c4c-S offers a targeted range of services to support the paediatric trials. Our services are designed to facilitate the development of new drugs and therapies for the entire paediatric population by facilitating input from sites, experts, participants, and families, ensuring that the needs of babies, children, young people, and their families are met.
c4c-S also offers combined services that integrate Expert Advice with Trial Support services, providing a holistic approach to paediatric drug development.
Expert advice service
Our Expert Advice service provides impartial, high-quality advice on all aspects of paediatric drug development. This service aims to expedite the development of high-quality, feasible paediatric plans and protocols, ensuring patient-centred study designs, and speed up the conduct of feasible paediatric clinical trials, allowing access to well-studied drugs for paediatric patients.
With access to over 450 clinical and methodological paediatric experts in more than 25 medical specialties, and including Young Persons Advisory Groups and participants and parent groups, c4c-S brings diverse perspectives to paediatric drug development. More information on the therapeutic areas and/or indications can be found in our Expert Groups section.
Our areas of expertise include:
- Developing innovative study designs that are patient-centered (protocols, PDPs, PIPs).
- Designing and conducting post-marketing surveillance studies to monitor the long-term safety and effectiveness of approved treatments.
- Identifying and selecting optimal patient populations to ensure robust and relevant study outcomes.
- Evaluating and providing feedback on the feasibility and acceptability of study designs to ensure they meet scientific and regulatory standards.
- Defining meaningful outcomes and endpoints that accurately measure the effectiveness and safety of new treatments.
- Ensuring that studies are designed and conducted in alignment with the current standard of care, providing relevant and reliable data for decision-making.
- Preparing and responding to regulatory inquiries and submissions, ensuring compliance with regulatory requirements.
Our Expert Advice service includes full logistical support for organising advice meetings, focus groups, and patient-parent involvement activities. We ensure that all aspects of the advice process are managed efficiently and effectively.
Trial support services
c4c-S offers four distinct trial services, in collaboration with the National Hubs:
Exploratory outreach covers a clinical study that is planned or is open and provides information about barriers and facilitators to the conduct of that study. Topics include the number and location of potential participants including pathways of care between hospitals, input about current or anticipated challenges to trial conduct, and information about standards of care. The service can be provided at country level or site level (with national coordination of site contributions).
Key features include:
- Development of methods for exploratory outreach that are specific to each study in consultation with relevant c4c-S NHs.
- Assessments within c4c-S countries in which the study in scope is open or planned, relating to e.g., surveys about challenges with recruitment, the extent to which any changes in therapeutic landscape are influencing and will continue to influence study enrolment, and any actionable opportunities to influence recruitment.
- Development of a consolidated report that summarizes the key findings of the assessments, highlighting critical actions that can be undertaken with respect to increasing recruitment for the trial in scope, including identification of referral pathways or referring physicians.
Our site identification service encompasses a comprehensive database-driven search, supplemented with country intelligence via National Hubs to identify additional sites in Europe.
Key features include:
- Large site network: A database consisting of more than 220 high-quality, trial-ready sites across 21 countries (supported by our National Hubs).
- Country intelligence: Insights from National Hubs complement our site database-driven search and allow to assess trial-specific requirements.
Our site feasibility service assesses the trial-specific feasibility of each site quickly and reliably, supported by our National Hubs.
Key features include:
- CDA cascade approach: A unique and lean CDA cascade approach requiring only one sponsor signature.
- Harmonised feedback: Coordinated feedback approach to ensure consistent and reliable feasibility assessments across countries.
- Feasibility reports: Detailed feasibility reports with site engagement lists to inform site selection decisions.
Our study support service helps ensure that every study is set up correctly from the start, delivered on time and to the highest quality through close relationship of National Hubs and sites, insights on local complexities and regulations, and extensive specialised expertise.
Key features include:
- Study setup: Review of informed consent forms and other patient-facing documents, collection of key documents for site opening, management of regulatory and ethics submissions, and support to contract negotiations and budgets.
- Protocol implementation and study conduct: Support for protocol implementation, trial progress review, communication facilitation, and collection of metrics and KPIs.
- Recruitment support: Regular review of recruitment progress, support in recruitment planning, troubleshooting recruitment challenges, and collection of metrics and KPIs.
Empower your paediatric trials
By leveraging our extensive network of experts, trial-ready sites, and innovative methodologies, c4c-S is committed to advancing paediatric drug development and ensuring that well-studied drugs are accessible to paediatric patients across Europe. For more information or to request our services, please contact us.
Success stories
Bolstering patient recruitment at the national level
Background
Client’s local team was facing challenges with patient recruitment (6m – 18y) for a chronic kidney disease study in Spain, due to disengaged sites.
Our approach
- Spanish NH worked with the national sponsor representative to define points to raise with specific sites, focusing on study design concerns & applicable patient lists.
- Met with specific sites and PIs to discuss defined points, thereby understanding the reasons for disengagement and encouraging joint action for reengagement.
- Followed-up with specific sites and PIs to ensure continued engagement. Provided a platform for sites and PIs to flag new or persistent issues or concerns affecting the client’s study.
Key achievements
- Accelerated pre-screening of patients by specific PIs (within one week).
- Recruited an additional 7 Spanish patients within 12 months.
Quote sponsor
“In combination with support from the local team, the National Hub has assisted with motivating sites to pre-screen patients for our study. This has had an impact on recruitment, and we are grateful for the support that the National Hub has provided.”
– Sponsor Trial Team
Rapid site contracting and set-up
Background
Client needed support in setting up sites for a rare disease study (2 < 18y). Delays with protocol submission meant that some of the client’s confirmed sites were not able to take part in the study. Client asked c4c to support site identification, contracting and opening.
Our approach
- The National Hub was able to reach out to sites within its network and identified a suitable site to take part, which was previously unknown to the Sponsor.
- The National Hub worked with the sponsor’s local team and site to understand any blocks to site opening.
- Supported the contracting process, enabling the site to move forward.
Key achievements
- Accelerated pre-screening of patients by specific PIs (within one week).
- Recruited an additional 7 Spanish patients within 12 months.
Quote sponsor
“The c4c National Hub suggested the involvement of a site which was already part of their national network, but new to us. The site was contacted for the feasibility process and then selected to participate to the trial. We have been impressed with the engagement and the interest from the PI on the study and appreciated the recommendation from the National Hub.”
-Sponsor Country-Level Team
Rapid expert advice to support regulatory agency response
Background
Client (top 10 global pharma company) faced a tight deadline to respond to regulatory agency feedback. Client requested a quick turnaround in obtaining expert advice, as part of their regulatory agency response.
Our approach
- Leveraged our contracting structure, using existing Master Service Agreements with the client and with experts.
- Pulled from our dedicated expert network and database, ensuring fluent and rapid communication on client’s needs and timelines.
- Accelerated the expert advice process, thereby leveraging our close relationship with the experts.
Key achievements
- Provide advice within 8 weeks from the scoping interview, which is 1.5 x faster than c4c-S’ aim (12 weeks),
and 4.5 x faster than advice obtained without c4c-S (on average 36 weeks).
Expert advice to enable PIP modifications
Background
Client (top 10 global pharma company) needed to increase their study feasibility, ideally by reducing the number of study participants.
Our approach
- Leveraging our expert network, an advisory board was set up.
- Experts advised on reducing the sample size and extrapolating data from pharmacokinetics and safety studies in adults.
- Expert feedback was shared with the client, for use in proposing changes to the paediatric investigation plan (PIP).
Key achievements
- Advice provided on how to reduce the number of study participants, resulting in an improved study feasibility.
- Client shared our recommendations with the regulatory agency, resulting in a modified PIP.
Expert advice on paediatric practice
Background
Client (top 10 global pharma company) wanted to develop a multinational guidance tool on end-to-end instructions for a paediatric treatment intervention.
Our approach
- Co-created a questionnaire in line with the client’s request.
- Identified relevant experts, National Hubs and sites throughout Europe and across clinical areas, to complete questionnaire.
- Compiled insights for the client to use.
Key achievements
- Cross-functional and multinational view.
- Comprehensive overview and better understanding of the types and brands of materials used, and methods of administration of the intervention.
- Additional information gathered on the intervention that is useful for the future.
Partnerships
At c4c-S, we believe that strong partnerships and collaborations are essential to advancing paediatric drug development and ensuring that the needs of babies, children and young people are met.
Our partnerships include:
eYPAGnet
c4c-S places a strong emphasis on Patient and Public Involvement (PPI) to ensure that the needs and perspectives of patients and their families are integrated into paediatric clinical trials.
This involves engaging Young Persons Advisory Groups, patients, and parent groups in advice meetings and study designs. As such, we aim to create patient-centred studies that are more likely to meet the needs of the paediatric population and improve the overall quality and relevance of clinical trials.
Young Investigators Community (YIC)
The YIC is a key component of c4c-S’s collaborative efforts. This community provides a platform for early-career researchers to engage with experienced professionals, gain valuable insights, and contribute to paediatric drug development.
The YIC fosters mentorship, networking, and professional development opportunities, helping to shape the future leaders of paediatric clinical research.
Learned Societies
c4c-S collaborates with various learned societies to leverage their expertise and resources in paediatric drug development. These partnerships enable us to stay at the forefront of scientific advancements and best practices in the field.
By working with learned societies, we can ensure that their methodologies and approaches are informed by the latest research and expert advice, ultimately contributing to the development of high-quality clinical trials for children.
Projects
c4c-S is involved in various (European Commission-funded, public-private) projects, where it aims to share its knowledge and expertise in paediatric research and enhance paediatric drug development.
Develop Child and Orphan Device Evaluation support
The DeCODe project will develop a pivotal platform for paediatric and orphan medical devices including their development, stakeholders, initiatives, and critical pathway analyses.
European Rare Disease Research Alliance (ERDERA)
ERDERA brings together a broad range of high-value services, resources and cross-disciplinary expertise to support rare disease research projects across Europe, from planning and design to efficient execution. The alliance represents 180 organisations from 36 countries to help make Europe a world leader in rare diseases research and innovation.
CompRehensive mEthodological and operational Approach to cLinical trIalS in rarE and ultra-rare Diseases (RealiseD)
The RealiseD project aims to accelerate the development of treatments for rare and ultra-rare conditions by changing the paradigm of clinical trials in ultra-rare diseases. The consortium will develop innovative tools and leverage key collaborations with research networks to help achieve this goal.
PIPELINE project
PIPELINE is developing and implementing a world-class, multi-country adaptive clinical trials platform to support research on novel diagnostics, vaccines, and therapies for infectious diseases in pregnancy and infancy.
Cookie policy