Periodic Reporting for period 5 - c4c (conect4children (COllaborative Network for European Clinical Trials For Children))
Période du rapport: 2022-05-01 au 2023-04-30
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. 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 (SPoC) for all Sponsors, sites and investigators; b) efficient implementation of trials adopting consistent approaches, aligned quality standards and coordination of sites; c) collaboration with specialist networks; d) high-quality input to study design and preparation through rigorous strategic and operational feasibility assessment.
c4c procedures and processes were developed to prepare the future network: a SPoC, an information system IT solution with SPoC functionality and SPoC database search of sites; National Hub (NH) trial readiness as national coordinators of sites; NHs to oversee trial-related site activity; Network Infrastructure Office and its processes to serve NHs, sites and trial teams; SOPs for trial implementation and c4c processes to support trials; site feasibility processes for industry-sponsored studies; a CDA cascade process allowing fast and efficient exchange of confidential information between Sponsor and SPoC, NHs and sites during site feasibility, study start-up and conduct; processes for Sponsors to seek expert Strategic Feasibility Advice (SFA) and centralized contracting procedure for SFA (54 requests received, 34 requests delivered); recruitment and onboarding of 400+ experts in 24 Expert Groups (16 Clinical, 8 Methodology) and Patients and Public Involvement (PPI) database to contribute to the SFA. The expert groups also ensure integration of c4c into the wider paediatric community; a cross-cutting paediatric data dictionary supporting harmonization and standardization of data commonly collected in paediatric clinical trials; performance metrics with specified targets and ranges for paediatric trials and processes to monitor performance of NHs and sites during study conduct; e-learning platform modules for site staff training (40 courses available on the c4c Academy platform); processes and structure to organize and coordinate paediatric Multi-Stakeholder Meetings (MSM) including regulatory agencies, academic experts, clinicians, paediatricians and industry representatives to discuss scientific and/or regulatory questions for a given indication or a class of compounds.
The focus in the fifth project year was to progress patient recruitment into the 2 non-industry proof of viability (PoV) studies; test site identification and feasibility processes for the industry-PoV studies; continue testing the SFA process; establish conect4children Stichting, a Dutch non-profit organisation, aiming to sustain the work done by the public-private consortium; organize the 3rd paediatric MSM on medical devices for children with Type 1 diabetes with the goal to bring together key stakeholders and opinion leaders in the field, raise and discuss the challenges and define potential next steps. The Paediatrics User Guide is now freely available on the CDISC website, to facilitate the development of medicines for children. The User Guide focuses on cross-cutting clinical concepts related to paediatric research and describes how to use CDISC standards to collect and structure data used in clinical trials to facilitate the aggregation of information, take advantage of big data and support data sharing. Adopting CDISC standards is an invaluable investment that leads to more meaningful, more efficient research by streamlining the flow of data throughout the research process. To know more about the achievements, please visit the c4c website.
The focus in the fifth project year was to progress patient recruitment into the 2 non-industry proof of viability (PoV) studies; test site identification and feasibility processes for the industry-PoV studies; continue testing the SFA process; establish conect4children Stichting, a Dutch non-profit organisation, aiming to sustain the work done by the public-private consortium; organize the 3rd paediatric MSM on medical devices for children with Type 1 diabetes with the goal to bring together key stakeholders and opinion leaders in the field, raise and discuss the challenges and define potential next steps. The Paediatrics User Guide is now freely available on the CDISC website, to facilitate the development of medicines for children. The User Guide focuses on cross-cutting clinical concepts related to paediatric research and describes how to use CDISC standards to collect and structure data used in clinical trials to facilitate the aggregation of information, take advantage of big data and support data sharing. Adopting CDISC standards is an invaluable investment that leads to more meaningful, more efficient research by streamlining the flow of data throughout the research process. To know more about the achievements, please visit the c4c website.
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 remains 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.
c4c aims to positively impact the number of new, innovative medicines developed and ultimately accessible to children by increasing the number of qualified clinical sites that work in a coordinated way to manage Paediatric clinical trials efficiently and at high standard. With the inclusion and training of about 240 clinical sites from 21 European countries, c4c has already created a novel degree of coordination that is independent of specific projects and individual Sponsors.. To further improve harmonization of site coordination across countries and quality of site operations, c4c has developed site standards and plans to pilot roll out to some sites. The SPoC and its links to national points of contact are ready to improve coordinated access to sites. Furthermore, driven by the feasibility requests related to the I-PoV studies, c4c is working together with speciality networks to find more capable sites and expand the c4c site database. To extend reach beyond Europe, c4c puts efforts into discussing possible ways to cooperate with clinical trial networks in other jurisdictions, having signed two-sided CDAs with two US and Canadian networks. c4c has started preliminary discussions with the European Joint Programme on Rare Diseases (EJP RD).
With close cooperation with the NHs, c4c has further refined a comprehensive set of network processes, SOPs and governance/quality documents to allow a network-wide, coordinated way to support set-up and conduct of clinical studies in all NHs and related sites. These processes, together with the standardized trainings modules generated and already rolled out to NHs and sites, led already to increased efficiency in the set-up of c4c-supported clinical studies.
A key aim is to promote the use of innovative methods in trial design and study planning by ensuring that the impact of patient/parents and clinical/methodological experts will increase. c4c has provided advice to additional studies from different specialties and from patients and parents to an increasing number of industry Beneficiaries, who have used this advice to improve study design and processes, and to support generation of regulatory documents and procedures. To further strengthen the input of young people and patients in the design of clinical studies, c4c PPI organized a session to test the “patient journey” related to one of the industry studies. c4c is currently in the process to recruit further interested young people and patients and parents to join the c4c Patient Involvement Expert database and to provide input to research proposals and/or study designs. All non-confidential deliverables are available on the c4c and CORDIS website.
c4c aims to positively impact the number of new, innovative medicines developed and ultimately accessible to children by increasing the number of qualified clinical sites that work in a coordinated way to manage Paediatric clinical trials efficiently and at high standard. With the inclusion and training of about 240 clinical sites from 21 European countries, c4c has already created a novel degree of coordination that is independent of specific projects and individual Sponsors.. To further improve harmonization of site coordination across countries and quality of site operations, c4c has developed site standards and plans to pilot roll out to some sites. The SPoC and its links to national points of contact are ready to improve coordinated access to sites. Furthermore, driven by the feasibility requests related to the I-PoV studies, c4c is working together with speciality networks to find more capable sites and expand the c4c site database. To extend reach beyond Europe, c4c puts efforts into discussing possible ways to cooperate with clinical trial networks in other jurisdictions, having signed two-sided CDAs with two US and Canadian networks. c4c has started preliminary discussions with the European Joint Programme on Rare Diseases (EJP RD).
With close cooperation with the NHs, c4c has further refined a comprehensive set of network processes, SOPs and governance/quality documents to allow a network-wide, coordinated way to support set-up and conduct of clinical studies in all NHs and related sites. These processes, together with the standardized trainings modules generated and already rolled out to NHs and sites, led already to increased efficiency in the set-up of c4c-supported clinical studies.
A key aim is to promote the use of innovative methods in trial design and study planning by ensuring that the impact of patient/parents and clinical/methodological experts will increase. c4c has provided advice to additional studies from different specialties and from patients and parents to an increasing number of industry Beneficiaries, who have used this advice to improve study design and processes, and to support generation of regulatory documents and procedures. To further strengthen the input of young people and patients in the design of clinical studies, c4c PPI organized a session to test the “patient journey” related to one of the industry studies. c4c is currently in the process to recruit further interested young people and patients and parents to join the c4c Patient Involvement Expert database and to provide input to research proposals and/or study designs. All non-confidential deliverables are available on the c4c and CORDIS website.