# MSc Model-based Drug Development

Year of entry: 2023

## Course unit details:Biostatistical concepts in clinical trials

Unit code PHAR69931 15 FHEQ level 7 – master's degree or fourth year of an integrated master's degree Semester 1 Pharmacy No

### Overview

This unit is designed to give the student an understanding of fundamental statistical concepts that are routinely used in designing clinical trials or interpreting trial results.

Emphasis is given to the use of simple hands-on exercises to build confidence in the application of concepts and in the practical use of software for statistical problem solving.

This unit is a prerequisite for the subsequent unit (PHAR69923) on advanced approaches to data analysis, including mixed effects models and population pharmacokinetics.

Theoretical knowledge will be disseminated first by lecture, but the unit will emphasise the application of theory by solving problems in workshops and coursework assignments. Some of the assignments will be computer based, to ensure students are comfortable with basic software for data analysis and presentation before encountering more specialised software in subsequent units.

Students will complete exercises using Microsoft Excel and will also be introduced to more specialised software such as R and Matlab

### Aims

The unit aims to:

• Introduce statistical concepts applied in the design of clinical trials and interpretation of clinical results in drug development.
• Provide practice in applying statistical methods to typical clinical trial data.

### Syllabus

Basic statistical concepts and definitions

Types of data

Probability concepts

Estimation

Confidence intervals

Hypothesis testing/ significance testing

Trial design

Choice of variables

Control groups

Randomisation

Blinding

Sample size and power

Statistical  analysis methods

Types of variables

Time to event data

Statistical analysis issues

The statistical analysis plan (SAP) in clinical research

### Knowledge and understanding

Students should be able to:

• Define and apply the statistical terms relevant to clinical trial data collection and interpretation.
• Explain the crucial roles biostatistics in guiding the design and conduct of clinical trials.

### Intellectual skills

Students should be able to:

• Apply basic biostatistical concepts and interpret statistical information arising from clinical trials.
• Perform power calculations and apply them in the design of a clinical trial
• Understand the statistical terms presented in clinical trial documents

### Practical skills

Students should be able to:

• Use computer software such as Excel or R to perform basic computational tasks including file input/output, calculations, and production of effective graphs

### Transferable skills and personal qualities

Students should be able to:

• independent learning, mastering new software

### Assessment methods

Method Weight
Other 60%
Written exam 40%

Biostatistics problems x 3 coursework - online - 60% of unit mark

Formative assessments x 4 - Model answers presented in tutorial - marks do not contribute to unit mark

ICH E9 Statistical Principles for clinical trials.  ICH E9 Statistical Principles for clinical trials.

Senn S. Statistical Issues in Drug Development. John Wiley & Sons. Chichester. 1997

Armitage P, Berry, G, Mathews JNS. Statistical Methods in Medical Research. Blackwell Publishing, Oxford. 2002.

BMJ statistics notes. (Altman et al.)

### Study hours

Scheduled activity hours
Lectures 10
Seminars 20
Tutorials 4
Independent study hours
Independent study 116

### Teaching staff

Staff member Role
Leon Aarons Unit coordinator