No One Ever Asked a Sick Child: Rethinking Pediatric Patient Engagement

The success of a clinical trial is often measured with metrics and milestones. But patient advocate Gunnar Esiason shared a different perspective during our CORE Symposium: the true indicator of success is far more human. 

ProofPilot recently hosted our first-ever CORE Symposium at the historic Fenway Park—an intimate clin ops event focused on tactical tools and strategies for study sponsors in a world of conferences that prioritize innovation over reality.  

Gunnar Esiason, a cystic fibrosis patient advocate and Senior Director of Patient Engagement at Raven, opened the event with his powerful, unfiltered view of clinical research from the patient's shoes, but with a twist: recounting his experience as a clinical trial participant. 

Gunnar Esiason, Raven (RA Ventures)

Recounting his lifelong journey with CF and his experiences within the healthcare system as a child, Gunnar revealed a perspective that few have ever heard. His ability to articulate his childhood experiences in research was a voice typically delivered by the parent. But as an adult, it lets us directly into the child’s mind, offering a profound and sometime chilling perspective. Yes, of course, the patient belongs front and center in trial design, execution, and enrollment. But from a pediatric lens, this concept took on a whole new dimension. 

Let’s dive deeper.

"No path forward without clinical trials"

Like many study participants, Gunnar turned to clinical trials when he had run out of treatment options for his cystic fibrosis. 

Diagnosed at the age of 2, Gunnar endured years of hospital visits, treatments, and procedures before every approach began to fail and he was left, in his words, “a shell of himself.”  

By the time Gunnar entered his first clinical trial at the end of high school, he knew all too well the realities of being a patient. He’d lived it all, from “this won’t hurt” moments (which of course, always hurt) to endless tests and treatment changes. 

You would think, then, that someone like Gunnar was well-prepared for the clinical trial experience. Yet even the most informed and resilient patients need support to navigate the complexities of a trial.  

"No one ever asked a sick person"

Once the first trial began, “it was clear no one ever asked a sick person if the design made sense.” He and his family were trained on tech and PRO systems that didn’t work. The visits were 8-12 hours long, taking place every other week.  

Gunnar’s challenge to study sponsors is to think about patients as a finite resource that is required to feed clinical trials and to move them forward. Without trial participants, there is no progress. It’s in the patient’s best interest to get them into a trial that is set up to succeed. And pediatric patients require an entirely different set of considerations. 

"A failure of pediatric patient engagement"

To set the stage for a good, hard look in particular at the pediatric experience in clinical research, Gunnar shared a cautionary tale.  

When a new drug for cystic fibrosis hit the market, it looked like a clear improvement over the standard treatment at the time: both delivered by nebulizer, but the new drug for just three minutes, three times a day, instead of 30 minutes morning and night.  

It sounds great on paper, but the reality is that most children quickly switched back to the 30-minute treatment. According to Gunnar, it’s simple: “Find any 12-year-old and ask, ‘Would you prefer to take a nebulizer in the middle of the day?’ and any 12-year-old would say, ‘never.’”  

Despite the shorter duration, the mid-day disruption was a tough sell for the pediatric demographic. Though the science may be sound, meaningful engagement throughout clinical development is needed to ensure the experience actually works for the age group.  

Simpler than it sounds

We talk about patient centric clinical trials like it requires moving mountains. But listen to any patient’s lived experience, like Gunnar’s, and you’ll see what it’s really about: 

• Understanding the patient beyond the protocol 
• Communicating clearly and often 
• Building trust through ongoing engagement 

Of course, we need innovation in clinical research. But innovation takes time, and patients need support and adequate engagement now. What can sponsors do to make it happen? 

• Organize tasks and technology to keep participants engaged and adherent 
• Offer step-by-step guidance throughout each stage of the trial to reduce confusion and improve understanding 
• Provide easy access to support for assistance with participation requirements 

Need a helping hand?

From enhancing engagement, to improving adherence, to simplifying participation, we work with sponsors every day to create a better experience for trial participants of all ages and their caregivers. Contact us to schedule a demo of our Patient Hub, or explore what our clinical experience platform has to offer.