c4c aims to enable hospitals and clinical sites across Europe to become ‘trial ready’ for paediatric clinical trials. We asked the team about the proof of viability studies that will determine if the network is working as planned
Many medicines that are given to children have never been tested in children, but thanks to an EU regulation that came into force in 2007, pharma companies are required to undertake mandatory research about safety and efficacy for drugs that are intended for use in paediatric populations.
The c4 project has put together a network of hospitals and clinical sites from across Europe that can undertake clinical trials for children, covering all paediatric age groups and diseases. They’re developing standards and procedures on the ‘how-to’ of successful trials with children, and training researchers and clinical trial site staff. The project has just embarked on a number of proof of viability (PoV) studies, where they use clinical trials to build and test the network. We spoke to the c4c project leadership team to get more details.
Q. What are the proof of viability (PoV) studies about?
A. 'The PoV studies aim to prove the viability of the c4c network. We would like to learn whether network processes, such as site identification and site feasibility, are working as planned. The studies will provide important information that will allow us to assess the performance and quality. The metrics will be supplemented by qualitative information (narratives, case studies) about what works and why, and how to improve the related processes.'
Q. You put out a call for clinical trials to be included in the PoV studies, and selected three studies from 57 expressions of interest – why were these ones chosen?
A. 'We were looking for studies from academic institutions which covered different specialty/therapeutic areas and study designs. Selection criteria included scientific value, operational feasibility and suitability for testing the c4c network. Despite extensive clinical need, funding for paediatric drug development is rare, and as such, the paediatric community, through the successful c4c-funded study teams, will benefit from funding for studies of high scientific standard that meet high clinical need and multiple ways to make their work easier.'
'These three academic-sponsored studies are funded by c4c, while another three PoV studies are being funded by the pharma industry. The latter are regular pharma industry studies performed with the support of the c4c network, using network processes and services. These studies are not part of the IMI action and not specifically designed for the IMI project, and are therefore best suited to test the c4c network under real-life conditions. Their study metrics will be used to evaluate the viability of the c4c network, supplemented by case studies.'
Q. What stage are those studies at?
A. 'All three non-industry studies will soon be recruiting participants – two have already started. The studies are testing the ability of the c4c network to provide information and advice that is not always available to sponsors (the organisation responsible for overseeing and/or funding the study). Investigators (the person/people responsible for carrying out the study at a site) sometimes find interaction with sponsors and contract research organisations (the organisation contracted by the sponsor to perform some of their duties) difficult. By allowing clinicians to talk with clinicians, the c4c network facilitates site identification, feasibility, and setup, including troubleshooting delayed ethics applications, site understanding of budgets and contracts, and trial procedures at site level.'
'For the industry studies, the first feasibility evaluations have been initiated for one study with the aim of recruiting the first patients into an industry PoV study during 2021.'
Q. You've set up advice groups. What are they for?
A. 'An important part of c4c is to provide expert advice to study sponsors on specific aspects of a paediatric development programme or study. Following a call for interest, experts were selected to join expert groups in clinical specialties or methodology. In addition, there is a c4c patient and public involvement database comprising individual parents and/or patients and patient organisations. The c4c network promotes the central role of participants in paediatric trials by supporting children, young people, and their families in activities, such as the review of study protocols, to ensure their opinions and needs are incorporated in the design and implementation of studies.'
'Depending on the nature of the request, appropriate clinical, methodology and patient/parent experts are brought together as ad-hoc advice groups in virtual meetings with the requestor. Meetings are taking place either virtually or face-to-face, as permitted by travel restrictions. During the course of the project, the advice activity is open to c4c beneficiaries, industry and non-industry. Several of the industry partners have already sought advice, with a number of companies returning with a second advice request.'
If you're interested in getting involved in the project, you can sign up to the c4c patient database.