The EU-PEARL consortium (EU Patient-centric clinicAl tRial pLatforms), led by Vall d'Hebron Research Institute (VHIR) and Janssen, has presented an innovative framework that allows the development of collaborative platforms of assays (platform assays). These types of assays are becoming increasingly important in clinical research, as they bring efficiency and agility to drug development processes. The paradigm shift is that they are based on a single disease, instead of a single compound, several are evaluated, and they are not addressed until an effective treatment has been found.

The framework is the result of more than 3 years of work by EU-PEARL, a public-private strategic partnership funded by the Innovative Medicines Initiative (IMI). The consortium has been formed by 36 partners, including academic organisations, hospitals, pharmaceutical laboratories, European infrastructures, regulatory bodies and patient representatives.

Patient at the centre of the assessment

Together with patients, EU-PEARL has developed an innovative and generic methodology to facilitate the design, configuration and implementation of platform trials. These tools and resources cover statistical, regulatory and operational aspects and ensure that the design of the trial includes the most important assumptions for patients. All the changes are designed to implement a paradigm shift in the traditional way clinical trials work. The new philosophy, known as the Integrated Research Platform (IRP), places patients at the centre of the study.  With the IRP, new treatments developed by different companies and organisations to treat a single disease can be used simultaneously, sequentially and adaptively.

Dr. Joan Genescà, head of the Liver Diseases group at VHIR, clinical director of Digestive Diseases at Vall d'Hebron University Hospital and scientific coordinator of EU-PEARL, emphasises that "patient involvement has been fundamental to the project. We hope that the new framework will allow them to access new treatments more quickly and with less exposure to non-treatment groups".

The benefits for patients of these types of clinical trials include the fact that larger groups can participate and have access to several medicines while increasing the likelihood of receiving the treatment and not being part of the control group. In addition, if the treatment is unsuitable or discontinued, there is the option to switch groups.

In addition, the large number of participants and the parallel assessment of treatments streamlines data generation and development, as well as allowing for further subgroup analysis and individualisation of treatment options. And the large volume of data monitored with a single protocol increases the safety and reproducibility of trials. And with the internationalisation of the process, the patient will continue to have a trusted local point of contact in his or her language.

First active platforms

To demonstrate the viability of this innovative market, four collaborative trial platforms have been developed for four very different pathologies: major depressive disorder (mental health), multidrug-resistant tuberculosis (an infectious disease), non-alcoholic steatohepatitis or fungal liver disease (a growing non-communicable disease) and neurofibromatosis (a rare disease). Currently, these 4 platforms are in a position to start clinical trials depending on the possibility of finding adequate funding and the availability of the different drugs to be tested.

This experience has enabled the development of guidelines for the management, protection and interconnection of real data and templates to support the design and development of trial platforms within clinical and patient networks, optimised and standardised through electronic health records.