At FSR forum, hundreds hear of sarcoidosis’ unmet needs, challenges
Almost 350 patients, caregivers and others join drug-development meeting
Nearly 350 people joined a virtual public meeting that the Foundation for Sarcoidosis Research (FSR) ran as part of a U.S. Food and Drug Administration (FDA) program to discuss the unmet needs and challenges faced by those living with sarcoidosis.
In the Externally Led Patient-Focused Drug Development meeting, held on Oct. 28, patients and caregivers repeatedly voiced an urgent need for new treatment options, and the disease symptoms they would like future treatments to address.
A video recording of the over five-hour meeting — hosted by Mary McGowan, FSR’s CEO, and moderated by John Carlin, who hosts the FSR Sarc Fighter podcast — also is available online.
“This meeting marks a critical milestone for sarcoidosis patients and caregivers,” McGowan said in a foundation press release. “The insights shared point to the urgent need for better therapies for the approximately 175,000 individuals nationally living with sarcoidosis and will support the creation of improved treatments that can enhance their quality of life.”
Sarcoidosis specialist, patients talk of disease’s varied impact
The FDA launched the Patient-Focused Drug Development initiative in 2012 as a way to systematically obtain patients’ perspectives on their disease and currently available treatments. In addition to FDA-led meetings under this initiative, the agency encourages patient organizations to organize their own, externally led drug-development meetings.
These meetings follow a “town hall” format, where most of the time is spent hearing from patients and caregivers about symptoms and how they limit their daily lives, available treatments and their issues, and opinions regarding an ideal treatment.
Sarcoidosis is a rare disease where small clusters of inflammatory cells, called granulomas, form in the body’s organs, interfering with how they function. Despite advances in research, it’s still difficult to diagnose sarcoidosis or treat the disease, with patients calling for new treatment options that might allow a better life.
Opening the patient-focused meeting, Lisa Maier, MD, a pulmonologist at National Jewish Health in Colorado and a member of FSR’s scientific advisory board, provided an overview of how sarcoidosis develops and manifests, and identified opportunities for future research.
Diagnostic delays noted as frequent and damaging
“One of the issues that I think we continue to hear from patients … is a delay in diagnosis of sarcoidosis,” said Maier, who is also a founding member of the FSR Global Sarcoidosis Clinic Alliance, “it still remains one of the great masqueraders.” A 2003 study showed that “up to half of the patients required three physician visits before they were diagnosed with sarcoidosis,” Maier added.
Such delays have consequences, allowing respiratory and other disease symptoms to worsen. In addition, when people “haven’t been able to get that diagnosis as quickly, they don’t trust the healthcare systems, they tend to use more healthcare dollars, and have more physician visits,” Maier said.
Maier also touched upon the results of a 2022 FSR patient survey that found an earlier diagnosis, easier access to expert care, less reliance on corticosteroids as a treatment, and more opportunities to take part of clinical trials were top priorities in terms of their needs.
Patients and caregivers joined the conversation, sharing their own stories, participating in live polls, and calling in or sending written comments about their perspectives. Concerns voiced ranged from managing disease symptoms and the side effects of current treatments to how the disease interfered with their ability to work.
Patients speak of lost jobs, side effects, poorly understood disease
“I worked as an EMS manager for 19 years, a job that I absolutely loved, and miss, every day,” said Brandi, noting she is a fourth generation “sarcoidosis warrior.” “When I am feeling my worst,” she added, “I am unable to walk unassisted, I can’t see well, I slur my words, and I have a massive amount of nausea.”
“So many people think you just have sarcoidosis, that nothing, it’s not cancer. No, sarcoidosis can really impact one’s life,” said Cheryl, who recounted spending 45 days in a hospital not being able to talk or walk.
“I just wish there could be something developed to help us … anything that could be designed specifically toward [the] side effects of this condition,” said Debora, mentioning short-term memory problems. She also spoke of having purchased an SUV for her family only to later to trade it in, because climbing into the vehicle had become too difficult.
Both McGowan and Carlin noted that participants spoke of multiple organs being affected by the disease.
Healthcare providers, industry leaders, and disease advocates also attended the meeting.
The FSR is accepting additional comments from patients and caregivers through Dec. 2. A comments form can be found on the webpage featuring the recording.
The foundation will develop a summary report to capture the shared oral and written perspectives, called the Voice of the Patient, which it plans to publish early next year as a resource in work toward more effective sarcoidosis treatments.
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