Contributed Commentary by Scott Gray, Clincierge 

January 13, 2023 | A clinical trial’s path from initial design through study execution and completion is exceptionally complex. From preclinical research to site selection, patient enrollment, and oversight of critical data collection, there are many moving parts for pharmaceutical sponsors and clinical research organizations (CROs) to consider. In addition, poor patient recruitment and low participant retention rates further complicate the process, especially in trials focusing on children with rare diseases.  

Approximately 80% of all rare diseases are genetic, with about half affecting pediatric patients. Due to the genetic component of these disorders, more than one child within a family unit can suffer from the same rare condition, amplifying the already complicated process of clinical trial participation.  

Pediatric trials are notoriously challenging, boasting unique barriers to successful completion. Since patients in pediatric trials are under 18 years old, participation commonly affects the entire family rather than just the patient themselves. The parent of a child with a rare disease wears many hats, including but not limited to chauffeur, advocate, researcher, nutritionist, pharmacist, and teacher. 

Challenges to Pediatric Trial Participation  

Many perceived and actual barriers to pediatric trial participation exist, resulting in reduced enrollment, lower retention and completion rates, and fewer advances in children's medicine, consequently depriving children of much-needed treatments.  

The National Organization for Rare Disorders (NORD) published a 30-year comparative analysis concerning the barriers to rare disease diagnoses. The research reported that of the over 7,000 rare diseases identified, 90% had no treatment, and nearly half of all pediatric trials go unfinished or unpublished. With such a critical need for advancements in pediatric medicine, where does one start?  

Removing the Barrier of Travel to Site Visits   

Some local physicians may have the expertise to diagnose and treat rare diseases. In other instances, patients must seek care from a specialist practicing at a more prominent medical center. These facilities tend to be in metropolitan areas and can present logistical barriers for those requiring frequent appointments, including transportation, lodging, and other accommodations.  

In the analysis mentioned above, NORD also reported that 39% “of respondents needed to travel 60 or more miles in 2019,” and a BMC Family Practice study noted that most parents’ work schedules are affected by their child’s medical treatments (DOI: 10.1186/s12875-016-0488-x). Time spent traveling, meeting with various specialists, and completing the many medical assessments add up quickly for families. With more than 30 million U.S. workers reporting they do not receive paid sick time, parents can face the added financial burden of lost wages and possible job loss due to time away from work.  

Keeping Trial Participants Engaged   

The challenges of recruiting pediatric patients and maintaining their clinical trial participation are often critical, as most patients suffering from rare diseases have few treatment options. Their symptoms are often progressive, leaving them tired and weak. Retention is scientifically and economically vital for pediatric trial success, and patient dropouts remain a pressing issue across the industry.  

Rare disease pediatric patients and their caregivers also drop out due to emotional burdens like anxiety and stress over missing school or work, invasive medical procedures, and economic insecurity. Managing the complex logistics of clinical trial participation is often handed to patients and their caregivers. Many cannot contemplate trial participation, given the illness's seriousness and the disease's unknown progression.  

In many pediatric clinical trials, families and caregivers are tasked with handling the burdens of trial participation. However, families can benefit greatly from someone assisting them with their emotional, logistical, and financial challenges. Relying on a single point of contact who is good clinical practice-certified and understands the study protocol will afford immense insight into the unique support needs of individual pediatric patients and their families.  

Participants and caregivers enrolled in such studies receive services like:   

It is important to recognize families will incur costs during their time away from home for things like meals, travel expenses, parking, tolls, and other incidentals. These costs can quickly add up and may seem overwhelming. Families enrolled in supported trials could receive expense reimbursements for these out-of-pocket costs. The personalized support of a patient concierge makes the reimbursement process quick and stress-free, allowing families to focus on caring for their children.  

Families caring for a child with a rare disease will face many challenges, but there is hope for those who can access experimental treatment via a pediatric clinical trial. Implementing a trial model pairing the patient and their family with a trusted partner to help them navigate the experience allows them a fighting chance at a life-changing treatment.  

Scott Gray is the co-founder and CEO of Clincierge, the leader in patient support services for clinical trials. Since 2015, Clincierge patient coordinators have managed logistics and reimbursements for more than 300 clinical trials worldwide. Gray can be reached at scott.gray@clincierge.com.