More than 5% of the UK population lives with an immune-mediated inflammatory disease (IMID) such as rheumatoid arthritis, Crohn’s disease, or psoriasis, yet the clinical trials meant to improve their treatment are often too expensive, too short, and poorly suited to rare or complex cases. This project develops better statistical methods to fix that. The team will design "basket trials" that test a single drug across multiple IMIDs sharing a biological pathway, allowing rare diseases to be studied without recruiting thousands of patients. They will also create adaptive trial designs that let patients switch to more effective treatments mid-study, and use existing patient data—from the IMID-Bio-UK cohort and NHS records in North-East England—to replace control arms or improve how trial results apply to under-served groups. If successful, the work will produce free software and training materials that make these methods routine. The immediate impact is on how trials are designed and interpreted; the long-term effect is that patients with chronic IMIDs will get answers about which treatment works best over years, not just weeks.
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Research question How can treatment for patients with immune-mediated inflammatory diseases (IMIDs) be improved through making trials and evaluations more efficient, informative and patient-centric? Background IMIDs are a group of conditions that share common inflammatory and immunity pathways. They affect >5% of the population and cause serious impact on patients and healthcare services. Clinical trials are essential for improving treatment of IMID patients but are expensive, often non-generalisable, and difficult to do for rare IMIDs. They often focus on short-term efficacy of a treatment and not how a chronic IMID should be treated over the long-term. To address these issues, there is a need for improved methods that are tailored to the nature of IMIDs together with overcoming barriers of their use in practice. Objectives 1. Develop innovative statistical methods tailored to IMID trials. 2. Develop methods for using additional sources of patient data to improve informativeness and generalisability of IMID trials. 3. Develop methods for identifying and evaluating the best long-term IMID treatment strategies. 4. Promote wide application of the new methods in practice. Methods Each objective links to an associated work package (WP). WP1: My team will develop statistical methods for improving the efficiency and patient-benefit of IMID trials. This includes methods for: better analysing complex trial outcomes that are used in many IMID settings; designing 'basket trials' that combine trials testing a drug that targets symptoms or biological mechanisms which are shared by several IMIDs, including rare ones; improved adaptive designs for chronic IMID settings that allow patients to switch to more effective treatments. These methods will be applied to several ongoing IMID trials. WP2: We will access cohort data from IMID-Bio-UK and routinely collected data from patients in North-East England. Methods will be developed for using these data to improve generalisability of IMID trials to under-served patient groups and patients with co-occurring autoimmune diseases. We will also investigate whether these other data sources can replace a control arm for rare IMID trials, thus reducing the sample size required. The developed methods will be applied to an ongoing trial in primary biliary cholangitis. WP3: We will consider how distinct sources of data can be used to identify the best long-term IMID treatment strategies. Methods will be developed to design an efficient and robust evaluation of these strategies. The developed methods will be illustrated using rheumatoid arthritis, with data available from cohorts, routinely collected data and trials. WP4: We will work with UK and international IMID clinical researchers to apply the developed methods in current and future trials. We will also develop free, high-quality, software and training materials to promote more widespread use of the developed methods. Anticipated impact Short-term: improved availability of better methods, and training of researchers in their use; proof-of-concept analyses of trials to improve interpretation of trial results; innovative design methods used in grant applications. Long-term: training materials and dissemination leading to widespread use of methods; changing practice leading to improved treatment of IMID patients.
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