Randomised trials are getting a complete redesign to make them faster, cheaper, and able to answer several questions at once. The problem is that traditional trials are slow and expensive, often testing just one treatment against a placebo. This means patients wait years for answers, and funders spend huge sums on studies that could have been more efficient. The researchers are developing six new trial designs that fix these bottlenecks: trials that safely test shorter or milder treatments, trials that can drop failing treatments mid-study and add promising new ones, trials that work even when benefits are short-lived or delayed, trials that randomise groups rather than individuals, trials tailored to specific patient types, and trials where some patients cannot receive certain treatments. If this research succeeds, medical evidence will arrive years sooner. A single trial could replace what currently takes multiple sequential studies, cutting costs and getting effective treatments into clinics faster. The statistical models ensure these faster designs do not increase the risk of false conclusions. For the public, this means quicker access to better treatments and fewer resources wasted on inefficient studies.
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Randomised trials are the best way to find out what treatments work, but they can be slow and expensive. We are inventing new and efficient ways to design randomised trials that can give clear answers, often answering multiple questions within the same trial. This work is supported by detailed statistical modelling to show how to analyse the new designs while strictly controlling the risk of wrongly concluding that a treatment is effective when in truth it isn’t. The new designs are implemented in our own trials, and this helps us to refine the designs and demonstrate their value for other trials. Our novel designs are for 1. trials to evaluate whether it is safe to shorten the duration of treatment or use a treatment with fewer side effects; 2. trials to evaluate multiple treatments, where we can drop treatments that are found to be ineffective, adopt the use of treatments that are found to be effective, and start evaluating new promising treatments; 3. trials that work even when the benefit of treatment is short-term or delayed; 4. trials where groups of patients are randomised rather than individual patients; 5. trials specifically designed to evaluate treatments tailored to specific types of patient; and 6. trials comparing a number of treatments, where some patients cannot receive some of the treatments. The results of our work are that treatments can be evaluated faster, more effectively and more cost-effectively.
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