New report highlights unmet needs and treatment burdens in sarcoidosis
Patient voices call for safer therapies and more patient-centered clinical trials
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Sarcoidosis can significantly affect day-to-day life, and commonly used treatments may carry substantial risks of side effects, according to a report from the Foundation for Sarcoidosis Research (FSR).
The report also finds that clinical trials testing new sarcoidosis treatments often do not fully reflect patient priorities, highlighting the need for more patient-centered approaches to trial design.
Titled Voice of the Patient, the 50-page report summarizes discussions from an externally led patient-focused meeting held in collaboration with the U.S. Food and Drug Administration (FDA) in late 2024. That meeting brought together nearly 350 patients, healthcare providers, industry partners, and advocates to elevate patient perspectives and identify priorities for future research.
“The insights captured in this report are pertinent to advancing sarcoidosis research and drug development,” Mary McGowan, president and CEO of FSR, who co-hosted the 2024 meeting, said in a foundation press release. “Patients have shared the challenges they face every day: delayed diagnosis, debilitating fatigue, chronic pain, and the heavy burden of long-term [corticosteroid] use. Their voices must guide the next generation of therapies and clinical trials that truly reflect the lived experience of this disease.”
How sarcoidosis affects daily life and well-being
Sarcoidosis is a chronic inflammatory disease marked by abnormal clumps of immune cells, most often in the lungs. However, it can also affect many other organs throughout the body, leading to a wide range of symptoms.
The report highlights that many sarcoidosis symptoms interfere with daily life. Fatigue was identified as the most debilitating physical symptom, often making it difficult for people to maintain employment, straining personal relationships, and limiting participation in everyday activities. Severe pain, cognitive difficulties such as “brain fog,” and breathing problems were also reported to significantly affect quality of life.
“Many patients describe a type of anticipatory grief marked by fear of flare and concerns that additional progression or changes in their disease will further reduce their quality of life,” the report states.
Anti-inflammatory medications known as corticosteroids remain the most commonly used treatment for sarcoidosis. These medications, including the sarcoidosis-approved prednisone, work by mimicking cortisol — a hormone the body produces in response to stress.
However, long-term corticosteroid use can lead to serious side effects, including weight gain, skin changes, and mental health problems. The report notes that these burdens have led many patients to call for safer, sarcoidosis-specific treatment options.
The report also notes that because most sarcoidosis treatments are used off-label, patients and their families also face additional financial strain, especially as the disease can limit a person’s ability to work.
Why current sarcoidosis trials may miss patient priorities
Clinical trials are the gold standard for testing the safety and effectiveness of potential new therapies for sarcoidosis. However, the report notes that many sarcoidosis trials are designed in ways that make participation difficult for patients — such as restricting enrollment to people with specific patterns of organ involvement.
The report also highlights that trial designs often fail to reflect the real-world experiences and priorities of people living with sarcoidosis.
In particular, trials often rely on standardized lung function measures, such as forced vital capacity (FVC), which reflects how much air a person can exhale forcefully in one breath. However, the report notes that for many patients, lung symptoms are not the main source of day-to-day burden.
Instead, people with sarcoidosis frequently prioritize disease stability, slower progression, reduced fatigue and pain, and fewer medication side effects when evaluating new therapies.
As a result, the report calls for a more holistic approach to trial design — one that considers symptom burden, quality of life, and the ability to reduce corticosteroid use, outcomes that patients often value most.
The FSR hopes that the report will help guide the development of safer, more effective therapies that better address patients’ needs.
“We are here not just to listen but to act,” McGowan said. “Together, we can transform the lives of those battling sarcoidosis.”
