Clinical trials yield important information on a medical product's safety, dosing, and effectiveness in the people who participate in research. Today, however, more than 50% of medicines do not have data in the prescribing information (i.e., labels) to guide their appropriate use in children.
As a result, many drugs used in children are used “off-label,” meaning without a specific approval in the label for use in children. That’s because most medicines prescribed to children have not been tested in children, so there is insufficient clinical trial information to indicate the best and safest usage. For more information on the association between prescribing information and clinical trials, read this article.
Children in Clinical Trials
In the past, it was believed that prescribing a medicine to a child was okay as long as it was given at a lower dose. But the truth is that many drugs may affect children differently than adults. Children are in a constant state of growth – their kidneys, liver, lungs, brain are developing. And the difference in newborns and infants are far greater even.
Regulatory bodies such as the FDA recognize the need for more clinical trials in children. And recent measures have been put in place to have more clinical trials include the pediatric population.
However, there are some important challenges with pediatric trials, including:
- Designing a clinical trial that legally and ethically meets children’s needs and provides them adequate protection (such as growth and development issues)
- Using medicine that is not easily taken (such as hard-to-swallow or large tablets) or medical devices not easily used by kids
- Enrolling adequate numbers of children into clinical trials without causing delays in the time planned to complete the study
In an effort to understand and tackle some of these challenges, the American Academy of Pediatrics (AAP) Section on Advances in Therapeutics and Technology has created a broad collaboration to help understand how to better overcome some of these obstacles – by asking the children themselves.
Inspired by a program in the United Kingdom, the Kids and Families Impacting Disease through Science (KIDS) program was piloted. The KIDS program is an advisory group of children, adolescents and families who help researchers understand a child’s perspective in medicine, including participation in clinical trials. A unique collaboration between the Connecticut chapter of the American Academy of Pediatrics and local children’s hospitals, the KIDS program focuses on understanding, communicating and improving the process of health and medical innovation for children.
The objectives for the KIDS Program are to:
- Learn, teach, and advocate for research in medicine, and innovation that improves the health and well-being of children
- Engage in the process through consultation activities with hospitals, researchers, and other partners in the public and private sections
- Provide input on research ideas, solutions, and unmet pediatric needs
- Contribute to the design and implementation of clinical studies for children (such as consent or assent forms)
- Serve as a critical voice for children and families in medical research
According to Hadleigh Thompson (aged 16) from KIDS Connecticut, “The KIDS program gives children and families an opportunity to expand their knowledge about research and innovation and to become active participants in their own health. Ultimately, we hope the program will provide them with a vital outlet to share their unique perspectives that will make an impact on the lives of children around the world.”
KIDS Research Summit
In late 2013, a KIDS Research Summit was held at Pfizer’s Laboratories in Groton, Connecticut. The summit which included children between 7 to 17 years had an educational and advisory component. During the educational session, children were taught the difference between 'consent' (forms that are filled out by parents) and 'assent' (forms filled out by kids). They were also educated about the stages of drug development.
In the advisory sessions, the Pfizer clinical development team asked the kids general questions about clinical trials and elicited their feedback on ways to make their research more child-friendly. Researchers came away with a clearer idea of what future children participating in clinical trials may need.
Some key learning points included:
- Understanding how important it is that the information given to children in a form ('assent') be easy to read and understand
- Finding out how many times blood sample collection were too many for them
- Listening to kids share what they felt about staying overnight at a clinical research center for monitoring
- Learning how kids felt about getting an MRI (magnetic resonance imaging - a type of imaging test) and their difficulty with staying still for a long period of time during the test
- Gaining insights on how to best monitor children for specific adverse events
- Hearing about their wish to learn more about research in children, and how older kids were interested in mentoring younger kids involved in a clinical trial
The KIDS program has begun expanding across the US and abroad with partners such as local AAP chapters, pediatric societies, children’s hospitals, public and private research entities, and schools. In addition, the growing number of global advisory teams are being organized into a consortium known as iCAN (International Children’s Advisory Network) as a means for the youth advisors to connect, share, and collaborate internationally in medicine, research and innovation.
Recently, iCAN representatives from the KIDS Connecticut team were invited to participate in an NCD Child event and to deliver a UNICEF ACTIVATE talk on children rights. By focusing on pediatric patient engagement and feedback, the goal is to improve clinical trials to best fit the needs of kids. Not only do researchers want to help children, but also kids want to help kids. It certainly makes sense – they have the best perspective.
Some members of the KIDS Connecticut Team at the Pediatric Academic Societies Meeting in Vancouver in May 2014
Charles A. Thompson, MD FAAP, is the Global Lead for the Pfizer Pediatric Center of Excellence and the Chair of the American Academy of Pediatrics Section on Advances in Therapeutics and Technology. Dr. Thompson is a Clinical Instructor of Pediatrics at the University of Connecticut School of Medicine, a member of the medical staff at Connecticut Children’s Medical Center, and a Board Member for the Hezekiah Beardsley Connecticut Chapter of the American Academy of Pediatrics.