Report

Teaser, summary, work performed and final results

Periodic Reporting for period 1 - c4c (conect4children (COllaborative Network for European Clinical Trials For Children))

Teaser

Paediatric medicines development is of great strategic importance for Europe and is embedded in policy, legislation and the work of the pharmaceutical industry. Currently, the potential of this effort is not realised for a number of reasons, for example fragmentation and...

Summary

Paediatric medicines development is of great strategic importance for Europe and is embedded in policy, legislation and the work of the pharmaceutical industry. Currently, the potential of this effort is not realised for a number of reasons, for example fragmentation and redundancy of effort between sponsors, sites and countries.

The overall aim of conect4children (c4c) is to generate a sustainable infrastructure that optimises the delivery of clinical trials in children through:

a) a single point of contact for all sponsors, sites and investigators;
b) efficient implementation of trials adopting consistent approaches, aligned quality standards and coordination of sites at national and international level;
c) collaboration with specialist networks;
d) high quality input to study design and preparation through rigorous strategic and operational feasibility assessment and
e) the promotion of innovative methodologies.

c4c is focused on enhanced study delivery, through efficient implementation and high-quality design.

The network will join the European Network of Paediatric Research at the European Medicines Agency (EnprEMA) and collaborate with similar global paediatric clinical trial networks.

Work performed

During the first year of the project, c4c procedures were developed to prepare the future network structure and processes for:

• seeking expert Strategic Feasibility Advice for study design;
• a Single Point of Contact (SPoC) for all Sponsors;
• National Hub (NH) trial readiness as national coordinators of trial sites;
• NHs to oversee site activity related to trials;
• a training platform for short courses and training modules;
• integration of c4c into the wider paediatric community by including clinical and methodology experts and patients (children and young people)/parents’ associations to contribute to advice during the study development;
• harmonisation and standardization of data terms commonly collected in paediatric clinical trials.

A core set of metrics, specific to paediatric trials and network functions, was selected to monitor c4c performances at both network and site level. Performances will be measured during the execution of four non-industry and four industry-sponsored studies.

During this first year, the four non-industry proof-of-viability studies were selected and will be implemented to test the appropriateness of the set up procedures and the efficiency of the network.

Final results

The current European paediatric medicines development environment is fragmented, redundant and inefficient in terms of cost and time. Overcoming these barriers is one of the greatest challenges but also a huge opportunity for success for this project. c4c is still unique in aiming to overcome these obstacles by establishing trial-ready infrastructure, operational excellence and consolidated interaction and communication at European, national and site level.

By defining better processes and interactions, c4c has the chance to contribute to a faster access to medicines for children. By also embedding the voice of parents/patients, as well as innovative methodology experts from industry, c4c will form a critical mass able to influence changes in regulatory policies and overcoming these barriers.


First year project’s direct impact:


• ACCESS
With the creation and implementation of c4c NHs in 19 European countries, the number of clinical sites prepared to conduct paediatric studies has already expanded directly impacting on the availability of trial-ready sites in Europe.

• EFFICIENCY
While c4c is in the process to set-up internal processes and to create needed network structures and processes to increase the efficiency of conducting paediatric studies in Europe, this preparatory work has tested a process where sponsors and sites are cooperating, reducing time to find expertise to design studies and the sites to perform them. With the selection of the initial 4 non-industry studies, these processes were tested for the first time.

• INNOVATION
c4c is engaging with experts as well as patient/parent and Young Persons’ Advisory Groups (YPAGs) for providing scientific input to sponsors during the creation of their Paediatric Development Plan and/or for designing a study by promoting use of innovative approaches for trial design and analysis; and by this demonstrating the positive impact of their direct contribution during the early phase of study design.

Website & more info

More info: https://conect4children.org/.