During PRA Health Sciences’ celebration of UNICEF’s World’s Children Day on November 20, experts from LSU-Shreveport, UNICEF, the Center for Information and Study on Clinical Research Participation (CISCRP), Jumo Health, the International Children’s Advisory Network (iCAN), and Sproutel were among the participants that converged on Blue Bell, PA. These dynamic speakers shared their expertise and insight into how they are making a difference and making the clinical trial experience more positive for pediatric patients.
Dr. John Vanchiere, Vice-Chair for Pediatric Research at LSU-Shreveport and a fierce advocate for children’s health in his home state of Louisiana, shared with the audience three major themes: the complexities of research specific to pediatrics, their successes to date, and the challenges ahead. As we all know, the decision not to vaccinate has become more prevalent and has a negative impact on our communities, especially for those that are immunocompromised. Dr. Vanchiere also briefly discussed the role mental illness plays in reaching children and adolescents, where he asked the audience “How do we ‘vaccinate’ children against the 5-fold increased risk of mental illness associated with Early Adverse Childhood Experiences?” It is his balanced approach to pediatric clinical research that has provided him insight into the challenges that lie ahead.
During a panel discussion, Lisa Latiolais, Nurse Manager of the Children’s Clinical Research Center at LSU-Shreveport, shared her perspective on what she sees as one of the industry’s key failures – patient reimbursement. Lisa, who works with a primarily indigent patient population, stressed that it is critical that we start to consider the impact clinical trials can have on the day-to-day activities of study participants. She described parents of patients who were unable to pay for basic transportation, let alone time lost from work. Lisa helped the audience understand many of the real world challenges these parents face when they agree to participate in clinical research.
Jennifer Paradis Behle, who serves as a New York Regional Board Member of UNICEF USA and leader of its Greater Philadelphia Committee, noted that World Children’s Day is historically a day of action. It is a day when children and young people stand up for their rights, advocating and raising awareness for the most pressing issues they face. She also shared that this year marks the 30th anniversary of the Convention on the Rights of the Child. This humanitarian treaty is the most widely accepted in history. It explains how governments, the United Nations, UNICEF, and other organizations work to make sure all children enjoy their rights. Since its founding in 1946, UNICEF has saved more children’s lives than any other humanitarian organization. UNICEF works in 190+ countries and territories to ensure that all children have the resources they need to survive and thrive.
Sisters Nicole and Melanie Mendez-Villarrubia, along with their mother Josie, suffer from Marfan Syndrome. They possess firsthand experience and unique insight into what young patients and families experience during clinical trials. Nicole stressed the importance of honest communication – researchers must do a better job talking and listening to kids throughout the trials. A major concern for Melanie was ensuring young patients actually understand what is taking place throughout the clinical trial. She expressed that she rarely fully understood what was going on, and that many times, those conducting the trial would answer her questions with confusing and complex medical terms.
Jill McNair, a Senior Director at the Center for Information and Study on Clinical Research Participation (CISCRP), shared her organization’s role in educating and informing the public, patients, medical/research communities, the media, and policy makers about clinical research. Jill stressed the importance of sharing clinical trial results with patients and the necessity of recognizing the contributions of the participants in their clinical trials. “You would be amazed how far a simple thank you can go,” she reminded the audience.
Rick Scorzetti, Sr. Vice President, Strategic Solutions at Jumo Health, communicated the unique approach he and his colleagues bring to producing actionable resources for patients of all ages. Explaining a clinical trial or particular disease to an adolescent would not be the same as explaining to a 7 year old, and may be insulting to an older teenager. Rick’s team accesses multiple media channels to communicate difficult-to-understand information about the disease, treatment or clinical trial participation. Then, they rework the content into an age appropriate format. All content is created with the patient voice front and center, following extensive interviews from pediatric patients about the particular condition.
Aaron Horowitz, co-founder & CEO of Sproutel, shared his company’s efforts to connect with younger patients through play. Sproutel uses a proprietary patient-centered, empathy-driven design process that takes the information they glean from the interview process and assembles visual frameworks to properly communicate that information. This empathy driven-approach – seeing what the child sees – has led Sproutel to create products that help children better relate to their illness and treatment. Embedded in these seemingly simple products is innovative technology that creates an immersive experience for the child, while providing a companion to share in their journey. Aaron and Hannah Chung founded Sproutel to create their first product, Jerry the Bear, which helps children with Type 1 Diabetes learn how to take care of themselves through taking care of Jerry's diabetes. Kids care for Jerry through an augmented reality experience in the Jerry the Bear mobile app. They can access virtual diabetes tools, a pantry of food, and a series of interactive storybooks. They discover how to monitor blood sugar, count carbs, and dose insulin. In partnership with Aflac, Sproutel applied their design process for children and their families undergoing cancer treatment to create My Special Aflac Duck, a plush robotic duck. The duck is interactive to touch, come with accessories, and a mobile app. It expresses a range of emotions, has a haptic heartbeat, and laughs when tickled! Like Jerry the Bear, the duck can be used in medical play and accompanies children throughout their treatment to provide comfort and joy.
The major theme of all these presentations was the absolute need to engage our children more directly. That way, we can fully understand what they are going through. When we design clinical trials, we become so concerned with the science, agency requirements, and regulatory or ethics considerations that we forget to consider how our design might impact our patients and their families.
Melanie Mendez-Villarrubia, the young patient and advocate, said it best: “We are people first and we have lives outside of your study.” If our industry can approach trial design with this sentiment front of mind, and prioritize ways to engage with the patients from the beginning, we can expect increased participation, better compliance, and a more positive overall experience for the participants. The need for this diverse perspective must continue to grow and evolve before we can say we fully understand the patient’s perspective.
When working with pediatric populations, play is a fundamental conduit to communicate with those who are not able to articulate specific emotions. When children engage in play, we as researchers can better understand a young child’s perspective. This perspective allows the clinician to communicate back to the child through a toy surrogate.
In Melanie and Nicole’s story of their challenging journey, they highlighted their continued desire to participate. This desire was tied to their belief that even if they did not personally benefit, they knew they were contributing to the greater good. While this is quite noble, we must remember not to rely on the altruistic nature of study participants as the primary impetus for their participation in a clinical trial. Rather, we must continue to evolve our understanding of what drives a patient’s interest to participate and incorporate that into our trial designs.
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