New medical treatments, for example drugs, operations, courses of therapy and many other interventions, are usually put through a series of rigorous trials before they are allowed to be used on the general population. These trials are usually randomized controlled trials, designed in such a way that the trial provides evidence of causality; that is, that the trial can conclude whether or not the intervention had an effect on the condition it is targetting. Controlled means that some participants are allocated to receive a control intervention (usually the standard current treatment) and randomized indicates that the method used to allocate participants to either the control group or the intervention group should be inherently random and unpredictable.

One of the first documented clinical trials was the investigation by James Lind into treatments for scurvy, aboard the HMS Salisbury in 1747.

However since then, the field has progressed enormously in sophistication and rigour.

Statisticians are crucial to the effective design and analysis of clinical trials. In the design phase, when the trial is being planned, we will be tasked with questions such as:

• How many participants should we recruit?
• How should we assign paricipants to the different trial groups?
• How should we take into account the varying characteristics of the participants?

Once the trial has finished, and the data have all been collected, we use statistical analysis to learn about the all-important treatment effect, and to answer the question "Does this intervention work?".

There are many possible routes this project could take, for example:

• Focussing on a particular type of outcome measurement, for example binary or time-to-event data
• Looking at a particular class of statistical model, such as mixed-effects models
• Contrasting the Bayesian and Frequentist ways of approaching the design and analysis of clinical trials
• Investigating more advanced trial designs, such as cluster randomized trials or adaptive trials
• Handling common issues in clinical trials, such as missing data

The statistical aspects of clinical trials are inextricably tied to the practical and clinical ones, and throughout the design and analysis of a trial one must always be mindful of the ethical and practical implications and considerations. This grounding in the real world makes the subject challenging and rich, and this project would be hugely beneficial to someone considering a career in applied statistics (clinical or non-clinical).

Contact me

Back