c4c and EU-PEARL – Working Together on patient-centric trial design

In a conventional clinical trial, adult or paediatric, typically half of the participants receive the drug under investigation and half receive placebo or standard of care. 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. Trials may not be designed to meet patient needs, and when a trial is studying a rare disease, it can be challenging to recruit enough patients. conect4children (c4c) and EU-PEARL are IMI2 projects that aim to make clinical trials more efficient and patient-friendly. Therefore, we are excited to announce that both consortia have now started discussions to explore avenues for collaboration.

What is EU-PEARL?

EU-PEARL is an IMI2 project that aims to transform the way clinical trials are designed by creating generic frameworks to allow more efficient and patient-friendly trials designs. This EU-PEARL framework will enable the set-up of Integrated Research Platforms (IRPs) that consist of platform trials in which multiple intervention owners can test their new medicines simultaneously against a shared placebo or standard of care group. These types of trials will use a single master protocol that allows adaptive trial designs in which new treatment groups can be added at any time and in which compounds that are found to be ineffective or unsafe can be dropped. This maximizes enrolment capacity and patients will benefit from an increased likelihood of being allocated a promising treatment rather than placebo. EU-PEARL will provide foundational elements for how to build an IRP (such as site networks, data infrastructure, legal framework, operational tools) and drive the scientific and regulatory debate around design and methodology of platform protocols.

EU-PEARL aims to transform the way clinical trials are designed by creating generic frameworks to allow more efficient and patient-friendly trials designs.

What is c4c?

c4c is an IMI2 project that aims to build a paediatric clinical trial network across Europe to facilitate the design and conduct of paediatric trials. Due to the implementation of the European Paediatric Regulation in 2007, the number of clinical studies for new drug approval, the provision of additional information for drug labels in children, and the number of children involved in such studies, have increased steadily in the EU. The 2017 10-year review of the EU Paediatric Regulation recognised a significant increase in the number of planned or ongoing paediatric studies, designed to support a paediatric indication. However, several barriers were identified in the conduct and completion of paediatric trials, and the clinical trial infrastructure did not develop as quickly or consistently across Europe. This led to delays in completing clinical trials and often delays in the availability of medicines approved for children. The overall c4c vision is to create a network with a central coordinating organisation, arranged around National Hub centres at the country level working with multiple well-trained sites within a country, and to provide advice to sponsors on how best to design paediatric clinical trials through expert advice.

c4c aims to build a paediatric clinical trial network across Europe to facilitate the design and conduct of paediatric trials.

Potential synergies between both consortia and ways to collaborate

With the shared aim to improve clinical trials, members of the two projects have now started discussions to share their scope and evaluate potential synergies and ways to collaborate.
Amongst the 4 conditions included in EU-PEARL, Neurofibromatosis (NF) is included as prototype for rare and paediatric diseases. Hence, the Work Package 7 NF working group has been selected as the disease area for initial discussions between the two consortia.

With the shared aim to improve clinical trials, members of the two projects have now started discussions to share their scope and evaluate potential synergies and ways to collaborate.

Representatives from the EU-PEARL WP7 and c4c WP4 (the work package providing scientific advice) will explore whether they can collaborate on how to improve the involvement of children in the development and design of the EU-PEARL IRP master protocol for NF. In addition, the Patients and Public Involvement (PPI) groups from both consortia have also indicated their interest to explore potential synergies. Other synergies between the two projects are still to be explored, and include leveraging each other’s networks and sites, and the possibility of placing IRPs, adapted to paediatric specificity, in the successor c4c organisation (following the completion of the IMI2 funding).

As EU-PEARL and c4c have identified the above potential synergies to collaborate on, more news will come your way in the (near) future. Keep up to date via our social media channels!

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