinicians sequentially make therapeutic decisions, adapting the type, dosage and timing of treatment according to evolving patient characteristics. The framework of dynamic treatment regimens (DTRs) or adaptive interventions formalizes this sequential decision-making process...

Big data. Multi-omics. Machine learning. Technology continues to increase our ability to both produce and summarize data on human health and function. Taking as a goal the production of knowledge about disease etiology or mechanisms for treatment effects in intensely followed observational cohorts, what are...

Our recent breakthroughs and advances in culture independent techniques (whole genome shotgun metagenomics, 16S rRNA amplicon sequencing) have dramatically changed the way we can examine microbial communities. But does the hype of microbiome outweigh the potential of our understanding of this ‘second genome’? There are...

General purpose MCMC software packages like WinBUGS, JAGS, and STAN enable users to define and fit almost any statistical model without having to worry about implementation details and have enabled significant progress in applied Bayesian modelling. However, these existing tools are largely unable to make...

How can we understand the effects of DNA variation on gene expression in single cells? What sort of studies should we conduct and what computational tools do we need for them to succeed? Following a short introduction to the field of single-cell biology I will...

The ViCBiostat Summer School is back in 2019, this time in partnership with the Australian Clinical Trials Alliance (ACTA). Each program is designed to provide an intensive introduction to a range of intermediate to advanced statistical methods that...

There is a pressing need to integrate innovative methodologies to improve clinical trials in the setting of small sample population groups. The objective of this talk is to present research that produces methods of general applicability as developed through multidisciplinary and close collaborations among researchers...

Electronic Health Record (EHR) data are used increasingly for comparative effectiveness research (CER). This growing source of rich clinical information provides a cost and time-effective opportunity to conduct retrospective cohort studies with large, representative samples of the diverse patient populations found in real-world clinical settings....

Multi-state models are increasingly being used to model complex disease profiles. By modelling transitions between disease states, accounting for competing events at each transition, we can gain a much richer understanding of patient trajectories and how risk factors impact over the entire disease pathway. In...

Whether associations found by observational studies are causal, and in which direction, are important issues with clinical and aetiological implications. We have developed ICE FALCON (Inference on Causation from Examination of Familial Confounding), an analytical approach to make inference about causation using data of twin...

This talk starts with the problem of Cox’s proportional hazards model estimation for a dementia dataset where informative right censoring is likely to exist. We adopt the method of maximum penalized likelihood, where dependence between censoring and event time is modelled by a copula function...

Much research to date has found that exposure to ambient fine particles is associated with asthma development and morbidity, but there is little work examining the effects of long-term exposure to coarse particles on respiratory health. Because of this research gap, it is difficult for...

We consider the situation where there is a known established regression model that can be used to predict an important outcome, Y, from a set of commonly available predictor variables X. There are many examples of this in the medical and epidemiologic literature. A new...

The assessment of risk of bias in randomized trials (RCTs) and non-randomized studies of interventions (NRSI) has evolved substantially in recent years. These changes are reflected in newly available tools (ROB 2.0 for randomized trials and ROBINS-I for NRSI; see www.riskofbias.info ). These new tools...

In 2003 I presented a Melbourne biostatistics seminar on an application of a newly emerging method – marginal structural modelling – to address time-varying confounding in observational studies of the effect of health care interventions. Penetrating questions revealed deficiencies in my understanding of the approach....

This year our Summer School is in conjunction with Cancer Council Victoria to present a five day Melbourne program.

Monday and Tuesday will be a course on meta-analysis methods presented by ViCBiostat. These can be taken together or as single day options. Wednesday, Thursday...

Classical regression models, such as linear or logistic regression are the standard approach in biostatistics.

Proper methodological planning and excellent logistics is the key to success or failure of clinical trials.

Large-scale data collection is costly, with the Australian Census costing $300 million in 2006, and $440 million in 2011.

Propensity score methods are commonly used to estimate causal effects in non-experimental studies. Existing propensity score methods assume that covariates are measured without error but covariate measurement error is likely common.

This talk will discuss the implications of measurement error in the covariates on...

ViCBiostat is hosting respected speaker Professor Elizabeth Stuart from Johns Hopkins University who will present this one-day workshop.

Propensity scores are an increasingly common tool for estimating the effects of interventions in observational (“non-experimental”) settings and for answering complex questions in randomized controlled trials....

Cluster randomised trials have been implemented increasingly over the past 30 years or so. More recently, design variants such as stepped wedge and crossover designs have been gaining favour (and flavour) due to emerging results that they have efficiency advantages over conventional parallel arm cluster...

Australian diagnostic pathology laboratories operate under strict regimes of quality monitoring testing, with programs run by both the National Association of Testing Authorities (NATA) and the Quality Assurance Program of the Royal College of Pathologists of Australasia (RCPA-QAP). In this talk I will report on...

The potential for overestimation of the treatment effect when a clinical trial stops early has been discussed extensively in the literature. However, there has been much less attention paid to the converse issue, namely, that sequentially monitored clinical trials which do not stop early tend...

Tyler VanderWeele provided two separate definitions of effect heterogeneity, which he referred to as “effect modification in distribution” and “effect modification in measure”. The standard epidemiological approach, based on effect modification in measure, is associated with a number of well-described shortcomings, and no consensus exists...

As medical research continues to push into new frontiers of discovery and personalized patient care, it is imperative that clinical trial designs evolve to address the forthcoming challenges. One key innovation is the use of adaptive clinical trial designs. Such designs allow certain trial design...

In studies with multiple incomplete variables, it is widely understood that if the data are missing at random (MAR) then unbiased estimation is possible with appropriate methods. While the need to assess the plausibility of this assumption has been emphasised, the practical difficulty of this...

The inaugural ViCBiostat Winter School will commence in June this year with a 5 day Melbourne program, designed to provide an intensive introduction to a range of intermediate to advanced statistical methods that are important in modern health...

The ACTN3 gene, known as ‘the gene for speed’, encodes a protein expressed in fast-twitch muscle fibres. About 20% of the population do not express the protein due to a loss-of-function mutation. Presence of one or two copies of this mutation is associated with reduced...

This seminar is presented in conjunction with the School of Mathematics and Statistics, University of Melbourne

The European Randomised Study of Prostate Cancer Screening has shown a substantial reduction in prostate cancer mortality, but a similar trial in the United States showed essentially...

In the development of drugs or therapeutic interventions, the use of trials with different designs is frequent. In particular parallel group and cross-over trials are often used. When there is a need to pool the results of such studies into a meta-analysis, combining results from...

Sanjoy Paul is Professor of Clinical Biostatistics, Epidemiology and Health Sciences Research, University of Melbourne and Director of Melbourne EpiCentre, a collaborative research centre within the Royal Melbourne Hospital, Melbourne, Australia. Prior to establishing his clinical trials research group in Australia, he created the Statistics...

In demography the actuarial estimator of the survival function has been used for centuries, typically with one year time intervals. This estimator was also the default in medical science and epidemiology until the advent of computers, when the Kaplan‐Meier estimator and later the Cox model...

This year our Summer School is back on the road, with a three-day program in Melbourne followed by a two-day program in Sydney. Each program is designed to provide an intensive introduction to a range of intermediate to...

In randomized clinical trials with baseline variables that are prognostic for the primary outcome, there is potential to improve precision and reduce sample size by appropriately adjusting for these variables. A major challenge is that there are multiple statistical methods to adjust for baseline variables,...

In this talk Professor Allore will present the evolution of longitudinal modeling in the field of aging using ordinal data of functional disability. The presentation will demonstrate the progression from time to event models to multistate models and recurrent events, latent trajectory and growth mixture...

PLEASE NOTE: This is very preliminary work, and unpublished as of yet.

The volume of high-throughput data makes it a daunting prospect to plot, but relying primarily on false discovery rate adjusted p-values is not enough. Making plots of the data is essential...

Antenatal mental disorders are often unrecognized, despite frequent contact with healthcare professionals throughout pregnancy. The UK National Institute for Clinical Excellence recommends maternity professionals use the two Whooley questions to identify depressive disorders – the most common antenatal mental disorder - in the perinatal period....

The number of births (parity) clearly bears a relationship to a woman’s age. A negative binomial regression model for parity is developed, in which mean parity is modelled with two components relating to age. The first is a parametric growth curve which operates during the...

Modelling epidemics has become a commonplace activity over the past 40 years, since the publication of Professors Anderson and May's first pathbreaking papers. Analytical techniques adapted from the hard sciences such as physics have given way to a field dominated by computational simulation models fitted...

In the first part of this talk I will introduce one of the methods to estimate the causal effect of time-varying exposures: inverse probability of treatment weighting (IPTW) of marginal structural models (MSMs). These models proposed by Robins et al (2000) allow estimation of causal...

Heritability, as a measure of (relative) variation in genetic causes, has been well-defined for a continuous trait for nearly a century, with wise caveats from its inventor, R.A. Fisher. Extension of this concept to a binary trait by invoking an unmeasured ‘liability’ construct has been...

Multiple endpoints are increasingly used in clinical trials. The significance of some of these clinical trials is established if at least r null hypotheses are rejected among m that are simultaneously tested. The usual approach in multiple hypothesis testing is to control the family-wise error...

This year we are taking our Summer School on the road, with a two-day program in Adelaide, followed by a five-day program in Melbourne. Each program is designed to provide an intensive introduction to a range of intermediate...

Understanding treatment effect heterogeneity is an important aspect of randomised trials, and process variables describing the intervention content are crucial components of this. Frequently these variables can only be measured in intervention groups.

Principal stratification, whereby control group participants are assigned to the latent...

Studying the mechanisms that explain the connections between exposures and outcomes, commonly known as “mediation analysis”, has a long history in social science and economics and has appeared more recently with increasing frequency in epidemiological research. The standard approach for constructing inferences for parameters in...

In clinical trials one traditionally models the effect of treatment on the mean response. The underlying assumption is that treatment affects the response distribution through a mean location shift on a suitable scale, with other aspects of the distribution (shape/dispersion/variance) remaining the same. This work...

A lot of health data is ordinal: even if the measurement process is interval or ratio level, a 10mmHg blood pressure difference doesn't mean the same thing at different starting points. Tests that only used the ordinal structure of the data would seem to be...

The ROC curve is a popular graphical method used to study the diagnostic capacity of biomarkers. In its simplest form it plots true-positive rates against false-positive rates. Both practical and theoretical aspects of the properties of ROC curves have been extensively studied. Conventionally, it is...

Stephen will present findings from the People of the British Isles project, recently published with Stephen as first author as a cover story in one of the world’s leading scientific journals “Nature”. In particular, he will show that using newly developed statistical techniques one can...

In the last 10-15 years, there has been an explosion of prediction models developed to predict risk of various diseases. The main objective of a risk prediction model is to predict the absolute risk of a disease. For this reason, cohort study design has been...

Dynamic treatment regimens (DTRs) are sequential decision rules that specify how to adapt the type, dosage and timing of treatment according to an individual patient’s time-varying characteristics. DTRs offer a framework for operationalizing the multistage decision making in personalized clinical practice, thus providing an opportunity...

Many important clinical questions are difficult to answer with a classically designed randomised controlled trial. Using innovative trial designs has the potential to increase the efficiency and capacity for conducting high quality trials. This one-day workshop will include presentations, discussions and clinical examples of alternative...

In 2010, Jian Yang and Peter Visscher showed how by applying mixed models to genome-wide association study data, it was possible to estimate how much of a trait's heritability is explained by all (common) genetic variants, and to partition this by, say, chromosome or variant...

Rates of cancer are increased following low-dose radiation from computed tomography (CT) scans used for medical diagnosis. However, when the lag period between a CT scan and diagnosis of cancer is very short, it is likely that the cancer was not caused by the radiation,...

Problem 1. How to evaluate the probability that bones found in a carpark are from a specified dead king: Although the evidence for bones found under a Leicester UK carpark to have been those of King Richard III seemed extremely strong even before...

DAY 1. Causal inference: concepts and methods

The past two decades has seen the emergence of a coherent theory of causal inference much of which is now being translated into practice. Causal concepts are increasingly being used in the design of studies and...

Censored data are common in medical studies. Survival time is a simple example. Recurrent events and paired interval-censored data (including detection limit as a special case) are more complex forms of censored data. The former include, for example, multiple disease episodes, hospitalizations, and injuries; the...

Data from individually-matched case-control studies, cohorts of twins and other paired designs provide a powerful resource that can be used estimate the magnitude of exposure-outcome associations free from confounding by shared factors. For binary outcomes, these data are typically analysed using conditional logistic regression (CLR),...

This talk describes two approaches used in economics to identify causal effects, and illustrates their use with applications drawn from health economics. The first approach described is the difference in difference estimator. This estimator is used widely in the economics literature that seeks to identify...

Spatial epidemiology is the description and analysis of geographically indexed health data with respect to demographic, environmental, behavioral, socioeconomic, genetic, and infectious risk factors. Common familiar regression models are not sufficient to analyse such data, as they do not account for inherent spatial correlation in...

Conservation is a key indicator of function in genomes, and can potentially be used to discover novel functional non‐protein‐coding RNAs and regulatory sequences. However, recent investigations have demonstrated that a simple dichotomy between conserved and non‐conserved sequence is too naïve a distinction to reflect the...

Normalization is a term that has come to describe a range of adjustments done to data prior to carrying out conventional statistical analyses.

In this seminar we present the distribution for standardized difference of means (SMD) estimators under the assumption that the data is sampled from a normal distribution which includes a normally distributed random effect component. This distribution, a rescaled non-central...

Recently there has been a number of drug safety concerns involving, for example, cyclooxygenase-2 (COX2) inhibitors such as Vioxx ® (rofecoxib) and Celebrex ® (celecoxib), erythropoiesis-stimulating agents such as Aranesp ® (darbepoetin alfa), Epogen ® (epoetin alfa) and Procrit ®...

Trajectory modelling provides a set of tools to analyse individual longitudinal data with a goal of yielding clusters of people who share 'similar' structure in the evolution of a variable of interest over time. In addition, the methods allow identification...

Variable selection is a common problem in regression modelling with a myriad of applications. This talk will present a new feature ranking algorithm (DEPTH) for variable selection in parametric regression based on permutation statistics and stability selection. DEPTH is: (i)...

Modern methods of psychometric analysis are model based. They can provide greater insight into the performance of items and scales than conventional, 'classical' approaches. This talk will introduce Item Response Theory (IRT) and variants such as the Rasch model....

Half of the world’s population is exposed to malaria, and with no vaccine for this disease, anti-malarial therapies are the first-line defence against malaria. Mechanistic within host models that characterize the relationship between the anti-malarial drug concentration...

CRXO designs are not uncommon in public health research and bounded discrete endpoints, such as pain scales, are common. The justification of sample size (i.e. the number of clusters, periods and individuals) is generally based on power calculations that...

Prof Ryan will discuss the use of Bayesian model averaging techniques for addressing uncertainty in how to adjust for covariates in the context of environmental risk...

Like many other labs around the world we are using next-generation sequencing data to identify disease causing variants, mainly for large effect size variants, such as those observed in single gene, or Mendelian disorders. Whilst this approach has delivered an avalanche of genes it also...

In joint work with Sue Finch, I draw on the seminal work of Edward Tufte and Bill Cleveland on excellence in statistical graphics to develop five simple principles for producing quality graphs. Our focus is on static graphics showing data, data summaries and inferences. We...

Stroke is one of the three most common causes of death around the world and the sixth most
common cause of disability worldwide. In this presentation we reflect on many facets of statistical and OR modelling for decision support in stroke care.

These include, but...

With the growing availability of large healthcare databases, non-experimental studies of prescription medications are becoming increasingly common.

However, appropriate design and analysis of such studies can be challenging. In this workshop we provide an intensive introduction to the field of pharmacoepidemiology.

We review...

Large non-experimental studies are increasingly used to evaluate the benefits and harms of medical interventions. One of the principal challenges in such studies is confounding - systematic differences between patients exposed to an intervention of interest versus the chosen comparator.

Recently, instrumental variable approaches...

Decisions about whether to subsidise a new pharmaceutical should ideally be informed by evidence that rates highly on two criteria: quality and relevance.

Typically the available evidence is of high quality (internally valid) because high-quality randomised controlled trials (RCTs) are mandated for marketing approval....

Disease risk prediction tools, often based on statistical regression models, are used for a variety of research and clinical purposes. They have a role in decision making for clinical treatment of patients, they can aid communication among patients, carers and treating health professionals, and they...

In the past 15 years there has been an explosion of analytical methodological development based on counterfactuals (potential outcomes) and utilisation of causal diagrams to understand and inform non-randomised comparisons in epidemiology and related areas.

These largely stem from the seminal works of Rubin...

The method of multiple imputation was first proposed by Donald Rubin in the late 1970's as an approach for dealing with nonresponse in large surveys.

He was particularly motivated by the requirement for U.S. federal agencies such as the Census Bureau to make national...

We describe selected artistic and statistical depictions of the force of mortality [hazard or mortality rate], a concept that has long pre-occupied actuaries, demographers and statisticians. We provide a more graphic form for the force of mortality function that makes the relationship between its constituents...

Patients with localized prostate cancer are frequently treated with radiation therapy. Following treatment, prostate-specific antigen (PSA) measurements are typically obtained at regular intervals for the purpose of monitoring and obtaining an early indication of disease recurrence.

In this talk I will present a statistical...