Listen to the Youth Voice for Patient-Centric Pediatric Drug Development

Patient Stories |
Trial Design

Editors note: Courtney Tate is a Senior Clinical Research Scientist in the Pediatric Capabilities Function supporting pediatric research at Eli Lilly and Company. Courtney obtained her bachelor’s degrees in chemistry and molecular biology from the University of Indianapolis and doctoral degree in biochemistry and molecular biology from Indiana University School of Medicine.


One of the best ways to become more patient-centric is to hear directly from the people who may use our medicines now or in the future. This includes getting input from youth for pediatric research. What is important to them? What would make their life better? How could we improve the medicines, materials, devices, trial designs, and apps that they will be using or taking part in?

In the past few years, key people involved in drug development and research (advocacy groups, patients/caregivers, clinicians, industry, and regulatory agencies) have been advocating for pharmaceutical companies to become more patient-centric. Linked to this, the FDA has a Patient-Focused Drug Development effort that includes developing a series of four patient-focused drug development guidance documents. These documents address how stakeholders can collect and submit patient experience data and other relevant patient information for medical product development and regulatory decision making.

iCAN works to bring patient-centricity to pediatric drug development

What is iCAN?

International Children’s Advisory Network, Inc. (iCAN), is a worldwide consortium of children’s advisory groups, known as Kids Impacting Disease Through Science (KIDS) and Young Persons Advisory Groups (YPAGS). These dedicated youth member groups work in unison around the world to provide a voice for children and families in medicine, research, and innovation.

iCAN members express their desire to be part of something bigger than themselves. They want to make a difference through meaningful contributions in the lives of children and youth living with a medical condition. iCAN members voice their true opinions, needs, ideas and experiences which enables their voices to be understood, be valued and have an impact.

Lilly and iCAN collaboration

Over the past few years, Lilly and iCAN have collaborated in person at the iCAN Annual Summit and virtually on numerous projects to gain insightful input from youth. We do this to ensure we are creating patient-facing materials that are age-appropriate, comprehensible, and useful to the child and caregiver/parent. For example, we’ve obtained feedback and perspectives from iCAN youth on educational and trial materials including:

  • a training brochure for pill swallowing
  • a placebo response video
  • the Understanding Clinical Trials Comic Books
  • our assent templates or documents for youth to give permission to participate in research

In addition, iCAN youth have given us input on device preferences, level of comfort, and their innovative ideas around continuous data collection in clinical trials, as well as vital information to guide our age-appropriate formulation strategies.

We also want to make sure we design trials that are feasible and are really measuring what is important to the youth and their family. They are the experts on what it is like to live with their condition, so when you listen to their perspective, you get a small glimpse into their life. Research for children is complicated for many reasons, which is all the more reason to ensure we get youth input when possible.

I remember one young man with Duchenne Muscular Dystrophy who shared that he didn’t care about the standard 6-minute walk test, which measures how far he could walk in 6 minutes. Rather, he cared about being able to put his shirt on, hold his own glass and toast with his friends, and get from his desk to his bed.

My experience with iCAN

The iCAN youth are a true inspiration. Their stories are both heart-wrenching and motivational and stick with me long after I hear them. These youth are dedicated, driven, extremely bright, and never cease to amaze me with their creativity and unique concepts. I can’t say enough about the invaluable partnership with iCAN. The youth voices are critical to pediatric drug development and research.

It has been over 20 years since I was a child. Of course, I’ve gained experience along the way to approaching middle age, but I’ve also lost the way I used to think and feel as a child. The world in which our children are growing up is so different from the world I grew up in, or that my parents and grandparents grew up in.

To stay current and tailor the way we approach pediatric drug development, we have to listen and respond to what the youth and parents/caregivers are telling us. Just like raising children takes a village, it takes all of us working together - the patients/parents/caregivers/families, industry, clinicians, advocacy groups, and regulators - to be successful in achieving Lilly’s purpose of uniting caring with discovery to create medicines that make life better for people around the world.

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