Hundreds of patients and their families, FDA staff, industry leaders, and researchers convened in Washington, D.C. to participate in the externally-led JIA Patient-Focused Drug Development (PFDD) meeting on August 2, 2018. The meeting served as a kickoff event to the 4-day Arthritis Foundation JA Conference East. Juvenile idiopathic arthritis (JIA) is the most common type of juvenile arthritis (JA). In many children, it’s a life-long illness with a high risk of disease and treatment-related morbidity. There is no cure, and treatments for children are limited.
This meeting, which is part of our strategy to help find a cure for this serious disease, provided an opportunity for patients, patient advocates, and caretakers to talk about the symptoms and issues that matter most to them. JIA parents and their families shared information about the impact of JIA on daily life, the symptoms that are most challenging, experiences with currently available treatments and the need for new and different treatments.
JIA patients and their families were invited to attend in-person and via a live webcast. They shared their experiences by responding to a live poll and discussion questions. Three patients and nine parent panelists told stories about how JIA impacts their daily lives and discussed the wide range of treatments they have tried.
JIA Patients Share their Stories
Nick, a high school senior, was diagnosed about a year ago. He described the emotional and physical challenges he has had to overcome – at times being unable to get up, dress himself, or chew his food. He said the hardest thing for him was to see his mom cry because she couldn’t take away his pain.
Andrew, diagnosed at age two, will start college in the fall. He wants to get into clinical pharmaceutical research. He described the challenges in finding a college that would be accessible due to the physical challenges of his JIA.
Harley, diagnosed at age 2, is now a senior in college, preparing to attend law school in 2020. While she maintains an active life style in spite of her disease, she shared some of the difficulties she has experienced in her 19-year journey with JIA. She described a point in her life where she needed to use a scooter to get around because her hips were damaged, and she couldn’t walk.
All of the parents described how JIA has changed not only their children’s lives, but the lives of their entire families. Most have become strong advocates for research and finding cures for JA. Their key message is that many families are out of treatment options and are desperate for more therapies.
Dr. Nikolay Nikolov, an associate director for rheumatology within the Center for Drug Evaluation and Research (CDER) at the FDA explained how the patient voices in this discussion make an impact in deciding the endpoints for research, identifying which outcomes are important to patients and their families. Dr. Rachael Glaser, a clinical team leader with Dr. Nikolov’s division, summarized the importance of the information gained from the JIA PFDD.
We will continue to share outcomes and updates from this landmark meeting. To learn more or to view the webcast recording, visit our website. The results of this meeting will be shared in the JIA Voice of the Patient Report, available on our website near the end of 2018/beginning of 2019.
Thank you to our partner and co-host, CARRA, for helping to produce an impactful meeting between patients, partners and industry leaders.
- More About the JIA Patient-Focused Drug Development Meeting
- More About JA Research
- Learn More About Participating in Research