The View From The Summit

"Scleroderma has no borders, thus this audience of patients, investigators, regulatory and biopharmaceutical professionals should not have any in collaborating for a cure to end this deadly disease."

This call to action, uttered by Monica R. during her opening address, reverberated across the congregants of the Pooks Hill Marriott ballroom. Her measured tone — marred by over a decade living with Scleroderma — clearly reflected the importance of collaboration and intense focus during this first-of-its-kind Patient Advocacy Summit, hosted by the National Scleroderma Foundation.

As a pharmacist, consultant, and someone living with systemic sclerosis with interstitial lung disease, Monica's words resonated with the intention I harbored long before arriving. A commitment informed by the weight of hundreds of patient stories I’ve heard through years of advocacy and community connection. 

During this summit, my aim was to elevate the unique life context and values of those patients into actionable insights — connecting with stakeholders during elevator commutes, dinner conversations, and the closing workshop — eventually to share what I learned with my community.

Friday through SundayThe summit opened Friday evening with a welcome reception before launching into a full day of programming on Saturday. The day moved from personal anecdote to scientific evidence, then advanced through a research review and the state of the disease and arrived at the clinical frontier with sessions on drug development, clinical trial landscape, and a panel discussion on the challenges and opportunities surrounding meaningful endpoints.

Sunday extended that arc into NIH investment, the regulatory landscape for rare disease, and an interactive workshop aimed at building alignment around research and development priorities. A fitting frame for a summit that positioned patients not as recipients of information, but as partners in shaping it.

Patient-experience friction was welcome

For patients with scleroderma, even something as routine as a clinical assessment can carry layers of uncertainty. And you learn to anticipate the variability between how one visit can feel different from the next. How something is measured, how it's asked, even how it's interpreted.

That reality came into sharper focus when the conversation turned to skin assessment.

The modified Rodnan Skin Score — seventeen areas, assessed in succession — remains the clinical standard for measuring skin thickness in systemic sclerosis. In experienced hands, it can be reliable. But experienced doesn't always mean identical. The pressure applied, the temperature of the examiner's hands, even the phrasing of a question. All subtle differences that don't just influence the score but shape the patient's experience of being measured.

So, when Dr. Andreea Bujor referenced spatial frequency domain imaging as a more objective alternative during her State of Scleroderma remarks, it didn't feel like a distant innovation. It felt like an acknowledgment of what I and other patients experience during these assessments, even if it hasn't always been captured.

That quality of exchange carried through the weekend. 

With a fellow patient describing how her contracted and digital ulcer laden hands couldn't operate a newly advanced auto-injector that, by design, should have simplified her treatment. Instead required her husband to learn to administer it so she could continue to manage her condition. 

Industry partners received this testimony openly — noting that pipeline products using similar delivery mechanisms may benefit from deeper consideration of how devices perform across the full spectrum of the scleroderma population. 

It was a reminder that patient insight, when welcomed into the room, can redirect innovation in meaningful ways no trial protocol anticipated. Especially when stakeholders choose to sit with the frictions rather than around them. 

Beyond Pooks Hill

The FDA continued where summit conversations ended — announcing, on Tuesday, an initiative to advance the implementation of real-time clinical trial safety signals and endpoints, as part of a push to accelerate promising therapies and build toward continuous trials across all phases of drug development. 

A topic the group at Pooks Hill spent the weekend engaging with.

The work, however, doesn't wait for federal announcements. It happens at the municipal level, in city halls and council chambers, where patients and advocates can move the needle without a Washington address. Scleroderma Awareness Month proclamations requests have already been secured in cities across the country — these and other small acts of recognition can accumulate into visibility, and visibility into momentum.

For patients, remember our story is evidence. Whether you are speaking at a summit, serving on an advisory board, or simply talking to your doctor, your presence provides the critical intel that healthcare's technical challenges require to be solved.

Grateful for organizations like the National Scleroderma Foundation that exist to amplify that presence and provide the medium for us to take up space at the table. 

The waiting room is where we've been. The summit is where we're going.