In Duchenne muscular dystrophy research, the six-minute walk test is considered the standard endpoint to determine if a treatment would be successful. Because walking abnormalities are prominent in patients, researchers measure how far a patient can walk in six minutes to determine if a drug holds promise.

Ian Terry
Ian Terry, senior user experience researcher

When the COVID-19 pandemic occurred, most children and their families could not travel to research facilities for these six-minute walk tests. Instead, researchers began conducting phone interviews to continue their research and grant funding. They learned that, although specific treatments may not improve walk test distance, individuals were sharing stories of how their legs were getting stronger by walking their dog or standing up and playing catch—endpoints that had nothing to do with how far they walked. 

“It took the stress of a complete pandemic shutdown for researchers to begin asking the children and their families, what’s truly important to you?” says Ian Terry, senior user experience researcher at Luna. Terry was instrumental in developing a new methodology based on community-driven versus expert-led research—research driven by what individuals living with the condition determine as a priority. 

Community Driven Innovation™, or CDI, is a participant-led methodology that addresses long-standing problems with traditional research approaches while providing an unbiased, clear understanding of a community’s priorities, values, and challenges.

“Community Driven Innovation uses tools to align research around the needs and priorities of a patient group—or any health-focused community with a research question or problem to solve,” says Terry. “The complex science is built-in and “under the hood,” so to speak, making CDI accessible to anyone, no Ph.D. required.” 

Often, parents, caregivers, and family members don’t think in medical terms, but by using contextual interviews and other listening approaches, CDI was able to reveal families’ top concerns.

Ian Terry, Luna

CDI has been applied to research championed by patient advocacy groups, including KCNT1 Epilepsy Foundation, The Aicardi Goutières Syndrome Advocacy Association (AGSAA), and Bobby Jones Chiari & Syringomyelia Foundation. Luna has also utilized the methodology as the engagement foundation of the Veterans’ Health Priority and Women’s Health Priority communities.

Kaitlyn Esposito, MPH,  who leads programs and research for Bobby Jones Chiari & Syringomyelia Foundation, used CDI to determine their community’s readiness for FDA-approved treatments and clinical trials.  “We learned a lot of really interesting things, including just how complex the symptoms are in these conditions.  We knew it was complicated but were surprised at how complicated it truly was,” shared Esposito.

“Using CDI, the Aicardi Goutières Syndrome (AGS) community uncovered the importance of muscle tone management as a top priority,” Terry says.“ Often, parents, caregivers, and family members don’t think in medical terms, but by using contextual interviews and other listening approaches, CDI was able to reveal families’ top concerns.”

Historically, the clinicians or researchers would be the one who determines the research question. With CDI, the community can become research partners and advocate, in a data-driven way, for the real challenge or question they need answered.

In rare disease groups, researchers can quickly get funding to focus on a topic that may not already be supported. Those researchers often embark on a project with the most accessible endpoint to demonstrate results. However, that endpoint may not be what’s essential to that rare disease community, as shown with the walk test in the Duchenne muscular dystrophy community.

It’s easy to talk about participant-focused research. It is easy to speak about being patient-centric,” says Terry. “But CDI, for the first time in the health space, gives researchers a tool that marries true participant-focused research with patient engagement in a way that is both scientifically driven and a low burden to affected individuals, their families, and caregivers.”

About Luna

Luna’s suite of tools and services connects communities with researchers to accelerate health discoveries. With participation from more than 180 countries and communities advancing causes including disease-specific, public health, environmental, and emerging interests, Luna empowers these collectives to gather a wide range of data—health records, lived experience, disease history, genomics, and more—for research.

Luna gives academia and industry everything they need from engagement with study participants to data analysis across multiple modalities using a common data model. The platform is compliant with clinical regulatory requirements and international consumer data privacy laws.

By providing privacy-protected individuals a way to continually engage, Luna transforms the traditional patient-disconnected database into a dynamic, longitudinal discovery environment where researchers, industry, and community leaders can leverage a range of tools to surface insights and trends, study disease natural history and biomarkers, and enroll in clinical studies and trials.