Three Partners, One Mission: How Sites, Sponsors, and CROs Strengthen Pediatric Oncology Trials Together

Written by:

Jacqui Whiteway, Senior Director, Center for Pediatric Clinical Development, ICON, plc

Melanie Villanueva, Medical Director, ICON, plc

If it takes a village to raise a child, it takes a city to support a child through cancer treatment and clinical research.

Parents and guardians, siblings, wider family members, physicians, nurses and hospital teams are the most visible parts of that city. They are the people closest to the child and family every day. Behind the scenes, sponsors and clinical research organizations also play an important role, working with sites to help make pediatric oncology trials safe, feasible and meaningful for the children and families who take part.

Principal investigators and clinical research coordinators often understand the CRO’s role well. Others across the site team may see only part of the picture: a monitor’s visit, a start-up request, a data query or a protocol question. In this article, we explain how CROs support sites and sponsors in pediatric oncology trials, and why that partnership matters.

The need remains urgent. Globally approximately 400,000 children and adolescents from birth to age 19 develop cancer each year.¹ While more than 80% of children with cancer in high-income countries are cured, less than 30% are cured in low-and middle-income countries.1 “Cured” means remaining cancer-free five years after treatment, but surviving is not thriving, with 60-90% of childhood cancer survivors developing chronic, long-term health effects. Lack of awareness of symptoms, access to healthcare, and misdiagnosis are all contributing factors. Opportunities to participate in clinical trials may also have an impact. In the U.S., cancer is the leading cause of death from disease among children from birth to age 14.² Cancer death rates in this age group have declined by 70% from 1970 to 2020, but of the estimated 9,550 annual new U.S. cases, about 1,050 children will die.2
Pediatric oncology trials are one way that better treatments reach children. They can evaluate new therapies, new combinations, new doses or medicines already used in adults but not yet fully understood in children. They explore how to maintain or improve cure rates while reducing toxicity due to therapies. The trials also help generate the evidence needed to improve treatment decisions, reduce burden where possible and support better long-term outcomes.

In many therapeutic areas, one of the greatest challenges is finding experienced investigators and sites with the capacity and interest to participate. Pediatric oncology is different in important ways. Many pediatric oncology teams are deeply experienced in clinical research and highly motivated to conduct trials because trials provide access to emerging or adapted treatment approaches for children.

That does not mean pediatric oncology research is simple. These studies involve complex legal, ethical, regulatory and clinical considerations.

Trial designs must account for developing bodies, family decision-making, assent and consent, school and family routines, long-term safety considerations and the emotional weight of treating children with cancer.

The challenge is often not a lack of commitment from sites. It is that there are still too few trials available for children, particularly trials designed around pediatric needs from the outset. That is where early collaboration among sponsors, sites, and CROs can make a meaningful difference.

Before a sponsor awards a study to a CRO, multiple CROs may be invited to submit proposals. As part of that process, CRO teams often ask sites to provide input through feasibility questionnaires or direct outreach. For sites, especially those approached by several CROs about the same study, these questions can feel repetitive.

We understand that frustration. But this early feedback is not a box-checking exercise. It is one of the first opportunities for sites to shape how a trial is designed and delivered.

Site input helps CRO teams understand likely operational challenges, assess whether enrolment assumptions are realistic and identify parts of the protocol that may create unnecessary burden for children, families or site staff. It also gives CRO experts the evidence they need to advocate for practical improvements before the study is finalized.

Improvements like reducing the number or frequency of blood draws, adjusting visit schedules, or improving family support measures may seem small on paper, but they can matter greatly in practice. Sites and CROs understand the importance of building in comfort strategies to reduce anxiety and identifying ways to minimize medical trauma for children already facing an overwhelming experience.

Early input from both sites and CROs helps uncover strategic considerations that sponsors can address upfront, reducing downstream operational and execution risks.

Many people working in pediatric oncology at CROs have backgrounds in cancer research, clinical care, sponsor-side drug development or site-based trial delivery. That experience matters because the CRO often sits at the intersection of sponsor objectives, site realities, regulatory requirements and patient-family needs.

A CRO can bring cross-study, cross-sponsor and global operational experience that helps pressure-test a trial before it begins. That perspective can be especially valuable in pediatric oncology, where the number of eligible patients may be small, the treatment context may be urgent, and the protocol must work for children, families and highly specialized site teams.

Once a study is awarded, sites may interact most often with a CRA, monitor, start-up associate or clinical trial manager. Behind those visible touchpoints is a wider delivery infrastructure that may include clinical operations, start-up, regulatory, medical, safety, data, biometrics, recruitment, logistics, and therapeutic-area expertise.

The purpose of that infrastructure is not to add complexity. It is to help keep the trial consistent, compliant and coordinated across sites, countries, vendors and regulatory environments. It also helps ensure that questions are routed appropriately, decisions are documented and site teams receive aligned, up-to-date information.

In pediatric oncology, that support can include access to physicians with relevant pediatric haematology-oncology experience, including clinicians who have worked at the bedside and understand the realities of treating children and supporting families through clinical research.

When a trial is awarded, the CRO’s robust team, with its broad array of expertise and roles, gets to work. Sites may be surprised by how many team members there are on the CRO team, as most are not visible to the site staff. Behind the onsite CRA/monitor and the emails and calls from a start-up associate, there are specialists working to ensure the trial runs smoothly. There are regional and global project managers (PMs), in-house CRAs, start-up leads, clinical trial managers (different from PMs), biometrics, analysts, statisticians, logistics experts, marketers, recruiting specialists, regulatory (global and country-specific), therapeutic experts, medical monitors and medical monitor assistants. This lengthy list is not exhaustive. Even for small trials, there are usually a dozen CRO team members. For larger studies, it is typical for there to be 100 project team members, and this doesn’t include all the other vendors that are often managed by the CRO.

Each role is filled by someone equipped with thorough training, clear responsibilities and accountabilities, and all working together. Beyond the assigned team, there are a multitude of other resources and experts available to support and back them up. For example, while a trial may have one medical monitor assigned to a trial, there is 24/7 coverage for all sites, so processes, training, systems and communication are refined such that a qualified physician with specific knowledge of that trial is available at any time a site anywhere needs it. This includes pediatric haematology-oncology physicians, most of whom have worked bedside for years in clinical practice conducting trials as PIs or sub-investigators.

There is a reason behind every question, even the ones that appear ridiculous or to have obvious answers. Some of these are automatically generated from the clinical database and CRO team members may sometimes feel uncomfortable asking what seems like an obvious question, but we are always happy to explain why it is being asked. CROs are responsible for ensuring that the data collected satisfy all trial requirements, are regulatorily compliant and ultimately lead to a successful submission. With vast knowledge of variable global regulatory requirements and best practices, CRO team members ensure that efforts made at every level are consistent and compliant. This means ensuring that all the work, time and effort made by sites, participants and their families contribute to a successful conclusion of the trial (whether that be a new medicine or not). For example, updated guidance, regulations and the logistics that entail when a child “becomes an adult” in a particular region can be complex and impact whether some data are used for a submission. One unique aspect of pediatric trials is that a participant may start the trial as a child but become an adult from the regulators’ perspective. This means that where the child initially provided their assent along with their parents’ consent, once they reach that legal age they must provide their own consent. Otherwise, any protocol-related data collected after their birthday may not be able to be used (regulations vary by time and region). Thus, one trial may have children becoming adults at age 14 in Austria, age 16 in the UK, and 18 in the U.S. The CRO must ensure that, across the trial and all regions, consistency is maintained despite such variability.

Site teams should understand that the CRO is an extension of the sponsor and to utilize their CRO contacts to streamline communication. Sponsors may get hundreds of questions from numerous different sites, slowing down and sometimes confusing the communication process. It may not always seem like it, but using your CRO contact improves communication so that sites get accurate up-to-date information faster. CROs have pediatricians and pediatric hematology oncologists with patient experience who are available to support sites at any time.

As the central point of contact for multiple site locations, CROs can separate the signal from the noise in study data. Participant safety is paramount, and CROs are there for adverse event reporting. But we’re also primed to look for quality indicators.

In one pediatric oncology study drawn from Melanie’s clinical experience, emerging data indicated that a therapy at a specific dose was delivering better outcomes than the standard of care. The CRO study team worked closely with the sponsor and investigative sites to rapidly collect, review and communicate the evolving evidence. This collaborative approach enabled the sponsor to act on the findings before the trial concluded, allowing children in the comparator arm to also receive the investigational therapy. Seeing her own patients benefit from this decision underscores a lesson that is often overlooked: meaningful advances in pediatric medicine are rarely the result of one individual or organization, but of a complex ecosystem of partners working together to improve children’s lives.

These are the treatment developments motivating everyone in the childhood cancer “city”. Close collaboration among sites, sponsors and CROs enables these breakthroughs, transforming treatments for children with cancer today and in the future.

References

1. Childhood Cancer Factsheet. World Health Organization. https://www.who.int/news-room/fact-sheets/detail/cancer-in-children. Published 2026. Updated 10 March. Accessed 29 July, 2026.
2. Childhood Cancers. National Cancer Institute. https://www.cancer.gov/types/childhood-cancers. Published 2025. Updated 25 April. Accessed 30 July 2026.