“Imagine how many lives could be saved if we spent as much energy on rare disease awareness and education as we do on celebrity culture.”

Patient advocacy groups (PAGs) are critical in bridging the gap between patients, clinicians, and the pharmaceutical industry, but their unique challenges often go unnoticed. Joyce Kullman, Executive Director of the Vasculitis Foundation, sat down to discuss her journey in patient advocacy, the evolving role of PAGs, and the insights researchers and industry professionals must grasp to collaborate effectively with advocacy groups.

Kullman’s entry into patient advocacy was deeply personal. Her father’s 1994 diagnosis of granulomatosis with polyangiitis (GPA), after 11 months and visits to 11 different doctors, revealed the urgency of greater awareness and care for rare diseases. Already familiar with the Vasculitis Foundation - then the Wegener’s Granulomatosis Association - she moved from a volunteer role to board member and eventually executive director. Her journalism background proved pivotal in crafting compelling narratives to amplify the Foundation’s impact.

"The biggest challenge is always funding - how to raise it and align it with our mission. But it doesn’t stop there. Raising awareness, encouraging research, and educating both patients and clinicians are continuous efforts for organizations like ours.”

Reflecting on the evolution of patient advocacy in research, Kullman highlights a transformative shift that patients are no longer an afterthought.

“In the past, studies would launch without any input from patients. Now, the patient’s voice is included from day one. We now get asked by pharma, ‘Will this study work for patients? Is it designed for their needs?’ Successful trials minimize the burden on patients, from reducing travel demands to covering associated costs.”

This patient-first approach not only improves recruitment but also fosters a deeper partnership between patients and researchers.

Kullman emphasizes the importance of “putting a face to the research.” Through focus groups, advocacy councils, and personal storytelling, the Vasculitis Foundation helps pharma partners and researchers see beyond clinical data to the lived experiences of those with vasculitis.

“When you meet someone who could benefit from a drug, it changes your perspective - the human connection ensures that study designs are not only scientifically sound but also empathetic to the realities of living with a rare disease.”

For researchers and industry partners, Kullman offers three critical takeaways:

• Patients want partnership: Rare disease patients are eager to contribute to research, but they seek a role as collaborators, not passive participants.
• Education empowers: Both patients and healthcare professionals benefit from better education on rare diseases. Not all patients can access specialized centers, making local clinicians’ awareness vital.
• Awareness saves lives: Awareness of rare diseases like vasculitis remains dangerously low, hindering early diagnosis and treatment.

“Imagine how many lives could be saved if we spent as much energy on rare disease awareness and education as we do on celebrity culture.”

Despite the hurdles, Kullman remains optimistic. She acknowledges the progress pharmaceutical companies have made, particularly in integrating patient advocacy into their processes. But she believes there’s still work to be done.

“Ultimately, we’re all striving to improve patients’ lives. The more we listen to patients and PAGs, the better outcomes we’ll achieve together.”

Under Kullman’s leadership, the Vasculitis Foundation has grown in size and stature and now employs 11 full-time staff to support its various initiatives. The Vasculitis Foundation has also been a key supporter of Sam (www.steroidsandme.com) and participated in focus groups and the initial pilot program that was published at ACR2024.