## Medical Statistics

 Unit code: MATH38072 Credit Rating: 10 Unit level: Level 3 Teaching period(s): Semester 2 Offered by Division of Population Health, Health Services Research and Primary Care Available as a free choice unit?: N

Prerequisite

None

#### Aims

This course unit introduces the application of statistical ideas and methodology to medical research.

#### Overview

Randomised controlled trials are planned experimental studies on human subjects designed to assess the benefit of medical treatments. Other important areas of application of statistical methods in medical research are epidemiological studies, which investigate the possible causes of disease from observational data, diagnostic studies, which methods of disease diagnosis and meta-analysis, which considers combining information from multiple studies. Many of the major developments in modern statistics have been motivated by problems in medical research. Whilst briefly outlining other areas of application in medical research, the lecture course will introduce the statistical issue associated design and analysis of randomised controlled trials and in meta-analyses.

#### Learning outcomes

On successful completion of this course unit students will be able to:

1. Appraise the design, analysis and interpretation of the results of randomised controlled trials (RCTs), with specific reference to minimising bias.
2. Choose, with justification, the design, including calculation of the sample size, for a RCT with a continuous or binary outcome.
3. Analyse data from RCTs of various designs (including: parallel, crossover or cluster; superiority, non-inferiority or equivalence), including the meta-analysis of several RCTs, and interpret the findings in the relevant context.
4. Derive key mathematical expressions with applications to RCTs or meta-analyses of RCTs.

#### Assessment methods

• Other - 20%
• Written exam - 80%

#### Assessment Further Information

• Coursework: weighting 20%
• End of semester examination: two hours weighting 80%

#### Syllabus

• Introduction to medical statistics. Randomised controlled trials: historical background and ethical issues concerning randomised experimentation on human subjects.
• Design and organisation of randomised controlled trials. Types of bias and methods for controlling bias including blinding and placebo treatments.
• Sample size estimation for continuous and binary outcome measures.
• Methods of treatment allocation including simple randomization, random permuted blocks, stratification and minimization.
• Implications of equivalence and non-inferiority hypotheses for sample size and statistical analyses.
• Statistical methods for the analysis of parallel group trials including methods for the adjustment for baseline data.
• Implications of protocol deviations and the motivation for the intention-to-treat principle.
• Multiplicity issues: sub-group analysis and multiple outcomes.
• Alternatives designs for randomised controlled trials: cross-over trials and cluster randomised trials.
• Meta-analysis and publication bias.

#### Recommended reading

• Matthews, JNS, An Introduction to Randomized Controlled Clinical Trials, 2nd edition 2006, Chapman & Hall/CRCPress

The first edition (2000) is also adequate for this course and there are copies of both in the John Rylands Library

#### Feedback methods

Feedback tutorials will provide an opportunity for students' work to be discussed and provide feedback on their understanding.  Coursework or in-class tests (where applicable) also provide an opportunity for students to receive feedback.  Students can also get feedback on their understanding directly from the lecturer, for example during the lecturer's office hour.

#### Study hours

• Lectures - 22 hours
• Tutorials - 11 hours
• Independent study hours - 67 hours

#### Teaching staff

Chris Sutton - Unit coordinator