Incorporating The Voices Of Parents And Children In Pediatric Clinical Trials
By Annick de Bruin and Shipra Patel, M.D.
A survey of families provides critical insights about their motivations and challenges.
Ensuring the health and well-being of children is essential in any setting, but pediatric clinical trials present an especially vulnerable environment in which children’s safety is paramount. Indeed, pediatric trials are far more complex and multifaceted than simply evaluating a therapy in the context of a child’s disease or illness. They are a family affair that must accommodate the diverse needs of parents, siblings, and often extended family members who care for children with chronic and acute illnesses.
However, pediatric studies do not always account for the unique needs of the family unit, nor is there sufficient research to fully appreciate what drives or deters families from participating. Understanding these factors is critical for engaging families to join clinical research studies.
Why Are Pediatric Trials So Important?
Children have different physiologic and metabolic processes1 than adults and thus often respond differently to the same medication. Additionally, as childhood diseases may have a different pathogenesis and progression of symptoms, a respective drug’s benefits, dose, formulation, and side effects — as well as its impact on normal childhood growth and development — must be evaluated in the populations who will be receiving it.
But according to the FDA, only 20% of drugs prescribed to children are approved for pediatric use,2 meaning the majority of drugs routinely prescribed to children have not been evaluated for their effects in children. As a result, children may be receiving medications that aren’t fully effective, in doses that may not be optimal, or for symptoms that may manifest differently than in adult populations.
So, what can the pharmaceutical industry do to more consistently engage families in joining pediatric clinical trials? The first step is to listen to patients and caregivers to better understand their motivations, barriers, and perceptions of clinical research.
Listening To Parents And Patients
In 2020, the Center for Information and Study on Clinical Research Participation (CISCRP) conducted an online survey of 500 parents and children in the U.S. to gauge their attitudes and experiences regarding clinical trials. The survey findings were instructive and provided a window into nuanced, but meaningful, elements that could increase children’s participation in trials.
Among the motivating factors, altruism was a key driver for both parents and children when deciding whether to participate in a trial. According to the survey, 63% of children listed their primary motivation as “the desire to help scientists learn more about their disease,” while 42% of parents listed “helping others with the same disease/condition” as their top motivation. While altruism is often cited among adult trial participants, this selfless sentiment from children should provide renewed motivation to communicate the value of clinical research in recruitment and educational materials designed for children and parents.
Survey results showed that pediatricians play a vital role in both the parents’ and children’s decision-making processes about participating in a clinical trial. While parents cited advertisements as a common vehicle for hearing about a clinical trial, parents were most likely to cite a doctor’s recommendation as the primary reason for deciding to enroll their child.
With this in mind, it is critical to provide comprehensive information and routine outreach to pediatricians about local trials that could interest their patients. Pediatricians must be well-equipped to fully explain to families what they can expect from a trial, including all the potential risks and benefits, so that parents and children are fully informed about the process.
Both parents and children affirmed that the more information they have, the greater sense of control they feel about the trial. Before deciding on whether to enroll their child in a clinical trial, 79% of parents said it would be “very important” to know the potential risks and benefits of the trial. Seventy-eight percent said it would be very important to know the types of medical procedures required, and 73% said it would be very important to know the purpose of the trial and if they and/or their child’s confidentiality would be protected.
One parent advocate stated that fear of the unknown was a key driver in her decision-making process regarding her son Jack’s treatments. “Rather than it being a reason for us not to participate, we used fear as a motivator to engage in research, find out about trials and bring them to the table with the medical team when they had appointments,” said Rachel Daley, whose son has a rare disease called Langerhans cell histiocytosis. “It’s really important to empower kids to make those decisions and know that they're in control.”
Little comforts can make a big difference in how kids relate to an activity or task in front of them. Sixty percent of children surveyed said that having free Wi-Fi at the site was very important, and 59% of children listed the availability of meals or snacks at study sites as also very important. Perhaps less expected was the importance of hearing from other children who had participated in research. Fifty-eight percent of children surveyed said that “getting to hear from other kids like me” who have taken part in a study was a priority.
As such, it is important to consider the inclusion of younger ambassadors who, with their parents’ permission, can share their experiences with other kids, either in person at a site, via videos, or through patient advocacy groups. Such a strategy could help alleviate one of the major barriers, that of fear of the unknown, identified by children in the survey.
Reducing Barriers And Burdens
Not surprisingly, parents reported burdensome experiences and high levels of disruption to their daily routines associated with their child’s participation in a clinical trial. In contrast, children ranked medical tests, blood draws, and taking a study medication as difficult, but they perceived study participation as less burdensome to their daily routines than their parents did. Eighty-three percent of children said they were “able do the things they normally would do” while taking part in the clinical trial.
The recent increase in decentralized trial approaches, necessitated by the pandemic, is significantly reducing the burden for families by replacing in-person site visits with telehealth visits, home-health nurses, wearable sensors, digital apps that upload data, and direct-to-patient drug shipments. In turn, these approaches are enhancing access to individuals who may live far from study sites and previously were unable to participate. Trials involving adolescents may be especially well-suited to using such tools, as teens are digital natives who prefer to communicate and share data via devices.
To be truly patient-centric, however, even a decentralized approach should allow for variations in families’ needs and preferences. On a recent decentralized trial with adolescents that required visits starting at 7 a.m. to obtain 12-hour pharmacokinetic sampling, home nurses were deployed to fit into teenagers’ schedules rather than requiring them to wake up exceptionally early for a clinic visit and potentially miss school.
Conclusion
When a child is enrolled in a trial, in essence the entire family is enrolled, because parents and siblings – and sometimes extended family members – must schedule their lives around the trial requirements. Many of the practical challenges identified in the survey can be mitigated through careful personalized attention to the preferences and challenges raised by patients, caregivers, advocates, and others directly involved in clinical trials.
As we emerge from the global pandemic, it is critical that we review, analyze, and apply lessons learned regarding innovative decentralized approaches and reducing clinical trial burden for families so that the momentum we’ve gained is not lost.
CISCRP is a Boston-based, globally focused, non-profit 501(c)(3) organization providing public and patient education and advocacy. CISCRP’s mission is to inform patients and the public about clinical research and the important role that it plays in advancing public health and to help stakeholders in drug development engage with patients and the public as clinical research partners. (www.CISCRP.org)
References:
1 https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3857037/
2 https://www.fda.gov/drugs/information-consumers-and-patients-drugs/drug-research-and-children
About The Authors:
Annick de Bruin is the senior director of research services at the Center for Information and Study on Clinical Research Participation (CISCRP). She is responsible for the design, implementation, analysis, and reporting of a variety of CISCRP research studies, including the Perceptions & Insights studies and numerous patient advisory boards. She has more than 20 years of experience conducting primary and secondary research studies in the healthcare, life sciences, and consumer goods industries. She holds an MBA from the Graduate School of Management at Boston University and a Bachelor of Science degree from Bryant University.
Shipra Patel, M.D., is the global head of pediatrics at Parexel. She works closely with a large group of pediatric experts to provide strategic guidance for biopharmaceutical clients worldwide. She is interested in incorporating patient- and family-centered approaches and optimizing clinical trials for pediatric patients with rare diseases. She is a board-certified pediatrician and pediatric endocrinologist who specializes in endocrine and diabetes clinical care and research.