When a patient learns they have a rare disease, sometimes after years of misdiagnosis or being told “we don’t know what’s wrong,” they often feel relief thinking they can now begin the path to healing. But that is when the real horror begins – with some diseases such as sarcoidosis, there is often no clear path to healing; and sometimes no chance of healing at all. This devastating situation is common for hundreds of thousands of sarcoidosis patients across the world. What can save these patients, many of whom face debilitating futures and even premature deaths? Frankly – there are very few answers for treatment let alone a cure: Doctors don’t know, researchers don’t know, and certainly patients don’t know.
The Foundation for Sarcoidosis Research (FSR) makes it our mission to find answers – we know too many patients and surviving family members who are desperate for improved treatments, research, faster diagnoses, and for hope. FSR has a very strategic and expert-driven response to these needs. We seek to de-risk the sarcoidosis space so that more research and more therapies can emerge, successfully moving the needle forward to help these patients.
FSR is the nation’s leading nonprofit organization dedicated to finding a cure for this disease and to improving care for sarcoidosis patients. Since its establishment in 2000, FSR has fostered over $6.5 million in domestic and international research efforts and has worked diligently to provide resources to thousands.
Our refined approach includes a specific research agenda that identifies and addresses the major gaps in sarcoidosis research. These are key components missing in the field which, if produced, would be transformational toward advancing research to help patients. FSR facilitates game-changing advances in sarcoidosis research, resulting in promising therapies that can move quickly from the laboratory to the patient. By providing education, advocacy, resources, direct funding, and collaborations, FSR counters the suffering of patients, and advances the potential for a cure.
Mission statements can sometimes be ethereal, therefore it can be difficult to assess the direct impact for stakeholders. How is the hard work FSR staff and volunteers are doing on a day-to-day basis accelerating research? How is that work living up to our mission and meeting the needs of patients, clinicians, and investigators? The following pages outline the true stories of stakeholders and illustrate how FSR’s efforts are addressing their real-life needs.
Removing Barriers
“The last 12 years of my life have completely revolved around how much Prednisone my doctor has given me on to combat my sarcoidosis. Sometimes I can function well and go grocery shopping, babysit my nieces, do things that give me enjoyment, even when breathing is hard. But when my sarcoidosis symptoms get too horrible, I have to go back up on the steroids, the side effects take over, and my whole life crumbles again. I need to choose whether to suffer from the disease or suffer from the medicines – most days it is just a question of which is worse. Every day I wait to hear there is finally a new drug for me – I hope it is soon!”
Patient, South Carolina
One of the major blockages in the sarcoidosis research is that there is not a solid, widely accepted disease model that the FDA accepts for new drug testing. And without this model, there is little to no chance of any new drug coming down the pipeline. While currently there are two drugs that are, FDA approved directly for sarcoidosis, there remains a massive need for therapies with less toxicity and/or that serve more types of the disease. Not only does this block chances of a new, more successful drug for patients, but the lack of a model also impedes any research from the start. It is just another huge gap in the field of sarcoidosis research – a lack of scientific resources means a lack of investigators and research activity. FSR provides major funding to investigator-led research around a disease model. Investigators around the world have been invited to submit proposals within this arena, and FSR will select several proposals for approximately $450,000 worth of funding. Once the early steps toward model development is funded by FSR, other stakeholders including industry, the government, and investors will more readily enter the field bringing larger funding initiatives.
Providing Data
“As an investigator, I am interested in studying sarcoidosis, but there is such a lack of information and resources that it is not an ideal disease area on which to base my career. Almost every area – epidemiology, treatment, diagnosis, burden – has little to no data; it is a very barren field to enter. Overall, the sarcoid space is not conducive to a prolific and valuable career output.”
M.D., Investigator, Georgia
While sarcoidosis has been recognized for literally hundreds of years, the pace of research has been significantly slow, with very little breakthroughs or high-impact findings. Very few investigators focus on the disease as there is such a lack of resources, from data to funding. FSR invests in the FSR Sarcoidosis Patient Registry to help counter this lack of preliminary data that could serve to drive hypothesis and provide a landscape of the scope of the disease. This online, IRB approved registry serves as a highly secure repository of longitudinal data that builds resources for investigators so the space is more inviting to study.
Fostering Collaboration
“Our company is interested in studying various drugs for re-purposing to treat sarcoidosis – we see a possible connection around the disease needs and the potential of several drugs used for other diseases. We would be interested in conducting clinical trials to collect data around the application and effectiveness, but it is just too difficult to build enough medical institute partners that could facilitate our trials. And the time and effort it will take to build a network of trial sites is just too costly for our company to consider; thus, we tend to focus on disease areas that have already developed clinical trial partnerships.”
Biotech Company CEO, Maryland
THE FSR Clinical Studies Network provides an existing consortium of partner institutes who are already poised to conduct multi-site trials – companies can come directly to FSR to propose studies and trials for the current eight sites, and save resources in the process. In addition, the international group of worldrenowned institutes develop and facilitate their own studies and publish the findings, further infusing the disease research space with new critical data and information. FSR provides grants, totaling $250,000 per grant period, to fund the partnering CSN sites to enable networking and collaboration. CSN serves to significantly reduce the resources needed to test the efficacy of therapies, the measure of impact of re-purposing drugs, and to screen promising new compounds.
Advocacy Initiatives
Sign-On Letters
These letters are sent to legislators to educate and influence their decisions on matters related to sarcoidosis, rare disease and general health efforts.
Ad Hoc Group Letter Urging Swift Passage of FY22 Appropriations
Friends of the National Institute of Environmental Health Sciences (NIEHS)
Patient-Centered Outcomes Research Institute (PCORI) and Coalition of Skin Diseases (CSD) Letter
Patient Letter to Congress on Accelerated Approval
Patient Letter to HHS on Accelerated Approval
FDA Comment from Rare Disease Patient Organizations
FORWHFY23A Appropriations Letter
FY23 302(b) Allocations Group Letter
[Friends of NCHS] Sign-on Letter – FY23 Funding
PPC Welcome Letter for the 118 Congress
NIAMS Coalition – Appropriations Letter
Friends of the National Institute of Environmental Health Sciences (NIEHS)
Friends of the National Institute of Environmental Health Sciences (NIEHS) 2
FNIEHS_LHHSletter_FY2024 Senate
FNIEHS_LHHSletter_FY2024 House
The Ad Hoc Group Fiscal Year 2024 Recommendation
FORWHFY24AppropriationsLetter_3.15.23
Health Partners Letter Opposing REINS Act 3-17-23
PPC Welcome Letter for the 118 Congress – Final
FY 2024 Ad Hoc Group Recommendation_Final 3.13.23
S. 652/H.R. 2630, SAFE STEP ACT
HELP Copays Act_PBM Reform_ACCC 071423
SafeStepAct PBM reform legislation
NHLBI FY 25 Appropriations Letter
Letter to Cosponsor SOAR Act – House 5-31-24
Letter to Cosponsor SOAR Act – Senate 5-31-24
NCOA letter to CMS on LIS recommendations
PBM Reform Patient Policy Priorities
Coalition for Health Funding FY25 NDD letter
Ensuring Continued Timely Access to COVID-19 Vaccines
World Oxygen Day Coalition_SOAR Act
Building Patient-Centric Measures
“My doctor measures the effects of the drugs I am on by doing Pulmonary Function tests and 6-minute walk test. Sometimes the results show a big improvement, but I still feel horrible. I am fatigued, depressed, and am having sever pain and tingling throughout. I am so frustrated because she says the drugs we are using must be working – but why do I feel so terrible still?”
Patient, Colorado
Whether it is the direct patient’s response to a treatment in the clinic or within a clinical trial, it is often an ongoing struggle to fully assess the efficiency a therapy in treating sarcoidosis. The issue is that many physicians and investigators have varying degrees to measure what constitutes “improvement”, called endpoints, for a sarcoidosis patient. In part, the difficulty of having consensus on more patient-focused endpoint may be because the disease is multi-organ and most often manifests in different ways in different patients. FSR invests in initiatives that will help lead to more concrete, patient focused, and widely accepted endpoints that will help with the assessment of treatments and trial findings.
De-Risking Sarcoidosis Trials and Projects
“I am an investigator who is partnering with industry for a large sarcoidosis clinical trial. However, the risk of not having this trial successfully recruit the required number of patients is huge! If it goes under-recruited, the trial will be a loss not only in the money and time it costs to develop, but it will also shut down future trials to be considered if the belief is sarcoidosis trails are too difficult to get patients into. It is important to have solid recruitment for any trial, but the risks for a sarcoidosis trial endanger the entire field. There needs to be a sarcoidosis-specific recruitment specialists to help.”
M.D., Investigator, South Carolina
FSR invests in our recruitment initiative SARConnect ™ to ensure that any sarcoidosis and related trial and studies are fully recruited and have patient engagement support to ensure fulfillment of trial requirements. Through a variety of significant outreach efforts, FSR serves as a recruitment specialist connecting academia, industry, and physicians to patients by serving as the message, while at no time risking the patient’s privacy or contact information. As a patient-centric third-party, FSR connects stakeholders to their desired constituents by providing direct information and links for them to participate in the trial, study, or project. This is a massive resource-saving mechanism that not only de-risks trials through recruitment but also through education on the importance of patient engagement in research.
Educating and Empowering Patients
“I was recently diagnosed with sarcoidosis after an emergency room visit and really just panicked. I had no idea what it was and what the outcome might be and my general practitioner had very few answers. I was able to find the Foundation for Sarcoidosis Research’s website which provided me with many of the resources I needed, including a directory that helped me find the doctors I needed. I also found out about a FSR Patient conference that is going to be held near my city so I could learn firsthand from experts and meet other sarcoidosis patients. The most important part to me was that FSR is led by experts in the field – just getting various pieces of advice off the internet is scary and dangerous!”
Patient, Texas
In an era where information can be accessed literally anywhere, it is critical that patients and other stakeholders have a reliable, expert-led resource for accessing sarcoidosis information. A patientservice oriented program, FSR Patient Resource Education Program (PREP) provides a specialized service that educates, informs, and resources patients, physicians, and other stakeholders. These online, in print, and in-person services include patient conferences where patients hear directly from experts about the most updated information on sarcoidosis and have a chance to interact with them and other patients, as well as webinars and consistently updated materials. FSR Physician Directory and Support Group Finder also connect patients to the information most often requested. Downloadable brochures and treatment protocol resource are available not only patients, but clinicians and care providers as well. PREP provides answers and information to patients in nearly every conceivable area of sarcoidosis – and when we don’t have it, we find it for patients, reducing the risk for patients of poor or inadequate information about their disease.