All treatments have both benefits and risks which need to be considered when clinicians and patients are making treatment decisions. Randomised controlled trials are considered the best method to gather evidence about treatments to base these decisions on however outcomes are usually considered individually, ignoring the potential trade-off between them. For example, a new treatment may improve the management of a disease but increase the side effects. Patients want a treatment to work but not at the price of poor quality of life so a trade-off must be made, and the recommended treatment depends on this trade-off. Benefit-risk assessment are methods that can be used to compare treatments based on two or more outcomes and assess the trade-off between them whilst also including patient preference, which is key in treatment decision making. Using these methods in RCTs can ensure the best evidence is being considered for the treatments recommended within the NHS. A project was completed to better understand when researchers should consider using a benefit-risk method within clinical trials. Here, the transparency and consistency of research results were considered important and improved by using these methods, particularly when multiple outcomes are being evaluated and a subjective trade-off might be made. Additionally, the need to combine multiple outcomes into one would be a key reason for using these methods.

Nikki Totton is a clinical trial statistician currently on an ESRC Doctoral Fellowship researching the potential for benefit-risk methods to be used in publicly funded clinical trials. She has experience of a broad range of trial designs as well as other study designs. Additionally, she is a quantitative advisor within the Research Design Service.