Health IT

Study looks at how autistic children, caregivers can shape clinical trial design

A clinician, a parent and an 8-year-old child with autism spectrum disorder. Odds are good they have fairly different perspectives, so the way they each would define the criteria for a successful healthy outcome of a clinical trial would probably be different too.

One PCORI-funded study being undertaken at the Children’s Hospital of Philadelphia wants to develop a better clinical trial design  so that outcomes are meaningful not only for the scientists, but also for parents and children participating in them.

The study will develop and evaluate novel methods for eliciting and prioritizing children’s health outcomes that are comprehensible to both children with autism spectrum disorder and clinicians, measureable, and ready for integration in clinical and CE research, according to a summary of the project.

A revamped clinical trial design would prioritize what children with autism spectrum disorder and their caregivers consider to be meaningful health outcomes with the drug or device under development. That criteria would give parents and children a more more meaningful stake in improving their quality of life. Dr. Katherine Bevans spoke about the study in a phone interview with MedCity News.


“A lot of times parents and children identify different sets of desired outcomes that don’t make their way into clinical research,” Bevans said. “That could include things like wanting to improve peer relationships or connections with other kids, or body image.” Often that is not included because it’s not the traditional indicator of success or researchers just don’t ask. “I think for a long, long time, people have had the inaccurate assumption that kids can’t report on their own health,” observed Bevans.

The autistic children at the center of the study are verbal and iPads and illustrations are used to engage them. This project is very much in line with the patient-centered movement, Bevans said. Its aim is to boost the involvement of patients, pediatricians and caregivers in clinical trials to be not just research participants but to be active, fully vested members of the research community.

One goal of the two-year study is to better define certain terms so that when physical activity, peer interactions and friendships are mentioned, everyone understands what they mean from the clinician to the child patient. Although, superficially, people know what these terms are, defining these terms and framing questions to get these insights will be a challenge.

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