Clinical Trial Trends: Early Phase Rare Oncology 

Early Phase Oncology trials are where the hard questions live, but for rare oncology, those questions get even harder.

The stakes are high, the populations are small, and the answers often live in the margins—the difference between a genomic variant that’s actionable and one that isn’t, or the subtle ways a drug’s mechanism of action can impact a niche subset of patients. It’s not just about dose escalation; it’s about strategy, creativity, and adaptability. 

Rare oncology trials often challenge us to rethink everything we know about trial design. Consider this: how do you dose a drug when there’s no existing data to anchor its toxicity in your specific population? How do you recruit globally while accounting for the fact that the disease itself may present differently in patients depending on their geography, environment, or even access to care? And how do you measure efficacy when traditional endpoints, like progression-free survival, aren’t feasible due to small sample sizes? 

These aren’t abstract questions—they’re the day-to-day realities of rare oncology research. Successful trials require adaptive designs that can pivot as we learn more about the disease, Bayesian modeling to manage the uncertainty of small datasets, and biomarker-driven strategies to ensure the right patients are enrolled. And let’s not forget the operational hurdles: multi-regional trials, rare populations, and the ever-present need to work in partnership with advocacy groups to ensure enrollment is ethical, feasible, and equitable. 

The reality is that early phase rare oncology research isn’t just about answering questions—it’s about asking better ones. And those questions drive the innovation needed to deliver meaningful results for some of the most underserved patient populations in the world.  

Understanding the Early Phase Rare Oncology Clinical Trial Landscape   

To get a better idea of what Early Phase Rare Oncology research looks like, we will use Citeline, a clinical trial intelligence platform, to outline what the Phase 1 and Phase 1-2 rare oncology clinical trial landscape looks like today. 

Early Phase Rare Oncology Trials by Status   

To date, over 1.05 million people have participated in an Early Phase Rare Oncology clinical trial, and there have been over 20,956 trials. Around 5,500 of them are active. Here is a breakdown by status.  

Early Phase Rare Oncology Trials by Status  

Citeline Trialtrove® – 22NOV2024 

Early Phase Rare Oncology Trials by Country   

Looking at planned and ongoing Early Phase Rare Oncology trials by site location shows where there is special interest in this therapeutic area. Here are the top 10 site locations for Phase 1 and Phase 1-2 Oncology research around the world.   

Top 10 Countries for Planned & Ongoing Early Phase Rare Oncology Clinical Trials 

Citeline Trialtrove® – 22NOV2024  

Early Phase Rare Oncology Clinical Trials by Phase  

Looking at Early Phase Rare Oncology trials, the ratio of Phase 1 trials to Phase 1-2 trials is 2:1.  

Early Phase Rare Oncology Trials by Phase  

Citeline Trialtrove® – 22NOV2024 

Early Phase Rare Oncology Trials by Start Date  

Let’s also look at Early Phase Rare Oncology trials by their start date. This gives us an idea of the pipeline for new drug development in rare indications as well as the competitive landscape and industry timing.   

Early Phase Rare Oncology Trials by Start Date, 2014-2024  

Citeline Trialtrove® – 22NOV2024  

Precision's Expertise in Early Phase Rare Oncology Research   

At Precision for Medicine, we have worked on more than 159 Early Phase Rare Oncology trials. We are committed to supporting our Sponsors through every phase of drug development, starting at First-In-Human.   

Trials by Phase   

Our experience follows the industry trend exactly. While we have worked on nearly 70 Phase 1 Oncology trials, we have worked around 33% more Phase 1-2 Oncology Trials.  

Precision for Medicine's Early Phase Rare Oncology Trials by Phase  

Citeline Trialtrove® – 22NOV2024  

Precision's Reach in Early Phase Rare Oncology Research   

Precision is active in all the places Early Phase Rare Oncology research is happening. Our Phase 1 and Phase 1-2 oncology studies truly span the globe.  

Precision's Early Phase Rare Oncology Trials by Site Country  

Citeline Trialtrove® – 22NOV2024  

Precision's Focus in Early Phase Rare Oncology Research   

Precision for Medicine’s Early Phase Rare Oncology experience is roughly split between solid tumors and hematologic malignancies. 

Precision's Early Phase Rare Oncology Trials by Tumor Type  

Citeline Trialtrove® – 22NOV2024  

We have also had the opportunity to participate in a large number of First-in-Human (FIH) trials. 

Precision's Early Phase Rare Oncology Trials by First-in-Human  

Citeline Trialtrove® – 22NOV2024  

Precision has also supported many Immuno-Oncology (IO) trials. 

Precision for Medicine's Early Phase Rare Oncology Trials by Immuno-Oncology Status  

Citeline Trialtrove® – 22NOV2024  

Precision makes a difference in rare oncology clinical development

Precision for Medicine has extensive experience in early phase rare oncology trials, supporting studies for some of the world’s most complex and underserved cancer populations. Our team understands the unique regulatory, operational, and scientific challenges associated with rare cancers. From navigating global patient recruitment to optimizing trial design for small, heterogeneous populations, we bring a tailored approach to every program. 

Our expertise extends to biomarker identification, precision diagnostics, and the evaluation of novel drug targets, ensuring your trial is equipped to generate meaningful data. With a global footprint and collaborative network, we are uniquely positioned to identify and engage the right patients, no matter where they are. 

Partner with Precision on your next early phase rare oncology program and see how our experience, innovation, and insight help transform groundbreaking science into tangible outcomes.