Children and young adults across the globe are largely underrepresented in clinical research. While legal actions such as the Research to Accelerate Cures and Equity for Children Act (RACE) are helping expand pediatric clinical research, pediatric patients continue to lack clinically approved treatments via randomized trials due to a variety of factors, including accessibility, health care disparities, and barriers to clinical trial involvement.
PRA’s World Children’s Day webinar featured young adult patient panelists and speakers from across the clinical and medical fields to discuss ongoing health care disparities for children, and how clinical trial participation can be improved to better serve this population.
Hosted by our Center for Pediatric Clinical Development, we celebrate World Children’s Day each year to commemorate the Convention on the Rights of the Child, which calls for equality for children and for improving lives of children across the world. For our 2nd Annual Celebration of this day, experts and attendees across the globe joined us on November 16th for our virtually held event, “PRA’s World Children’s Day: Defying Health Care Disparities and Clinical Trial Barriers in Pediatric Populations.” This event featured young adult patient panelists and speakers from across the clinical and medical fields to discuss ongoing health care disparities for children, and how clinical trial participation can be improved to better serve this population. Thank you to all who attended!
Mark Sorrentino, MS, MD, Center for Pediatric Clinical Development, began the event by reminding us the importance of giving children the same welfare and the same platforms for their voices to be heard as adults. Heather Peterson, RN, BSN, Pediatric Strategy Liaison, Center for Pediatric Clinical Development, introduced the speakers and hosted a Q&A after each of the experts’ presentations.
Dr. Hemant Malhotra, MD, FRCP, FACP, MNAMS, FICP, FUICC, FIMSA from Jaipur, India, was our first speaker. He addressed the many levels of different health disparities and determinants when it comes to disproportions in patient populations with diagnoses such as cancer. The importance of addressing these issues stems from the fact that disparities exist in adult patient populations already, which could mean that these same issues trickle down to the pediatric populations.
“We need to educate our clinicians to remove these disparities,” said Dr. Malhorta. “We need to have diverse populations and diverse clinicians in our clinical trials so that you get a more real-world experience in the data which you are publishing. We need to show more consideration for social determinants, and we need to address the social determinants of health so that we can ensure more equitable cancer care.” Factors such as the patient’s geographical location, access to healthcare, ethnicity, and gender all play a role when it comes to a patient’s ability to get the treatments they need.
Dr. Nathalie Gaspar, MD, PhD, Pediatric Oncologist, Head of adolescent and young adult (AYA) unit, Chair of AYA program and Gustave Roussy Cancer Campus in Villejuif, France, spoke about the underrepresentation of AYAs in clinical trials and how to improve AYA access to innovative therapies. She discussed how pediatric development is delayed compared to adult treatments, and how this delay keeps pediatric patients from accessing new and effective drugs that have already been approved in adult populations. Dr. Gaspar explained that there is no increased risk for adolescents to include them in more adult, early phase trials, and so we need to consider pediatric patient populations when it comes to including them in adult clinical trials.
“Pediatric oncologists and medical oncologists must work together to increase awareness and be really pragmatic when we are doing things,” Dr. Gaspar said. “We’re trying to increase the efficient recruitment of adolescents.”
With the focus being on the pediatric and young adult patients, this event featured young adults living with challenging conditions. Emily Grombala, 17, a member of the Live Like Bella Childhood Cancer Foundation who is currently battling Ependymoma, shared a pre-recorded video. This video highlighted her experiences with surgeries and holistic treatment, with her key message inspiring us to, no matter what, keep moving forward:
Following the video was a live discussion between a panel of young adult advocates moderated by Dr. Gaspar. Max Williamson and Benjamin James shared stories about their conditions with cancer and rare diseases. Benjamin, a patient with a rare disease from the UK and a trustee for Pathfinders Neuromuscular Alliance and supported by Action Duchenne, and Max, a patient with cancer and advocate for the UK National Cancer Research Institute and the ACCELERATE FAIR TRIALS Working Group Focus, discussed their experiences as patients and moving treatments forward with Dr. Gaspar.
Dr. Gaspar, Benjamin, and Max shed light on psychosocial factors and the fact that patients can help each other as people who’ve been through the process. The conversation took on the different issues that surround adolescent challenges when it comes to getting treatment and sharing different young perspectives—how they manage the disease, their involvement to change and improve care, talking about transitions into everyday life, and keeping optimistic perspectives, and a forward-moving attitude.
Benjamin talked about the time when he was approached about participating in a clinical trial. However, he decided not to partake due to the burdens of traveling far distances while also juggling life as a young adult in college. He described that the timing wasn’t right, and he didn’t want to have to worry about managing his condition on top of managing the many other factors of being a student. Dr. Gaspar asked about what would have to be different for him to participate. Benjamin said that, had the clinical center been located closer to him, he would have been more willing to participate in the trial. “I think if I had perhaps been closer to the clinical center that was partaking in it, which was back in London, and I wasn't in London at the time, I probably would have taken part in it more easily or I would have said yes,” Benjamin said.
He also offered his perspectives around what is important for young patients when it comes to these trials, that clear communication and understanding the logistics are all important. In his discussion with Dr. Gaspar, he emphasized the fact that the choice to partake in a trial takes time from a young person’s life, and not only impacts them but their family who helps them attend visits required by participation in the clinical trial. “It's about understanding the practicalities and the logistics of making it all work, which is quite challenging, and I think that can be overlooked,” Benjamin said. “The family might have to support them, or a young person might want support in attending that clinic or the clinical trial, so that should be considered as well.”
Dr. Gaspar asked about his transition between pediatric and adult wards during his treatments. He said that the conversations to transition started towards the last year two years of his pediatric care, but that it there wasn't any real transition, and that he thinks transition to adult care depends on the maturity of the individual. “I was quite mature, so for me it didn’t matter so much, but it's not always as smooth as it should be in the UK. Fortunately, I live just outside of London, so I have access to specialist center, but for some people it's very hard to get access to those specialty centers in the UK. It’s also more on you to arrange your own support and finding out about clinical trials.”
When she asked about changing that transition, Benjamin said that it would be helpful to have a particular person to go to during a transition, a “key worker,” someone whose job it is to coordinate that transition.
Max, a patient with cancer who was treated beginning in 2012 when he was 15 years old, joined the discussion as well. “I think that the priority really is making sure that everyone or every young person can be involved in research in some shape or form, across the world, and beyond cancer as well.” In the UK, Max works with young people and focuses on inclusion and being culturally sensitive in approaching patients, as well as helping to expand how patient information sheets (i.e., assent forms and parental information sheets) are clear and understandable for different reading ages. “For example, we’re expanding outpatient information sheets on trials so that we increase in terms of language or learning understanding and reading age. We also advocate for the removal of age limits on clinical trials that might exclude young people based on the fact that there may be under 18 for another trial over 21 for a pediatric trial.”
Dr. Gaspar also asked about his transition between pediatric and adult care. “The transition is a really interesting experience and I think everyone has a very individual take on it,” Max said. “I was relatively lucky that we have dedicated young person council wards in a few of the main centers across the UK and I was really lucky to have been treated at one of them. That meant I was crossed in between the pediatric, the young adult wards and the adult wards. It was a really weird experience, I must admit, and it was a complete change of tone between the way I converse with my pediatric doctors and my adult physician and the kind of patients who surrounded me to being young and having people around much older than me. I was really overwhelmed by it…It’s something we definitely need to work on.”
Max and Benjamin then shared their inspiration to be a part of the advocacy work that they do. Benjamin said that for him, it was “hearing from other adults and hearing from some of the charities…that has definitely helped me think about it and particularly some of the other people who also advocate for the expansion of clinical trials in the UK.” For Max, it’s about giving back to his community. “If I can, as a patient with an experience of the disease, contribute anyway to research, I'm doing my best to.”
The attendees on the call were thrilled to hear these young voices sharing such important insights and perspectives. Hearing them speak out about their experiences tied nicely into the final presentation given by Annick de Bruin, Senior Director, Center for Information and Study on Clinical Research Participation (CISCRP). Her presentation on insights in clinical trial participation from the perspective of children and their parents highlighted findings from 2020 CISCRP pediatric perceptions. She shared that patients who have children who’ve been in clinical research report highest levels of understanding of clinical research, awareness of studies actively enrolling, and willingness to have their child participate. Though children report burdensome study experiences, more indicate that the study exceeded their expectations and they’re willing to participate again.
“The altruism demonstrated by our panelists validates to our industry they are not the barrier to equality or clinical trial participation,” says Heather Peterson. “We can do better.”
The event ended with a lovely compellation by Henri Olldashi of young adults across the world sharing in their respective languages their gratitude towards health care physicians.
With studies showing findings like these, it’s important for clinical research stakeholders to hear from young patients willing to share their experiences with receiving treatment and care for their conditions. Each presenter offered ideas and solutions to help break down barriers to care, such as expanding treatments from academic centers to more localized, regional locations, improving trials with better technology and utilizing smart devices and decentralized research, implementing diversity trainings, and more. It’s crucial that we help elevate young voices across the globe so that they can help drive a movement towards better treatments and better access to care.
Our industry is still guessing the clinical needs of children by settling for a fraction of what works in adults. We’re changing that. At PRA’s Center for Pediatric Clinical Development, we have the unparalleled expertise and resources needed to run exceptional pediatric trials from start to finish. Our in-house built network of pediatric centers (PRA’s Pediatric Site Network) allows us to consult with other global pediatric experts. Additionally, our internal inter-departmental pediatric collaboration team and our extensive global pediatric regulatory understanding gives us the rapid ability to tap into the world’s best pediatric experts to deliver pediatric-focused insights across all aspects of a pediatric drug development.
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