Paediatric CRO Services | Complex and rare disease studies

PHARMExcel provides specialised Paediatric CRO Services for sponsors conducting clinical trials, including complex and rare disease studies, in infants, children and adolescents. We work with biotechs, medtech companies, charities and NHS or academic sponsors to help deliver paediatric studies that are scientifically robust, operationally realistic and aligned with the needs of children, families, investigators and specialist sites.

Paediatric clinical research introduces a distinct set of scientific, regulatory and ethical challenges that require more than a simple adaptation of adult study designs. Differences in developmental biology, drug metabolism, disease progression and safeguarding requirements mean that paediatric studies must be planned carefully from the clinical trial stage onwards. PHARMExcel supports sponsors with the governance, regulatory strategy and clinical operations needed to run these studies safely, ethically and in line with real-world paediatric care.

Regulatory strategy and paediatric development planning

Paediatric research is shaped by clearly defined regulatory obligations, particularly where sponsors need to align adult and paediatric clinical pathways. PHARMExcel supports sponsors with the regulatory planning needed to move paediatric studies into and through the clinical trial stage, including input around Initial Paediatric Study Plans (iPSPs), age cohorts, study sequencing and wider paediatric obligations.

Our teams help sponsors prepare regulatory documentation, support ethics and authority submissions and structure clinical programmes in a way that is both compliant and workable in practice. By embedding paediatric regulatory planning early in the clinical trial pathway, we help reduce delays, avoid unnecessary amendments and ensure that paediatric requirements are addressed in a proportionate and practical way.

Study design and age-appropriate paediatric research

Designing clinical trials for children requires a different approach from adult studies. Paediatric populations span multiple developmental stages, each with distinct physiological, behavioural and clinical characteristics that can affect dosing, endpoint selection, safety monitoring and follow-up requirements. Neonates, infants, children and adolescents cannot be treated as a single group, and trial design must reflect these differences.

PHARMExcel works with sponsors and investigators to help shape protocols that are appropriate to the age group, disease area and clinical context of the study. This includes careful consideration of assessment burden, visit frequency, formulation acceptability, age-stratified cohorts and the feasibility of collecting reliable data in routine paediatric settings. Our role is to help ensure that studies remain scientifically robust while also being practical for children, families and specialist sites.

Family-centred recruitment, retention and trial delivery

Successful paediatric trials depend not only on the child or young person, but also on parents, carers and wider family routines. Recruitment and retention are therefore closely linked to trust, communication and the practical burden of taking part. Travel, school attendance, work schedules, anxiety around procedures and the emotional weight of clinical decision-making can all affect whether a family is able or willing to remain engaged in a study.

PHARMExcel does not directly recruit patients. Instead, we provide governance and oversight of site-led enrolment by helping sponsors identify suitable paediatric centres, assess realistic referral pathways and work with investigators who understand the local patient population and family context. We also help sponsors and sites think carefully about child-friendly procedures, visit burden and the clarity of family-facing materials, so that studies remain acceptable and deliverable over time.

Specialised paediatric expertise and clinical operations

Paediatric trials require site teams and operational models that reflect the realities of working with children in clinical settings. PHARMExcel provides governance and oversight across study start-up, site selection, monitoring, project management and close out, helping sponsors align their study with the right children’s hospitals, paediatric departments and specialist investigators.

Our teams focus on ensuring that trial processes are clearly understood by sites and adapted to age-specific needs, particularly where studies involve repeated assessments, long-term follow-up, safety monitoring or communication with families and carers. As a smaller, boutique CRO, we are able to provide the flexibility, continuity and personal service that larger organisations often struggle to match, which is particularly valuable in paediatric research where responsiveness and careful coordination matter at every stage.

Ethical oversight and developmental considerations

Ethical oversight is central to paediatric research. Clinical trials involving children must meet high standards of patient protection while still generating meaningful evidence for populations that are often underserved by clinical research. This means careful attention to parental consent, child assent where appropriate, safeguarding, minimising discomfort and ensuring that procedures are justified and proportionate.

PHARMExcel supports sponsors and sites with the governance and oversight needed to manage these responsibilities properly. We recognise that developmental biology plays a major role in treatment response and that children are not simply smaller adults. Clinical trial processes therefore need to account for growth, maturation, behavioural differences and long-term developmental considerations in a way that is both scientifically credible and ethically sound.

Clinical trials across neonatal, infant and adolescent populations

Paediatric studies can vary significantly depending on the age group involved. Infant trials may involve particularly close safety oversight, complex consent considerations and specialist hospital environments. Trials in children and adolescents may bring different challenges, including school disruption, age-appropriate communication, assent processes and long-term follow-up into later developmental stages.

PHARMExcel helps sponsors plan studies that reflect these differences, with governance and operational oversight tailored to the specific patient population and care setting. Whether a trial is focused on early safety, dose evaluation, efficacy or longer-term follow-up, we help ensure that the study design is feasible, well governed and aligned with the clinical environment in which it will be delivered.

Partner with PHARMExcel for paediatric CRO services

PHARMExcel brings together regulatory expertise, practical clinical operations and a quality-driven approach to paediatric trial governance. Sponsors choose us because we offer direct access to experienced senior staff, continuity of the same core team and a more personalised way of working than many larger CROs can provide.

Our aim is to reduce operational burden, support regulatory readiness and help sponsors deliver paediatric clinical trials that are safe, ethical and feasible in practice as well as robust on paper. For organisations looking for a collaborative and responsive CRO partner in paediatric research, PHARMExcel provides practical expertise grounded in real-world clinical trial delivery.

Paediatrics CRO FAQs

What are Paediatric Investigation Plans (PIPs) and why are they important?

Paediatric Investigation Plans are regulatory documents used to define how a medicine will be studied in children, including age groups, study design and timelines. They are an important part of paediatric regulatory planning and help ensure that data in children are generated in a structured and responsible way.

How are paediatric clinical trials different from adult clinical trials?

Paediatric trials must account for developmental biology, age-specific physiology, safeguarding requirements and the need for parental consent and child assent where appropriate. They also need to minimise burden on children and families while still collecting reliable and clinically meaningful data.

Why are age-appropriate study designs important in paediatric research?

Children at different developmental stages may respond differently to medicines and procedures, which affects dosing, endpoint selection, monitoring and follow-up. Age-appropriate study design helps ensure that data are scientifically robust while reducing unnecessary burden and risk.

What challenges are involved in enrolment and retention in paediatric trials?

Enrolment and retention in paediatric studies depend heavily on family decision-making, trust and practical feasibility. Travel, time commitments, school disruption and anxiety around procedures can all affect participation. PHARMExcel helps sponsors and sites address these issues through realistic, family-centred trial planning and governance of site-led enrolment pathways.

Does PHARMExcel support global paediatric clinical trials?

PHARMExcel can support paediatric studies involving regional or international regulatory coordination where needed, but our role remains focused on the clinical trial stage and on providing practical governance, operational oversight and regulatory support. We help ensure studies are aligned with the relevant ethical and regulatory expectations for the regions involved.

Why should sponsors choose a specialised paediatric CRO like PHARMExcel?

A specialised paediatric CRO brings expertise in paediatric trial governance, regulatory planning, age-appropriate study design and family-centred delivery. PHARMExcel combines this with the flexibility, continuity and personal service of a smaller, boutique CRO, helping sponsors deliver paediatric studies that are ethical, compliant and workable in practice.