EU-PEARL logo

FACTS & FIGURES

Start Date
End Date
Call
IMI2 - Call 15
Grant agreement number
853966

Type of Action: 
RIA (Research and Innovation Action)

Contributions
IMI Funding
12 004 953
EFPIA in kind
11 342 796
Associated Partners
2 307 562
Total Cost
25 655 310

Summary

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.

Achievements & News

Researcher wins prestigious award for platform trial simulation study
October 2021

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 scale of the funding enables real impact’ – IMI Associated Partner the Children’s Tumor Foundation
May 2021

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 WHO’s COVID-19 clinical trial is ‘adaptive’. What does that mean?
November 2020

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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Participants Show participants on map

EFPIA companies
  • Abbvie Inc, North Chicago, Illinois, United States
  • Astrazeneca AB, Södertälje, Sweden
  • Institut De Recherches Internationales Servier Iris, Suresnes, France
  • Janssen Pharmaceutica Nv, Beerse, Belgium
  • Novartis Pharma AG, Basel, Switzerland
  • Novo Nordisk A/S, Bagsvaerd, Denmark
  • Otsuka Novel Products GMBH, Munich, Germany
  • Pfizer Limited, Sandwich, Kent , United Kingdom
  • Sanofi-Aventis Recherche & Developpement, Chilly Mazarin, France
  • Teva Pharmaceutical Industries Limited, Netanya, Israel
Universities, research organisations, public bodies, non-profit groups
  • Assistance Publique Hopitaux De Paris, Paris, France
  • Bundesinstitut Fur Impfstoffe Und Biomedizinische Arzneimittel, Langen, Germany
  • Charite - Universitaetsmedizin Berlin, Berlin, Germany
  • Eatris Eric, Amsterdam, Netherlands
  • Ecrin European Clinical Research Infrastructure Network, Paris, France
  • Erasmus Universitair Medisch Centrum Rotterdam, Rotterdam, Netherlands
  • Euroscan International Network Ev, Koln, Germany
  • Forum Des Patients Europeens, 1040, Belgium
  • Fundacio Hospital Universitari Vall D'Hebron - Institut De Recerca, Barcelona, Spain
  • Katholieke Universiteit Leuven, Leuven, Belgium
  • King'S College London, London, United Kingdom
  • Ludwig-Maximilians-Universitaet Muenchen, Munich, Germany
  • Medizinische Universitaet Wien, Vienna, Austria
  • The European Institute For Innovation Through Health Data, Gent, Belgium
  • The University Of Manchester, Manchester, United Kingdom
  • Universita Degli Studi Di Milano, Milano, Italy
  • Universita Vita-Salute San Raffaele, Milano, Italy
  • University Of Newcastle Upon Tyne, Newcastle upon Tyne, United Kingdom
  • University of Oxford, Oxford, United Kingdom
Small and medium-sized enterprises (SMEs) and mid-sized companies (<€500 m turnover)
  • Berry Consultants Llp, London, United Kingdom
  • Documental Ou, Tallinn, Estonia
  • Information Technology For Translational Medicine (Ittm) SA, Esch-sur-Alzette, Luxembourg
  • Team - It Research SL, Barcelona, Spain
Associated partners
  • Children'S Tumor Foundation, New York, United States
  • Global Alliance For Tb Drug Development Non Profit Organisation, New York, United States
  • Springworks Therapeutics Inc, Stamford, United States
Third parties
  • European Aids Treatment Group Ev, Duesseldorf, Germany
  • Inst Cardiometabolisme Nutrition Ican, Paris, France
Project leader
Ann Van Dessel
Janssen Pharmaceutica Nv