In a conventional clinical trial, typically half of the people participating in the trial receive the drug under investigation, and half receive a placebo. Trials may last years and cost a lot of money. In addition, if there are many trials in a disease area, it can be hard to find enough patients for all the trials taking place at a given time. For their part, patients struggle to find the right trial for their needs.
EU-PEARL aims to revolutionise the way we do clinical trials by making them more efficient and patient friendly. The project team will do this by setting up adaptive clinical trial platforms which allow multiple companies to test their candidate drugs simultaneously against a shared placebo group. Furthermore, in an adaptive platform trial with a single master protocol, new treatment groups can be added at any time, and candidate drugs that prove ineffective can be dropped. This will make it easier to enrol enough patients, while patients will benefit from an increased likelihood of being allocated a promising treatment instead of the placebo.
EU-PEARL will build the platform on a number of pillars. A clinical network of hospitals will provide the environment for the clinical trials and a place where different stakeholders can come together and interact. Patients are partners in the project and will contribute to the design of the platforms. EU-PEARL will also develop a comprehensive set of tools and methods for the planning, implementation and analysis of the trials. Finally, it will address regulatory issues as well as data governance.
EU-PEARL will focus its efforts on four disease areas: major depressive disorder, tuberculosis, the liver disease non-alcoholic steatohepatitis (NASH), and neurofibromatosis. However, the project outputs will make it possible to set up similar trial platforms in other disease areas in the future.