Children's Hospital Colorado

Randomized Controlled Trials

Urology | January 05, 2018
This image shows a family talking to a healthcare provider about pediatric clinical 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 one percent 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.

First Study to assess barriers to participation in pediatric surgical randomized trials
<1% Of eligible patients participate in surgical trials
<0.2% Published pediatric surgical literature includes 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

This image shows that 8 parents, 6 pediatric urologists participated and 24 referring pediatricians participated in the study.

 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. 

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. 

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.

Tags: Urology


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