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April is Sarocoidosis Awareness Month, an opportunity to raise awareness, promote understanding and research, and create hope for the affected patients and their families.
This year’s theme is “Facing Sarcoidosis Together.”
To explore how effective collaboration between the biopharmaceutical industry, the advocacy community, and other key stakeholders can speed the path to a cure, Karin Hoelzer, Senior Director of Patient Advocacy at the Biotechnology Innovation Organization (BIO) and Tricha Shivas, Chief of Staff and Strategy at the Foundation for Sarcoidosis Research, explore the vital progress in the diagnosis and treatment of sarcoidosis, a rare autoimmune disease, fueled by effective cross-sector collaboration.
The quest to find a cure for sarcoidosis
Sarcoidosis is a rare inflammatory disease characterized by the formation of granulomas—tiny clumps of inflammatory cells—in one or more organs of the body. When the immune system goes into overdrive and too many of these clumps form, they can interfere with an organ’s structure and function. Left unchecked, this chronic inflammation can lead to fibrosis, which is the permanent scarring of organ tissue. Sarcoidosis affects the lungs in approximately 90% of patients, but it can affect almost any organ in the body. Despite increasing advances in research, sarcoidosis remains difficult to diagnose with limited treatment options and no known cure. The disease impacts an estimated 150,000–200,000 people in the U.S., and disease presentation and severity can vary widely among patients. In some cases, the disease goes away on its own. In others, sarcoidosis can be debilitating and life-threatening.
The Foundation for Sarcoidosis Research (FSR) is dedicated to finding a cure for sarcoidosis and improving care for sarcoidosis patients through research, education, and support.
“There is so much we still don’t understand about this disease, and there is currently no cure,” explained Shivas. “That is why since its establishment in 2000, FSR has fostered over $7.2 million in sarcoidosis-specific research efforts.”
Cross-sector collaboration is key to expedite the development of a cure
“Patients have no time to waste,” explains Shivas. “We need better treatments now and we owe patients our very best efforts to make this happen quickly and effectively. Through collaboration and candid conversations, we can shave years off the long research and development processes.”
The key focus of these initiatives for FSR is on growing the understanding of sarcoidosis, addressing the causes of the disease, countering the suffering of patients, and advancing the potential for a cure.
As Shivas describes, “We are actively working with the world leaders in sarcoidosis, investing in innovative, patient-centered research efforts, and providing educational resources, support, and opportunities to accelerate research to patients worldwide.”
And, this strategy is proving successful.
“FSR-supported research in omics, biomarker identification, and disease model creation have helped to deepen our grasp of the disease mechanisms, potential therapeutic pathways, and understanding of why some forms of sarcoidosis advance into multi-system sarcoidosis while others level out, remit or seem to improve over time.“ says Shivas.
She continues, “FSR is striving to create game-changing collaborations and partnerships with the pharmaceutical industry, biotech companies, regulatory agencies, medical institutes and professionals, academic institutes, researchers, and patients from across the globe to speed the path toward a cure.”
Early collaboration is key to inform and de-risk clinical trials
Successful collaborations help to de-risk drug development and can avoid potentially perilous roadblocks later on. This includes identifying and breaking down barriers that delay start-up for new trials or slow down the recruitment and retention of trial participants.
“Over the last 10 years, FSR has grown our collaboration with biotech and pharmaceutical companies by building a comprehensive strategy to de-risk clinical trials in our space,” states Shivas. “Through the establishment of the FSR Sarc-Patient Reported Outcomes Registry which has over 7,000 participants from over 60 countries, FSR has been gathering vital information on patients’ diagnostic and treatment journeys, their lived experiences, and the most pressing needs for treatment and drug development.”
FSR is working alongside researchers, industry partners, and the FDA to support early collaboration with the patient community, including the establishment of valid endpoints, functional eligibility criteria, and the most meaningful data analysis strategies reflective of the needs of the sarcoidosis community.
“FSR is in constant communication with the FDA and has hosted multiple patient listening sessions and most recently, an Externally-Led Patient Focused Drug Development Meeting,” explains Shivas. “FSR is also a part of working groups with the FDA and policy advocates committed to finding the alternative strategies to expedite clinical trials in rare disease. “
At the trial site level, FSR is working to de-risk clinical trials through the establishment of FSR Global Sarcoidosis Clinic Alliance (FSR-GSCA), a member program consisting of clinics, hospitals, individual providers, patients, and caregivers committed to finding a cure and offering evidence-based, patient-centric care for those living with sarcoidosis. FSR provides logistical support for registry establishment at the site level, gathers site feasibility information to help in site selection, and provides educational and training programs to increase clinical trial readiness.
Listening to patients to identify and break down barriers to clinical trial participation
As Shivas explains. “Through education and open fora, FSR has fostered a remarkable interest and commitment to clinical trial and research participation. FSR has trained patient volunteers to serve as expert reviewers and commentators to provide feedback on clinical trial protocols, consent forms, and recruitment materials from the perspective of patients and caregivers to ensure that the patient voice and patient understanding are central to every step of trial design. “
FSR’s efforts to identify and break down barriers to trial participation by no means end here. In 2024, FSR launched the Coalition for Clinical Trial Equity (CCTE) which now has 33 member organizations from medical societies, patient advocacy groups, industry, and academia.
“FSR has taken our commitment to expanding clinical trials beyond rare disease with a commitment to reducing barriers to allow for better representation,” says Shivas.
The CCTE is focused on addressing barriers to trial participation identified by the member organizations, and builds on a framework initially created during FSR’s Ignore No More: ACTe Now! Campaign. (ACTe stands for Advance Clinical Trials for Equity in Sarcoidosis.)
During this campaign FSR conducted the first of its kind, IRB-approved national patient survey for Black Americans to better understand the challenges and experiences Black Americans with sarcoidosis face as it pertains to clinical trials and their disease journeys. Sarcoidosis disproportionately impacts Black people, in particular women, who have both a higher disease prevalence and often experience more severe disease and worse health outcomes. Better understanding the underlying causes may hold valuable clues for a cure.
FSR also conducted a Key Opinion Leaders Thought Workshop (KOL) and Patient Focus Group to explore the findings in greater depth and to identify recommendations for how to improve clinical trial access and overall care of Black sarcoidosis patients.
One of the key early successes for the CCTE has been to receive clarity from the Department of Labor (DOL) guaranteeing those eligible for Family Medical Leave will maintain job security when participating in clinical trials through the Family Medial Leave Act (FMLA) protections. FSR, in collaboration with lawyers, identified that these barriers stemmed from the lack of clarity on whether the concept of “therapeutic benefit and treatment” extends to clinical access to clinical trials. DOL provided this clarification in a November 8, 2024 letter in direct response to FSR.
“Understanding this clarification is something everyone who is recruiting clinical trials should be aware of,” Shivas said, “as it guarantees that all who qualify for FMLA leave, whether patient or caregiver, will have their jobs protected while participating in clinical trials.”
FSR and CCTE are working to spread the word to expand the opportunity for clinical trial participation. FSR and the CCTE have also built a program which encourages corporate America to provide time off for clinical trials. The program recognizes companies that provide at least one day to employees through traditional vacation and PTO or through corporate social responsibility and volunteer programs.
“The uptake to date has been encouraging,” said Shivas; “We already have 6 companies who have agreed to provide their employees with time off for trial participation. Anyone interested in learning about the Coalition for Clinical Trial Equity should visit www.ignorenomore.org and complete the interest form.”
“Collaboration is key to accelerating clinical trials in rare diseases like sarcoidosis,” concludes Shivas. “FSR is proud of our efforts and grateful for the patients, caregivers, clinicians, researchers, industry, and regulators to carve a new path towards better therapies and improved patient outcomes. We are immensely grateful to Bio for creating an environment that nurtures collaboration and encourages collective innovation. The future is brighter for all impacted by chronic and rare disease because of these extraordinary efforts.”
