Until recently, the first time parents and patients heard about research for a specific disease would be when a doctor told them about a study or they were asked by a researcher if they’d be interested in enrolling their child.
That is changing to a more family-centered approach, and The Children’s Hospital of Philadelphia is leading the charge.
Now researchers and physicians are asking parents and patients to join them as active members of the research team, getting involved from the beginning by helping to frame basic questions such as: What is this research studying? How will it be measured?
“The ultimate goal is for research to help parents with decision-making on which treatment option is best for their child,” says CHOP family consultant Amy Kratchman, who is a parent representative on a national research advisory panel and also active with several local research studies. “So it makes sense for parents to shape the research from the start.”
For instance, a research study on children with autism spectrum disorder (ASD) includes family members. Initially, researcher Katherine Bevans, PhD, planned to ask: “If there was a cure for autism, what would it look like?” But the families who partnered with Dr. Bevans on this project felt that had a negative tone and proposed: “How does ASD impact your child’s life, and how would you like it to change?”
“Engaging parents as fully vested partners in research fundamentally changed our approach,” Dr. Bevans says. “They helped correct some of the researchers’ false assumptions about how kids and parents see ASD and other conditions. They also changed the language we used with research participants. Initially, the changes seemed subtle, but they had an immense impact. Basically, our family partners make sure our research creates knowledge that is meaningful and relevant to kids and families, not just to researchers.”
The federal legislation that created the Affordable Care Act in 2010 also created the Patient-Centered Outcomes Research Institute (PCORI) to conduct comparative effectiveness research to answer questions about which treatments work better. The act also spelled out that patients (and by extension parents of patients) should be named to research panels along with doctors and researchers, and the lay people should be given the support and education they need to fully participate.
CHOP’s Christopher Forrest, MD, PhD, was named chief investigator for a PCORI grant tasked with establishing a pediatric research network called PEDSNet. Eight U.S. children’s hospitals are collaborating on research that uses their electronic health records to more quickly determine the most effective treatments. All PEDSNet research studies will include patients and families.
“For a study that seeks to compare the effectiveness of three drugs to treat a disease, for example, clinicians are most concerned with which one decreases the disease activity while families want to know which one allows their child to best participate in school and activities with his friends,” Forrest explains. “When parents are engaged in research, we can prioritize outcomes by what clinicians and parents feel is important.”
Marie DePascale, whose daughter has been a CHOP patient for 22 years, is a frequent participant in focus groups convened to help form research questions. “Research isn’t just about DNA and finding cures — while those are certainly important,” DePascale says. “It’s also how treatment affects the daily life of kids with disabilities and chronic illnesses. We steer researchers to consider what is important to families. I enjoy helping very much; it gives me a way to share my 22 years of experience and know-how.”
The Pediatric Research Consortium (PeRC) at CHOP, which studies outcomes in primary care, is adding family members to its research teams. Other research groups also have asked families to participate at a deeper level. Grant requests now frequently include family and patient participation.
The Family Relations Department has responded by coordinating parent and patient volunteers through its Family Partners Program. Starting in the spring, it will provide “Research 101” training that introduces the basics of research protocol and terminology, teaches how to give constructive feedback, and focuses on
empowering volunteers to ask questions when a discussion gets confusing.
“It’s easy to get intimidated when doctors and researchers start throwing around acronyms and statistics that a layperson might not understand,” Kratchman says. “We will help prepare volunteers before meetings so they are true partners on the team.”
If you are interested in getting involved in research at its core, email firstname.lastname@example.org.