EU-PEARL

Elias Laurin Meyer, a scientist from IMI’s EU-PEARL project, has been awarded the Arthur-Linder-Prize for developing simulations that will help design platform trials. ### Meyer received the award for his paper ‘Decision rules for identifying combination therapies in open-entry, randomized controlled platform trials’. The goal of platform trials generally is to test as many investigational treatments as possible over the shortest duration. Meyer and his colleagues investigated platform trial design in the context of combination therapies, in which one of the two therapies a common, backbone monotherapy.

‘From a statistical point of view, platform trials constitute a new paradigm of clinical trial design, which in turn requires the development and/or re-calibration of statistical tools, since methods, software and quality criteria of more traditional trial designs might not be applicable,’ Meyer told the EU-PEARL project.

‘My contributions (in EU-PEARL) will be mainly in aiding development of new statistical methods and software for the simulation of complex platform trials. Ideally, the tools and software provided will aid clinical trial design and help shed light on the impact of certain design choices and assumptions on the operating characteristics of the trial. This, for example, includes the required number of patients in the trial or the required number of patients on placebo/control treatment arms.’

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The Children's Tumor Foundation is one of IMI's Associated Partners. In an interview with the IMI Programme Office, foundation president Annette Bakker says that the unique IMI model ensures alignment between industry and public partners.###

The Children’s Tumor Foundation is a renowned international non-profit organisation that seeks to advance research into a disease called neurofibromatosis, a genetic disorder of the nervous system. They are involved in EU-PEARL and also contributed to the work of PARADIGM. In addition, the organisation has been a member of the Strategic Governing Group (SGG) on oncology, which feeds topics to IMI.

Asked about the benefits of IMI, Dr Bakker says: ‘The scale of the funding enables real impact. Also the unique business model ensures alignment between industry and the public partners on what matters most. The commitment of all the stakeholders is also a great benefit.’

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The record for the fastest vaccine ever developed is about four years from lab to patient. The new coronavirus vaccines are set to smash this record. In some cases, it will be thanks to the use of adaptive trial platforms, which are trials that run studies of multiple drugs at the same time and ‘pool’ the placebo group.###

While adaptive trial design is extremely good at reducing drug development times, it is also very complex. That’s why in 2019, IMI launched EU-PEARL to help provide the evidence and support for those who want to design their own adaptive trials, with the ultimate aim of getting medicines more quickly to market.  

EU-PEARL’s partners include some of the world’s foremost experts in adaptive platform trials. These experts were consulted by the World Health Organisation during the design phase of the Solidarity trial, a study to test multiple COVID-19 treatment, diagnostic and prevention options, including vaccines. New treatment groups can be added at any time, where necessary, and candidate compounds that don’t work can be dropped.

Though the adaptive trial concept has existed for years, there is still lack of uniformity and understanding as to what an adaptive platform means and how it works in practice. ‘The process of designing and running these trials is incredibly complex,’ says EU-PEARL project lead Ann Van Dessel of Johnson & Johnson. ‘To support researchers who decide to take on the task, we are putting in place the tools that are necessary to make sure they succeed. The project will set up a new entity whose job it will be to help facilitate these very complex new studies, and to maintain the tools and guidelines developed during the project.’

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