I recently had the privilege to serve as a Co-Chair of this year’s Access USA Rare Disease Summit, which means I had the privilege of hearing so many engaging and rich discussions, led by passionate leaders in the rare disease industry. One of the most powerful aspects of the experience was what emerged when conversations began to echo with one another… in a positive way. 

Across conversations spanning development, access, commercialization, and patient experience, a shared set of questions helped anchor discussions in real-world realities: 

• Are we designing how care is delivered?  

• Are patient and caregiver voices shaping decisions early enough?  

• Are we clear about what evidence can and cannot tell us today?  

Several themes stood out, not as theoretical ideas, but as practical signals for where rare disease strategy must continue to evolve. 

Clinical Trial Design Shapes Access Long After Approval 

Clinical trial design is an access decision. Early choices, endpoints, eligibility criteria, and diagnostics often become the basis for payer coverage long after approval. From my role at PANTHERx, I see how those upstream decisions show up downstream for patients, shaping who can start therapy and how long it takes to get there. That’s why access cannot be treated as a post-approval problem to solve later. Designing trials with real-world use in mind is essential to ensuring evidence supports not just approval, but meaningful, timely access for patients. 

One Size Doesn’t Fit All in Rare Disease 

Throughout the Summit, one phrase kept resurfacing: “When you’ve seen one patient, you’ve seen one patient.” It captured a shared reality across discussions, that even among patients with the same diagnosis, experiences can differ dramatically. 

Speakers and patient advocates reinforced that rare disease burden extends far beyond what patient data captures. Emotional realities like isolation, frustration, and loss of independence often shape whether patients can stay engaged in care, even when the therapy itself is effective.  

In my role at PANTHERx, we see how these non-clinical factors directly influence adherence and trust. That’s why hyper-personalized support is not a ‘nice-to-have,’ it’s foundational. Designing care around the whole person, not just the diagnosis or the trial protocol, is what makes rare disease care work in the real world. 

Central Role of the Patient Voice 

A clear takeaway from the Summit was that patient advocacy and patient voice are not nice to have; they are central to how rare disease care works. Advocacy partners shape trial design, support patients emotionally, build community, and translate lived experience for regulators, payers, and providers. 

As a pharmacist by training, I’ve always seen care as something that happens in the community, not in isolation. Medications don’t succeed on their own; people do, supported by advocates, caregivers, and care teams who understand the realities patients face every day. When advocacy and patient voice are embedded early and authentically, they strengthen evidence, improve program design, and create care models patients can trust and sustain over time. 

Looking Ahead 

The questions that framed the Summit still matter most: are we designing real-world care, listening early enough to patients, and being honest about what evidence can—and can’t—tell us? In practice, progress only holds when those questions are answered in ways that reflect how patients live with and access care. 

A heartfelt thank you to Informa and Access USA for their work to create an engaging conference. I’m deeply thankful to the speakers, advocates, and everyone who participated so openly. Your willingness to share experience, challenge assumptions, and listen to one another is what makes this community special, and what turns good conversations into better outcomes for patients and families.