Randomized Controlled Trials

Research background: Randomized controlled trials are important, but it is difficult to recruit pediatric participants

Randomized controlled trials are studies that randomly assign participants to an experimental group or a control group. The only expected difference between the control and experimental groups is the outcome variable being studied.

This method is considered the gold standard for determining efficacy of treatment. Unfortunately, it has been historically difficult to recruit patients to participate in surgical trials. Less than 1% of patients participate in surgical trials; even fewer in pediatric surgical trials.

A study by researchers in the Department of Pediatric Urology at Children's Hospital Colorado was the first to assess barriers to participation in surgical randomized controlled trials and to assess whether those barriers are unique compared to medical randomized controlled trials.

Research methods: A study of parents, pediatric urologists and referring providers

Study participants included a volunteer sample of parents at Children's Colorado (via focus groups), regional pediatric urologists (via focus groups) and regional referring pediatricians (via surveys). Primary or secondary caregivers of children under the age of 2 with grade 3 or 4 congenital hydronephrosis seen in the Department of Urology between 2010 and 2012 with less than a year of follow-up were eligible to participate.

The primary goal of the study was to explore potential barriers to participation in surgical randomized controlled trials and to identify potential differences compared to medical randomized controlled trials. General factors considered in the study included:

• Intrinsic characteristics of treatment options
• Personal impressions
• Economic concerns
• External information

Research results: Potential barriers to participation in pediatric surgical trials identified 

 Participants

 Key themes

Researchers identified four key themes related to what would influence participation of children in surgical randomized control trials:

• Responsibility to my child
• Responsibility to the field
• Responsibility to my patient
• Irreversibility of surgery

Responsibility to my child

Parents feel it is the obligation of the physician to inform them about relevant clinical studies. Parents trust and support their child's primary care provider as the medical provider who knows their child best. 

Parents felt that participation in a randomized control trial could lead to closer follow-up and possibly better care. However, they feared their child would be a "guinea pig" or be under-treated if enrolled in a trial.

All parents felt the burden of making a decision for someone else and felt a need to be able to tell their child they made the "best choice." This need to be in control of the decision and trust in the physician to help them make the right choice ultimately limited parents' willingness for their child to participate in a randomized controlled trial. 

Responsibility to my patient

Physicians acknowledged their obligation to inform parents about relevant clinical studies to provide the best available care for their child. To reduce the risk of coercion, providers wished to leave the parental discussion to a member outside the clinical team. Parents were comfortable learning about a study from a front desk person, nurse or research coordinator. 

Providers noted that the nature of the clinical setting could limit the recruitment to randomized clinical trials. Providers expressed concern about the uncertainty in treatment that comes with clinical trials and a parent's ability to accept it, regardless of clinical or research setting. 

Like parents, providers felt participation could benefit patient care through close follow-up. Providers were deeply concerned about the age of the child and ability to participate in the decision.

Responsibility to the field  

Providers consider research part of routine care. Parents felt it was important to teach their children altruism and help similar children with the same condition. Parents also believed research offered an opportunity for support from other families with children who have the same condition. 

Some providers were concerned that any unnecessary risk associated with a randomized control trial could harm the parent/physician relationship. Pediatricians preferred to refer patients to a practice with ongoing research due to a perceived benefit in care.

Irreversibility of surgery  

Parents felt the risk of surgery and anesthesia in young children outweighed surgical benefits. In addition, surgical randomization was viewed as fundamentally different than other randomized treatments because surgery is irreversible and was perceived as having increased risks. Providers did not identify a difference in the potential benefits and harms of medical versus surgical treatment. They also felt the severity of the disease and potential for mortality could influence a parent’s willingness to participate in randomized trials. 

Research conclusion: Fewer barriers to observational studies than surgical trials

Both parents and providers expressed more comfort with observational studies compared to randomized controlled trials. Unlike providers, parents felt that surgical trials were inherently more concerning due to the irreversible nature of surgery and perceived higher associated risk. 

Parents and providers are more likely to participate in studies when joint decision-making can take place even when there is no "right answer." These findings suggest that recruitment in surgical randomized clinical trials may be improved if there is an option for participation in an observational arm. 

The study was published in the June 2016 issue of Journal of Pediatric Urology.