Rare Cancer Day 2026

By Dr. Fernando Lasso de la Vega, Executive Director, Clinical Operations

On September 23, the NORD Rare Cancer Coalition marks Rare Cancer Day, an opportunity to bring visibility to diseases that can often go unseen. This year, the conversation centers on timely access to innovative diagnostics and comprehensive care, greater community visibility, equity in treatment access, research funding, and support for patients and caregivers.

These challenges also shape many of the studies we work on at Stiris.

In more than two decades in clinical research, I’ve had the opportunity to see oncology from both a medical and operational perspective. One lesson has remained consistent: rare cancers don’t just need more research. They need demonstrated execution and the track record to prove it. 

No Oncology Trial Is “Standard” When the Cancer Is Rare

Every therapeutic area has its complexities, but the challenges of oncology studies become even more pronounced when the cancer in question is rare.

Visit schedules are often organized around treatment cycles, while standards of care can vary between countries and even between regions. Whether a patient receives treatment as scheduled may also depend on their health status at that moment. Delays, then, are part of the reality of the disease, not simply the logistics of the trial.

Many studies also depend on companion diagnostics, where the presence of a specific biomarker determines eligibility before a patient can enroll. To manage this complexity, sponsors are increasingly turning to adaptive designs that allow for pre-planned modifications as new data emerge.

None of this is incidental complexity. It’s part of the science itself. And it helps explain why so many of the challenges patients face remain so persistent.

Late Diagnosis and the “Oncology Lottery”

Families navigating a rare cancer diagnosis often describe similar experiences: nonspecific symptoms that go unnoticed, a lack of screening programs capable of detecting the disease early, and low clinical suspicion until the cancer has already advanced.

By the time a diagnosis arrives, the window for some of the most effective interventions may already be closing.

Even after diagnosis, access remains uneven. Tissue available for biopsy can be limited, and access to genomic testing varies considerably between regions and health systems. A targeted therapy may exist and still be out of reach simply because of where a patient lives.

The financial impact can also be significant. Patients with the same disease may face very different costs for the same medication depending on their coverage and geography. It’s a kind of “oncology lottery” that can place enormous pressure on patients and their families.

Clinical trials should help close that gap, but doing so in rare cancers is particularly difficult.

Even experienced multidisciplinary teams face the challenge of applying evidence-based protocols to diseases that, by definition, have been understudied and can be highly heterogeneous.

Behind each of these gaps is a patient and a caregiver carrying a very real burden: isolation, confusion, financial pressure, and often the sense that few people truly understand what they’re going through.

We’ve seen a similar challenge in glioblastoma: research doesn’t necessarily lack innovation. What it needs are clinical trials capable of proving that innovation works.

I believe the same principle applies to rare cancers more broadly.

What Sponsors Look for in an Oncology CRO

Over the years, in conversations with sponsors, sites, and study teams, I’ve seen a consistent pattern in what sponsors expect from an oncology research partner.

They look for real therapeutic experience across solid tumors and hematology; familiarity with tumor assessment endpoints; experience with immunotherapy, dose escalation, and dose expansion; and a clear understanding of the safety profiles associated with new drug classes.

But they also need something more operational: strong, stable teams that can respond quickly.

In my experience, that matters especially in oncology. Safety oversight is critical when working with new molecular entities and first-in-class treatments. Having professionals who understand the information from a clinical, not just an administrative, perspective can make a meaningful difference in how quickly and accurately safety issues are identified and reported.

Site relationships matter just as much.

Strong relationships across the site are equally important. Principal investigators and key opinion leaders provide critical scientific and strategic insight, while coordinators, nurses, and clinical research staff are essential to the day-to-day work of identifying patients, coordinating visits, and ensuring assessments happen when they should.

Oncology centers also rarely function as a single department. A study may involve imaging, pathology, laboratory services, pharmacy, radiation, and surgery, alongside scientific reviews, resource assessments, ethics review, and budget negotiations. Understanding that institutional structure, and working with it rather than against it, is essential to keeping study start-up on schedule.

That track record, not just therapeutic knowledge but demonstrated execution, is what sponsors are ultimately vetting for.

How Stiris Research Advances Rare Cancer Clinical Trials

At Stiris, our oncology portfolio has been deliberately shaped around areas of high unmet need and low prevalence. These are studies where recruitment can be genuinely difficult and where operational decisions have real consequences for patients, caregivers, and sites.

We’ve seen what becomes possible when stakeholders align early and study teams maintain clear visibility into progress.

In a multicenter pancreatic cancer trial requiring coordination across oncology, surgery, pharmacy, and IRB, consistent communication with site coordinators helped complete recruitment ahead of schedule and kept the study on track despite personnel changes along the way.

Our experience in complex oncology programs has also shown us the importance of exploring new approaches for diseases that have seen far too little progress for far too long.

Our oncology experience also includes stepping in when studies need to be rescued. In a Phase II castration-resistant prostate cancer (CRPC) trial, the project exceeded the sponsor’s recruitment target following our intervention.

None of this removes the fundamental difficulty of researching rare cancers. It doesn’t change the biology of the disease or increase the number of available patients. What it can do is help ensure that when a promising therapy exists, the trial designed to prove it works doesn’t become the obstacle standing in its way.

Beyond Recruitment: Supporting Patients and Caregivers Through Complex Oncology Trials

Recruitment is only part of the challenge. For patients and caregivers navigating a rare cancer trial, complex schedules, unfamiliar processes, and ongoing participation demands can add another layer of burden to an already difficult experience.

Stiris developed the Patient Caregiver Liaison (PCL) Program to help address that gap. The program provides patients, families, and caregivers with a dedicated, non-clinical point of contact throughout the clinical trial journey. The PCL helps identify participation barriers early, supports consistent communication, and strengthens collaboration between patients, study sites, and sponsors.

The idea behind it is simple: successful trial execution isn’t only about managing the protocol. It’s also about understanding and supporting the people being asked to participate in it.

Rare Cancer Day: From Visibility to Action

Rare Cancer Day brings together a community that too often faces these diseases in isolation, while calling for more timely diagnosis, greater equity in treatment access, and stronger investment in research.

For those of us working in clinical research, it’s also a reminder of our responsibility.

Behind every protocol we design, every relationship we build with a site, and every question we help resolve, there is a patient and a family waiting.

That’s the work. And it’s work that deserves our long-term commitment.