Optimizing expert and patient input in pediatric trial design: Lessons learned and recommendations from a collaboration between conect4children and European Patient-CEntric ClinicAl TRial PLatforms

Advice from multiple stakeholders is required to design the optimal pediatric clinical trial. We present recommendations for acquiring advice from trial experts and patients/caregivers, derived from advice meetings that were performed through a collaboration of the Collaborative Network for European Clinical Trials for Children (c4c) and the European Patient-CEntric ClinicAl TRial PLatforms (EU-PEARL). Three advice…

c4c General Assembly

Between 13th-15th June 2023, 165 c4c beneficiaries met in Newcastle, UK for the c4c General Assembly. This meeting has been a great opportunity for colleagues to meet again (or for the first time!) and to collaborate further for the benefit of the c4c network. The focus of this meeting was to provide an update on…

Solve-RD Project

Within the Solve-RD project, EURORDIS-Rare Diseases Europe, initiated the Community Engagement Task Force (CETF) – a multi-stakeholder community of patients, scientists and clinicians to support the needs of undiagnosed and recently diagnosed patients and leave a legacy of a strengthened undiagnosed community. This task force has created an infographic setting out the patient journey to…

c4c team: Sabah Attar

Meet the team behind c4c! We are a diverse group of people from many different backgrounds, but all of us are working towards a common goal: better medicines for children through European clinical trials. Today’s spotlight is on Sabah Attar, from the University of Liverpool.

The flood of study feasibilities and the value of a centralised approach

Due to an increase in multicentric paediatric clinical trials after 2007 following the EU Paediatric Regulation, there has been a substantial increase in the number of feasibility questionnaires (FQ). Paediatric clinical trial conduct has made unique advances through the conect4children (c4c) pan-European network, funded by the Innovative Medicines Initiative (IMI2). We examined the role of…

Paediatric clinical trial needs and requirements within Belgium

Due to the Paediatric Regulation in 2007, the number of paediatric clinical trials within Europe has substantially increased. Consequently, potential sites for paediatric clinical trials were overrun by trial opportunities, infrastructure pressure and their limited experience. To support sites and facilitate research, the pan-European network conect4children (c4c) was established and funded by the Innovative Medicines…

Collaboration in neonatal and paediatric clinical pharmacology: involving medical students within (Inter)national clinical trial networks

Between 50 and 96% of drugs prescribed to neonatal and paediatric populations are used off-label, creating a clear need for clinical pharmacology studies and subsequent labelling. Key to the successful future of paediatric drug development is creating a collaborative community of professionals focusing on neonatal/paediatric clinical pharmacology. Offering the opportunity for students to participate in…

New living evidence resource of human and non-human studies for early intervention and research prioritisation in anxiety, depression and psychosis

In anxiety, depression and psychosis, there has been frustratingly slow progress in developing novel therapies that make a substantial difference in practice, as well as in predicting which treatments will work for whom and in what contexts. To intervene early in the process and deliver optimal care to patients, we need to understand the underlying…

The future of child and adolescent clinical psychopharmacology: A systematic review of phase 2, 3, or 4 randomized controlled trials of pharmacologic agents without regulatory approval or for unapproved indications

We aimed to identify promising novel medications for child and adolescent mental health problems. We systematically searched https://clinicaltrials.gov/ and https://www.clinicaltrialsregister.eu/ (from 01/01/2010-08/23/2022) for phase 2 or 3 randomized controlled trials (RCTs) of medications without regulatory approval in the US, Europe or Asia, including also RCTs of dietary interventions/probiotics. Additionally, we searched phase 4 RCTs of…