This unit of study will focus on the strengths and weaknesses of different clinical study designs. Designs considered will include cohort (retrospective and prospective), cross-sectional, case-control and randomized controlled designs. The different phases of clinical trial designs in the development of therapies will also be examined including phase I (first in man), phase II/pilot and phase III comparative designs. Extension and adaptation of randomized designs will also be covered including cluster and factorial designs and adaptive pilot studies. Students will gain the skills necessary to choose between these designs for best practice. Types of outcomes (continuous, categorical, time-to-event) will be discussed. Methods of allocating participants to interventions (randomization), as well blinding and allocation concealment will be covered together with aspects of protocol development. On completion of this unit, the student will be familiar with the differences between study types and study designs, as well as the principles and practice of randomisation. It is also expected that the candidate will be able to develop stratified randomisation schemes for their own studies.

The candidate will develop the skills necessary to synthesize evidence both in preparation for conducting a trial and how to incorporate trial results into existing evidence. This will include being able to conduct a systematic review of the literature, including understanding how to appropriately assess evidence. The principles of meta-analysis to combine the results of multiple trials will also be examined as well as the interpretation of these results and how they can be used in clinical practice guideline development. As part of the critical appraisal of available evidence, different patient outcomes and the corresponding summary endpoint measures will be examined. Additionally the sources of biases arising from different trial designs and outcome measures will be covered and incorporated as part of the critical appraisal of available evidence (including published papers).

Statistical principles and concepts required to design clinical trials and analyse trial results will be introduced, including the appraisal of the appropriateness of analyses appearing in previous trial reports. Concepts which will be developed include an introduction to hypothesis testing, confidence interval estimation and understanding of univariable and adjusted analyses. Students will undertake analyses of study data where outcomes are continuous, binary and time-to-event variables Concepts and issues involved in performing landmark analyses and in identification of key prognostic variables and their interpretation in a clinical trials context will be introduced. The basis for and understanding of sample size calculations for clinical trials will be covered. Analyses will be performed using statistical software. SPSS software will be supported but students may use any package they are familiar with and have available. It is the student's responsibility to purchase the software. Details will be given at the beginning of the semester.

This unit of study will cover the principles of operationalizing a clinical trial. Students will acquire the knowledge needed to conduct quality studies providing research data and to comply with Australian regulatory and ethical requirements of a clinical trial. Areas covered will include single and multi-centre trials along with the roles and responsibilities of different trial personnel and committees. Clinical trial project management including thecosts of study resources and budgets, management of recruitment and follow up, and monitoring study progress will be covered, along with the ethical and legal framework governing clinical trials. This will also include aspects of ethics approval and patient consent in various patient populations. Aspects of quality assurance in clinical trials including documentation of study procedures, clinical trial audits, and study monitoring will be discussed. The development of clinical trials reports, results and publications will also be covered.

This unit addresses a number of key issues that arise in the analysis of clinical trial data. It will equip students with the ability to critically evaluate and interpret trial analyses, as well as provide them with an understanding of the principles underpinning good analysis practices. Modules will provide an introduction to the interpretation of treatment effect estimates, adjusted analyses, subgroup analysis, interim analyses, and how to reach appropriate decisions about the continued evaluation of an intervention, or its recommended implementation in practice.

The candidate will understand how to effectively form, lead and successfully manage a clinical research project. The subject will address issues related to resource management (including team and finance). The key elements of putting together a solid funding application and developing a study protocol will also be covered. Issues involved in study start up (initiation) and study outcome collection and review will be introduced. Aspects of scientific leadership including skills to address/solve problems in recruitment, follow up and event assessment will be taught. Students will gain a detailed understanding of how to plan for issues arising during a clinical trial in a practical sense including dealing with unexpected events during a trial, addressing event rates lower than that expected, changes in outcome definitions and ethical dilemmas. This is a key subject where students will integrate knowledge from prior studies and gain an understanding of how to apply theory to practice.

Candidates will be taught skills to design and interpret equivalence trials, non-inferiority trials and cluster randomised trials. Specialised designs including enrichment and discontinuation designs will be discussed and special aspects relating to cross-over studies will be taught. Techniques to validly incorporate composite, co-primary and surrogate endpoints will be covered. Distinctions between event and chronological time directed outcomes will be discussed. Skills to incorporate sub-studies into clinical research projects will be covered in this unit.

Identifying the right patients for the right drug at the right time is a major goal for improving patient care. Using biomarkers to help us personalise medicine is one strategy. The discovery of new biomarkers and the development of new biomarker-directed therapies raise high hopes for personalised medicine. Despite their promise, claims of clinical benefit of biomarker use still requires careful evaluation. This unit will teach candidates how to embed biomarker studies and translational research into clinical trial designs. The special skills and techniques of translational research will be highlighted in this unit. Candidates will understand the potential uses for biomarkers in clinical practice; how to use study designs relevant for biomarker evaluation and how to incorporate biomarker studies and biospecimen (tissue and blood) collection into clinical research projects. Candidates will learn the difference between prognostic, predictive and surrogate biomarkers and biostatistical considerations in their analysis. The complexities of international regulations regarding patient consent, biospecimen collection and shipment will be highlighted.

This unit of study provides a broad overview of the processes involved in translating a new drug, formulation and/or medical device from a laboratory setting to an approved product. It is targeted at people interested in or already working in the pharmaceutical or medical device industries, and advisors in the regulatory sector. Three core areas are covered: (1) the regulatory organisation, (2) requirements during drug discovery and device conception, manufacture and clinical trials, and (3) post-registration pharmacovigilance and pharmacoeconomics . Students will gain knowledge of the Therapeutic Goods Administration (TGA) and guidelines for the registration and regulation of medical devices and medicines. Students will also learn the importance of international regulations, harmonisation and application to the Australian market. The unit covers R and D; manufacturing and clinical trial requirements; the concepts of good laboratory and manufacturing practices (GMP, GLP) and quality by design (QbD); as well as regulator accepted laboratory methodologies used for submission of product dossiers and medical device documentation. The basics of clinical trial design will be analysed, as well as concepts of pharmacokinetics, dynamics, pharmacoeconomics and clinical endpoints for registration of new products using case studies and online tutorials. Special requirements for the registration and testing of generic medicines will also be part of the unit.

This unit of study develops knowledge in current state­ of ­the ­art therapeutic technologies. The principles of mode of action are investigated, along with methods of manufacture and registration. The unit is targeted at people already in or interested in the pharmaceutical and medical devices industries, and advisors in the regulatory sector. It covers 4 core areas of regulation in Australia: (1) biologicals and personalised medicine, (2) cell based products, (3) medical devices and (4) classical formulations. The principles that underpin these innovative therapies are covered in terms of development, targeting and manufacture along with the application of genomics in personalised medicine. Classical formulations (i.e. oral, respiratory and injectable dosage forms) will be covered and advances within the field such as regulation of nanotechnology will also be discussed. Students will investigate the processes of manufacture, verification and validation testing to ensure regulations are met. The emerging area of cellular immunotherapy for cancer treatment will be discussed. Students will gain knowledge of the different types of therapies within this space. Case studies will be evaluated, including the challenges associated with bringing these therapies and devices to market.
Classical formulations (i.e. oral, respiratory and injectable dosage forms) will be covered and advances within the field such as regulation of nanotechnology will also be discussed.

The capstone unit is a project which requires each student to bring together concepts, principles and applications developed in the previous units of coursework study into a workable research proposal and plan for the initiation and management of a clinical trial. The capstone will test the student's ability to integrate and consolidate their learning.