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.
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