Close to 200 patients, FDA staff, industry leaders, members of the media and academic personnel listened or participated in the groundbreaking Osteoarthritis Patient-Focused Drug Development meeting on March 8. The meeting was a forum for OA patients to share their experiences with the disease and talk about the signs and symptoms that matter most to them.
Patient-Focused Drug Development & 21st Century Cures Act
The21st Century Cures Act, signed into law in December 2016, has a close tie to patient-focused drug development. One of the Act’s provisions states to incorporate patient-focused drug development strategies, allowing the patient’s voice and experience to be considered during new drug development. Our OA PFDD meeting did just that. The patient perspective is critical in helping the FDA ensure the drug development programs capture the things that are most important topatients.
OA patients were invited to attend in-person and via a live webcast. They shared their experiences by responding to a live poll and discussion questions. Ten patient panelists told stories about how OA impacts their daily lives and discussed the wide range of treatments they have tried, including massage, acupuncture, physical therapy, medications, surgeries, and other therapies.
OA Patients Share their Stories
The youngest panelist, Angela, was 19 years old. She shared her fear for the future knowing that she may have a diagnosis of OA by the time she is 30 due to knee injuries from playing sports in high school.
Another panelist, Donna, shared her experience of having pain for years before her diagnosis.
Ed, who is in this 60s, shared how the OA in his hands limits his ability to grip things such as hammer or a rope. This prevents him from doing things he loves like gardening and boating. He had pain in his hands in his early 30s, but was not diagnosed because the physicians did not recognize the disease. Ed is concerned that after all these years, physicians have not improved much in their ability to recognize and diagnose OA.
Several military veterans spoke about how their training and service resulted in post-traumatic OA.
Each patient had a unique story to tell, but there were common threads throughout. A common topic discussed was the amount of time it took to be diagnosed and a theme emerged that patients wanted options that did not include opiates or joint replacement surgery. They want more choices for how to treat OA and how to prevent the progression of the disease.
Representatives from the FDA participated in the event as observers and speakers. Dr. Suzette Peng spoke about the lack of drugs available to stop disease progression. Dr. Janet Maynard was struck by the numbers of patients that referenced the “invisibility” of the disease.
Invisibility refers to families, friends, and co-workers not recognizing that just because OA patients look fine, doesn’t mean they are. It doesn’t mean they aren’t in pain or suffering. Many OA patients lack a social life and can’t do the things they love because of the limitations of their body so they aren’t seen in the community.
FDA药物评估和研究中心(CDER)的主任珍妮特·伍德考克博士解释了在这次讨论中患者的声音是如何产生影响的。“所有这些都将纳入我们的利益/风险框架，我们用来确定一种新的治疗方法是否应该被批准用于一种疾病。因为毕竟，在考虑一种新的治疗方法是否应该被批准时，必须权衡的是对患者的好处和对患者的潜在危害。”
We will continue to share outcomes and updates from this landmark meeting. To learn more or to view the webcast recordingvisit our website.
Related Resources
- Arthritis Foundation to Host Meeting for Osteoarthritis Patients to Share their Stories with the FDA
- 2016 Accelerating Osteoarthritis Clinical Trials Workshop