Developing efficient perpetual platform trials to study multiple treatments and multiple biomarkers

Clinical trials are used to test the effectiveness and safety of new treatments. In recent years they have become more and more expensive and have high rates of failure. New technology means that a large amount of biological information (summarised by measurements called biomarkers) can be measured. Often this information is likely to tell us how much a patient will benefit from being given a treatment. To make best use of this information, new approaches to clinical trials are needed. A new type of clinical trial design allows testing of the effectiveness of several new treatments simultaneously while considering that the effect may depend on biomarker measurements. A number of real trials are using this approach. Some of these trials will allow new treatments to be included as they become available. This has some benefits and makes the cost of developing and testing treatments lower. However the effect of doing this on important characteristics of the trial, such as the chance of incorrectly recommending a poor treatment for use in practice, is not well understood. We will work on ways of doing these trials in the most appropriate way. This will include getting a good understanding of how the trials work. It will also involve developing the best approaches to when new treatments should be added and when treatments in the trial should be recognised as being effective. Our team includes a wide range of expertise that will mean we can develop suitable methods and support their use in real trials. The developed methods will help patients on trials have a better chance of getting the most suitable new treatment. It will also mean better quality information comes from the trial and that future patients will be treated more effectively.