Realizing the Benefits of Patient and Family Engagement

To mark our fifth anniversary, we asked patients, caregivers, clinicians, and researchers to describe their experiences with engagement of healthcare stakeholders in research and share their thoughts about building a patient-centered clinical effectiveness research community. The views expressed are those of the author and not necessarily those of PCORI.

How did you become involved in clinical research? 

Amy L. Kratchman: I have a son who was diagnosed with autism 15 years ago at Children's Hospital of Philadelphia. I am now a family consultant there and also the family collaboration director for their Leadership Education in Neurodevelopmental Disabilities program. A research director in the program, Katherine Bevans, PhD, reached out to me when she was writing a proposal for one of the first PCORI funding cycles. She asked if I was interested in being co-investigator to examine what health outcomes are important to families of children with common pediatric conditions, including autism.

Why is it important to bring voices of patients and their families into the research conversation?

Kratchman: We're the ones who live the disease. Who better to ask than us? Why waste the time and energy to study something patients and families don't really care about?

One focus group in Dr. Bevans’s first PCORI project was for parents of children with autism who are severely impaired and fully dependent. A literature review conducted by Dr. Bevans revealed a strong focus on the treatment of aggressive behaviors in children diagnosed with autism spectrum disorder.   However, in the responses given by parents during the focus group, aggression was rated as a much less important challenge than helping their children have a nice life with someone who will care about them and for them. And the researchers listened. Future research might consider intervention strategies to help children have meaningful lives, rather than focusing narrowly on aggression.

As another example of parents’ voices having power, Dr. Bevans started out one discussion with the question, “If there was a cure for your child's illness, what would you want the cure to be?"  We let her know that, while we appreciated her intention, the question sounded negative. We don't look at high-functioning autism, for instance, as needing a cure. And as a result, the researchers changed the question to “What is your biggest challenge in the treatment of your child’s condition?” Seeing that change happen was unbelievable to me.

How can you bring your voice to research in a way that it will be heard, valued, and acted on?

When I recruit other families for PCORI proposals, I try to emphasize that no question is a bad question. When I first went into all this, I didn't understand how you collect data, the difference between a focus group and a survey, and a lot of other terms thrown around by researchers. I was fortunate in having a wonderful Principal Investigator (PI) who helped me feel empowered, so I was able to raise my hand and say, “I didn't understand that, can you explain it?”

Beyond that, I think training is a big piece of success, for researchers and for patients and families. There's often a learning curve for both. Researchers need to learn how to explain things in layman's terms and how to work with patients and families. On the flip side, caregivers need to understand how to work with researchers. Some of this is basic knowledge: terminology, acronyms, processes.

The best thing is having a PI who gets it, who makes it clear to everyone at the first project meeting that we're equal members of the team.  I've also learned how patients and family members can change how a study goes, making the outcome more meaningful to people living with a health condition.

What have researchers learned from you, and what have you learned from them?

Kratchman: In working with researchers, I've emphasized how crucial engagement is: Not just giving patients and families a seat at table, but making fully sure they understand everything that's being discussed. When I'm meaningfully engaged, I can make lots of important contributions, such as changing the phrasing of the research question that patients and families are asked.

And I've learned a lot from investigators. The most humbling part was being reminded how passionate they are about children's health care, how much they want to help. 

What about the future? Will patient engagement become the norm in research?

Kratchman: I hope so. I feel PCORI has really changed things: starting the conversation, laying out best practices, and changing the culture of how researchers work—not isolated in silos, but with patients and families. And the word is spreading. I'm now on a patient engagement advisory panel with representatives from government agencies that are looking at using patients and family members in research. The stage has been set. More and more people are realizing the benefits of patient and family engagement, and the only way to move is forward.