Author: Earl Seltzer, Associate Director-Global Feasibility, Member-Quintiles Pediatric Center of Excellence
When it comes to treating pediatric patients, we cannot treat them like tiny adults and simply give them smaller doses of the same medicines. Their developing organs and changing metabolism affect how they process drugs, and that must be taken into account when treating them.
The only way to do that is to conduct pediatric clinical trials to validate the safety and efficacy of treatments and proper dosing in pediatric patient populations. Currently, only a small percentage of drugs and devices approved by the Food and Drug Administration (FDA) or the European Medicines Agency (EMA) are labeled for pediatric use. But that is beginning to change.
Over the past 18 years, regulators in the US and Europe have rolled out a combination of incentives and regulatory requirements to spur more pediatric clinical trials. But managing the logistical, legal, and cultural considerations that come with such research can be daunting, especially for developers who may not have a comprehensive understanding of the safety and ethical issues that govern this research or may not have the appropriate operational experience to manage pediatric trials.
One potential solution, which I addressed in my DIA presentation this year in a collaborative session with FDA, academics, and industry, is to establish working relationships with a pediatric clinical trials network. Such a network would be an alliance of clinical research sites, typically as part of academic institutions that collaborate with each other and with industry to plan and manage pediatric clinical trials safely, effectively, and efficiently. These networks would be focused on the conduct of pediatric clinical drug or device trials in a variety of areas and the success of such a network would largely be dependent on buy in and cooperation with all key stakeholders inside and outside the network.
Pediatric research networks promise to reduce risk and uncertainty inherent to pediatric clinical trials by enabling collaborators to engage the best investigators and benefit from the shared expertise and best practices of all partners. Furthermore, when CROs work with such alliances, all partners may benefit from the collective expertise of the organizations, which allows for less time spent on administrative tasks related to business processes and finding sites, and more time recruiting and managing patients within the trial. There are many benefits to working with a global pediatric research network:
- Collaborators can take advantage of centralized contacts, which may reduce the time and cost normally spent navigating a laborious pre-study planning and site feasibility process to set up such trials.
- Collaborators can gain better insight into the time and cost expectations of a trial up front, based on past experience working in pediatric research, which can help them develop more realistic budgets and times to study completion.
- Collaborators may benefit from the shared regulatory input and knowledge from experts at the local, regional, and national levels. Such knowledge helps address regulatory needs during the planning phase of the trial (especially at a local level), helps them navigate the pediatric regulatory landscape, which may vary substantially by country, and reduces risks related to fulfilling regulatory obligations that are often the reason for the trial being conducted.
- Collaborators can take advantage of pre-engaged specialty sites worldwide that are uniquely positioned to oversee pediatric research and have a clear interest in pediatric clinical trial participation. In knowing that these sites exist and are willing to participate in clinical trials, collaborators can effectively reduce the time identifying sites for pediatric trials and potentially reduce administrative time in start-up.
- These networks seek to include or may have the most seasoned investigators around the globe, who understand the intricacies of recruiting pediatric patients and addressing the concerns of their families. Such understanding of the particulars of pediatric clinical research is absolutely essential to set appropriate expectations up front for patients/families and may help reduce the possibility that sites may not enroll any patients. An additional benefit of pediatric networks is that seasoned investigators can mentor more less experienced investigators as they begin work within the network which may help lessen the burden and learning curve associated with the conduct of controlled clinical trials.
However, to ultimately be effective working together in this collaborative format, the sites, sponsors, and CROs participating in these networks must release some degree of autonomy to work within the network, while agreeing to jointly share in the overall risks of the study. This may require a fundamental culture change within and across organizations that have little history of participating in truly collaborative efforts. But the benefits of this collaboration far outweigh the risks for biopharma and the site networks themselves as we are collectively focused on improving the lives of children around the world, for which clinical trials are essential.