What Is Patient-Centered Outcomes Research (PCOR) —and Why It Matters to Rare Families
By Laura Will, Rare Mom
When most people think of medical research, they imagine doctors in white coats with test tubes and a checklist agenda. But patient-centered outcomes research (PCOR) looks nothing like that. As a participant, it has looked like a recurring monthly zoom call, a virtual room full of patients and caregivers, with the physician researchers there to listen, and take lots of notes. This type of research flips the script. Instead of starting with a newly discovered drug compound, PCOR starts by turning to us—the patients and caregivers—and asking: What matters most to you?
What questions do we want answered? What symptoms do we wish were better managed? What keeps us up at night?
Our rare experiences are at the heart of this approach. It begins with our most honest questions and concerns about lived experiences and then grows into structured research—guided by clinicians, caregivers, and other stakeholders, but rooted in the stories of patients and families.
Asking the Right Questions
When shaping any research question, the process often starts with three seemingly simple questions:
What do we WANT to know? What do we already KNOW? What do we NOT yet know?
This framework helps researchers identify gaps in understanding while ensuring the direction of the study is meaningful to the people most affected.
As a current participant in PCOR, I’ve been amazed at how genuinely curious the researchers are about the nitty gritty of my life as a rare caregiver. The researchers ask follow up questions trying to understand the subtleties in my observations. The researchers don’t just want general insights—they want to know what specifically matters in our family’s day-to-day life. They ask: In your experience, what is hindering your child’s access to better outcomes? What questions do you want us to answer? Where do you see the most urgent need for change? How do we ensure you, the caregiver, are better supported?
My Family’s Story in Research
This is new for us. This is the first time we have qualified to be research participants. The PCOR project I am a participant in is exploring how recreational physical activity impacts my child’s condition and wellness. This initial step is collecting a lot of qualitative data – patients’ stories and caregivers’ perspectives. The questions I would like research to answer span wide—from the physical benefits of movement, to the emotional and social side:
Can physical activity help prevent injuries, reduce hospitalizations, lower the risk of pneumonia?
How might it help my child build friendships and confidence? A sense of self beyond his condition and resulting disability?
At the end of the day, as a rare mom, my focus is nothing short of how I maximize his quality of life. Every hospital stay avoided, every new friendship made, every ounce of independence gained matters; and none of that is simple. Now, as a research participant, I gather with other stakeholders (patients, caregivers, physical activity program managers, physical therapists and physicians) at regular intervals and we explore these concepts extensively, grouping experiences, categorizing priorities, and ultimately generating the research initiatives that will drive future understanding and expertise.
It is so meaningful to know that the struggles and frustrations my child experiences are not just being heard, but are being thoughtfully entered into the calculations of the researcher team. Patient-centered outcomes research is showing me that the questions of rare patients and caregivers are not only being acknowledged—they are shaping the future of medicine; and that gives me hope.
