By Faith Eriks, Senior Associate, Medical Affairs Operations
In putting this blog together, we wanted to explore the importance of patient advocacy groups in the clinical trial process, what participants need from trial sponsors and clinical teams, and how advocacy partnerships help make sarcoidosis research more patient centered. We spoke with representatives from the Foundation for Sarcoidosis Research (FSR), the Sarcoidosis Research Institute (SRI), Sarcoidosis UK, Caring Hands, Sarkoidose Selbsthilfe OWL, and Xentria in order to get perspectives from those on both sides of clinical trial partnerships.
These conversations spanned the US, UK, and EU. We asked everyone we interviewed about the biggest challenges facing people living with sarcoidosis today. Despite differences in healthcare systems, funding models, and languages, many of the same themes surfaced.
Willing Participants but Limited Awareness
"The message the patient base needs to hear is that people are actually out there researching their disease." – Graham Bloye, Sarcoidosis UK
In 2022, FSR conducted a national (Institutional Review Board) IRB-approved survey of Black Americans living with sarcoidosis as part of its Ignore No More: ACTe Now! Campaign. The survey drew 406 respondents, and the most common reason respondents gave for not participating in research was that they were never given the opportunity. A striking 61% reported never having been invited to join a clinical trial. Among the 39% who had been invited, only 6% declined. Despite the risks involved, 85% of respondents believed clinical trial participation was an opportunity worth taking.
Across our conversations, advocates described a similar solution: meeting the patients where they are. That concept, though broad on its own, resonated throughout each interview regardless of time zone. Reaching potential participants in an honest, direct way is essential to raising awareness and supporting recruitment, but what that looks like can vary by community.
In the US, Cheryl Bradford, a patient advocate pointed to church bulletins, community health days, local newspapers, and doctors’ offices. She first recognized the potential of the approach after seeing a breast cancer trial flyer posted in a supermarket. In Germany, Katja Schillhorn described sharing trial announcements across multiple channels at once – social media, website, mobile app, and local self-help groups – so word travels quickly. Sarcoidosis UK emphasized something different: providing enough advance notice for advocacy organizations to plan a newsletter and social campaign properly.
In the sarcoidosis community, we are fortunate to be able to utilize these trusted collaborative advocacy networks to increase awareness about clinical trials rather than starting from scratch.
Barriers Within Reach
Learning about a clinical trial is only the first step. What happens after that is where advocates described some remarkably practical barriers.
Many people living with sarcoidosis are of working age and hold full-time jobs, and clinical trial visits can take up a significant portion of the day. Others are on disability or work in jobs without flexibility, where taking a day off can mean a day unpaid. Transportation can create another burden, particularly for participants who live hours from a trial site or center of excellence. At Sarcoidosis UK, the most common barrier reported is simply geography, because when a trial is within reach, interested patients generally find a way to participate. Advocates emphasized the importance of acknowledging these burdens and offering reimbursement opportunities should an individual be eligible to participate.
Some clinical trial sites have started designing around these realities. Tom Matthews of Xentria pointed to Johns Hopkins, where research facilities open early in the morning so participants can come in before work, and in some cases, stay open after standard hours. Hospitals run on business hours; patients’ lives do not. Scheduling is one of the few burdens a site can lift without changing anything clinically.
The issue of time also points back to protocol design. Study teams must balance the data they need against the amount of time participants can realistically give. Advocates described participants withdrawing from trials, including open-label extensions, when the visit burden became unmanageable. Assessment types, visit frequency, and visit length are established during protocol design, long before anyone is approached about enrollment. Those decisions can ultimately determine who can realistically take part.
Then there is the fear of losing a job entirely, a barrier FSR decided to work on directly. Through the coalition that grew out of Ignore No More, FSR petitioned the US Department of Labor to clarify how the Family and Medical Leave Act (FMLA) applies to research participation. The resulting clarification addressed how FMLA protections can apply to eligible employees participating in clinical trials, as well as eligible family members serving as caregivers. According to FSR, the clarification removes a barrier for an estimated 60 million Americans.
Trial Education and Treatment Alteration
One of the most common hesitations we heard involves the clinical trial investigational treatment itself. Patients who have spent years arriving at a medication regimen that controls their symptoms may understandably be reluctant to disrupt it, and in a fully blinded trial, participants may not know whether they are receiving active drug or placebo. Advocates were candid that the possibility of spending weeks without “active treatment” may be difficult to accept when considering trial participation.
What advocates asked for was thoughtful study design rather than simple reassurance. Depending on the study design, randomization ratios can be weighted so that more participants receive active drug compared to placebo in earlier phase trials. Protocols can define in advance what is permitted if symptoms worsen during the trial, including adjustments to existing medications so symptoms continue to be managed by the participant's own treating physician throughout the process.
Community physicians can also serve as local monitoring touchpoints. They can manage changes in a participant’s sarcoidosis between scheduled trial visits, so care does not depend entirely on travel to that site.
The language around this deserves scrutiny, too. Protocols often describe a participant whose symptoms worsen beyond a certain threshold as a "treatment failure," which misguidedly places a failure label on the participant. As Noopur Singh of Xentria put it, “Participants don’t fail trials. Protocols can be written with parameters that allow someone to remain involved in the trial while their symptoms continue to be treated and managed by their own physician – disease management is the priority, not the trial itself.”
Study coordinators are the first line of education and key point of contact for someone considering entering a clinical trial. It is important that trial sponsors provide thorough protocol education and accurate study messaging to help steer the conversations between the doctor, the participant, and the study coordinator.
Those Living with Sarcoidosis Want Information, Not Promotion
Advocates were remarkably consistent about the desired tone a clinical trial sponsor should take when communicating about clinical research. Information about research should be neutral, basic, and honest because that is what actually reaches patients. Anything that feels promotional or transactional can make it harder to receive candid feedback from the community.
Educational materials also have to account for different audiences. Those materials need to ensure a valuable conversation can take place with the participant and their doctor regardless of where they are on their journey.
Paula Polite of the Sarcoidosis Research Institute suggested another way to make clinical research more approachable to all audiences. Alongside clear, gentle explanations and honest information about risk, sponsors could anonymously profile the experiences of past trial participants – positive and negative alike. Firsthand accounts from past participants could help make the unfamiliar process feel more concrete.
Advocacy organizations hold themselves to a similar standard. Sarcoidosis UK shares information about industry-sponsored trials with its patient community on a deliberately agnostic basis and discusses available research and treatment options without recommending a particular product, leaving treatment decisions to patients and their physicians. For the organization, transparency and neutrality are the conditions of the relationship rather than a courtesy extended to it.
Continue Engagement When the Trial Ends
At the conclusion of a clinical trial, participants may have questions about what happens next. Continued communication can help reduce some of that uncertainty.
Participants also deserve to understand what happened in the trial they helped make possible. That means developing clear materials about next steps and providing updates as the research process continues. The Sarcoidosis Research Institute suggested sharing that information through the same advocacy networks that spread awareness in the first place. Those include but are not limited to advocacy group meetings and websites, patient conferences, and printed information shared at sarcoidosis centers of excellence.
From the advocates’ perspective, continued access to an investigational therapy should be treated as part of the sponsor’s responsibility to participants rather than an afterthought. The possibility of continued access can also influence the decision to enroll in the first place. When feasible, open-label extension trials or expanded access programs give participants who benefited from an investigational therapy a clearer path forward once their participation ends.
Why Patients Say Yes
Those eligible for a clinical trial weigh the potential opportunities and risks and ultimately make the choice that’s right for them. For many, the decision extends beyond their own circumstances. As Cheryl Bradford described it, many patients in this community tend to think past themselves. If a treatment helps them, it might help someone else living with the disease.
That same sense of shared purpose is reflected in what advocates ask of sponsors, which is accessible communication that can be disseminated clearly and honestly. Advocates described a rare disease landscape in which sarcoidosis organizations routinely work together, sharing what they learn to address hurdles that people living with sarcoidosis experience across the world.
"I came into the rare disease space, and it is so unique and such a fabulous community because all the rare disease organizations, we all work together collectively understanding the power of collaboration, and we're all struggling with many of the same things." – Mary McGowan, CEO of FSR
For individuals weighing participation, that collective mindset can matter more than it might seem. Participating in a clinical trial can be one way to contribute to the wider sarcoidosis community by helping advance research that may inform future understanding and treatment of the disease.
Building More Patient-Centered Trials
Many of the same principles apply to the relationship between sponsors and advocacy organizations. The traits advocates name – trust and transparency – come from a relationship that isn't purely transactional.
Engaging advocacy organizations early gives them an opportunity to identify meaningful opportunities for engagement and consider how disease burden may affect participation. They can also offer insight into practice patterns that could influence recruitment and where the community gets its information.
That reflects a broader shift toward involving patients and advocacy organizations earlier in research. Historically, research could progress substantially before patients had an opportunity to influence how studies were designed or conducted. Increasingly, the lived experience of patients is being considered earlier in the process.
Some of that shift is already visible. Over the past six years, sarcoidosis has seen more industry-sponsored trials than ever before, and patients have more opportunities to engage with and learn about research – the result of organizations giving them a way to contribute to research rather than only to awareness.
Study sponsors should prioritize making research more accessible and ensure their study participants have what they need to feel empowered in a clinical trial setting.
As industry and advocacy partnerships continue to evolve, McGowan sees the role of advocacy extending across the entire research lifecycle:
“I hope advocacy continues to serve as a bridge between the lived experience of sarcoidosis and the decisions that shape research…The impact that can be seen across the spectrum from designing studies to recruitment to interpretation of the results and sharing the impact,” she said. “It doesn't just stop right after the therapies are created in today's world. We have to ensure that the patients have access to these wonderful new therapies that are being developed from the research.”
Acknowledgements
We are grateful to everyone who gave their time and perspective to these conversations:
- Mary McGowan, Foundation for Sarcoidosis Research
- Paula Polite, The Sarcoidosis Research Institute
- Graham Bloye, Sarcoidosis UK
- Mary Stratton, Sarcoidosis UK
- Cheryl Bradford, patient advocate
- Katja Schillhorn, Sarkoidose Selbsthilfe OWL
- Allison Walker, Caring Hands
- Noopur Singh, Xentria
- Tom Matthews, Xentria
- Lesley Baxter, Xentria
Their insight shaped every section of this piece.
About the Author
Faith Eriks is Senior Associate, Medical Affairs Operations at Xentria, where she leads operational infrastructure and process optimization for Medical Affairs, and supports clinical and strategic communications efforts for XTMAB-16.