Clinical Trial Trends: Rare Oncology vs Non-Oncology

In rare disease research experience is sometimes treated as binary. Clinical research organizations (CROs) tick the metaphorical box by outlining their previous work in trials that have included a rare indication as a focus. However, the composition of a prospective CRO’s trial portfolio, and the balance between oncology and non-oncology indications, provides important insights into more than their experience, it profiles their true expertise. 

All rare indications experience small patient populations and the challenge of recruitment/retention, but once you start looking at the activities in executing a Rare Oncology trial compared to a Rare Non-oncology trial, there are some big differences. 

Oncology rare disease vs non-oncology rare disease in clinical trials 

Within Rare Oncology, there is tumor heterogeneity (more so than Non-rare Oncology), but non-oncology rare diseases tend to impact a wider range of organs and systems. That requires its own type of expertise. In addition, statistically, there are more procedures in oncology trials versus non-oncology trials and more datapoints collected. The protocols themselves are even longer.  

In this article, we are going to discuss trends in Rare Disease research, breaking out Oncology vs Non-oncology. We will look at the trials that are planned or ongoing around the world and point out trends in each category. We will also highlight Precision for Medicine’s rare experience in the same way. 

How many rare oncology trials are started each year? How many rare non-oncology trials are started each year? 

The number of planned and ongoing trials is over 92,000 right now. Clinical trials with a rare disease focus are much less common, Rare Non-oncology trials most of all. As you can see from the chart below, there are comparatively fewer Rare Non-oncology trials than Rare Oncology, around 4,700 vs 16,200 respectively. 

Rare oncology vs. rare non-oncology clinical trials by start date 

Citeline, Trialtrove, 1 August 2024 

However, despite there being so many fewer Rare Non-oncology trials than Rare Oncology ones, the number of patients is actually much closer at 1.45 million vs 1.92 million.

Oncology vs non-oncology rare disease research by phase 

Let’s start by looking at the rare disease trials that are currently in progress or planned. 

Planned and ongoing rare trials by phase, oncology and non-oncology

Citeline, Trialtrove, 1 August 2024    

 There are more active Phase 2 Rare Oncology trials than there are Phase 2 Rare Non-oncology trials, on a relative basis. At the same time, there are more Phase 3 Rare Non-oncology trials than Phase 3 Rare Oncology trials, again looking at the numbers relatively. 

Where are rare oncology vs rare non-oncology trial sites? 

Next, we examined the top 10 site countries to get a better idea where clinical trials are being conducted within these therapeutic areas. 

Top 10 planned and ongoing rare trial site countries, oncology and non-oncology 

Citeline, Trialtrove, 1 August 2024 

Many of the top countries are the same. Australia, China, France, Germany, Italy, Japan, Spain, the United Kingdom, and the United States appear in both lists, albeit in a different order. However, in Rare Oncology trials, South Korea is in the top 10 while in Rare Non-oncology trials, Canada takes a spot. (In Rare Oncology trials, Canada is in the 11th spot, while in Rare Non-oncology trials, South Korea is 15th) And, in all active clinical trials, rare or not, oncology or not, the top 3 countries are China, United States, and India. 

Precision for Medicine’s experience in rare oncology vs rare non-oncology 

Precision for Medicine is committed to supporting even the most challenging indications. We have a long history of working in rare disease research, both oncological and non-malignant indications. 

Rare oncology vs. rare non-oncology clinical trials by start date 

Citeline, Trialtrove, 1 August 2024 

Oncology vs non-oncology rare disease research by phase 

Precision for medicine has also supported rare disease trials in different phases. As seen below, our experience is centered around early phase research in in Rare Oncology trials while we have worked on more Phase 3 trials than Phase 1 trials in Rare Non-oncology indications. 

Precision for Medicine’s rare trial experience by phase, oncology and non-oncology

Citeline, Trialtrove, 1 August 2024          

Where are Precision’s Rare Oncology vs Rare Non-oncology trial sites? 

When we reviewed the planned and ongoing trials above, we spotlighted the top 10 site destinations for Rare Oncology vs Rare Non-oncology. Here, let’s look at Precision for Medicine’s experience in a similar fashion. 

Precision’s top 10 rare trial site countries, oncology and non-oncology 

Citeline, Trialtrove, 1 August 2024 

There is a lot of similarity between the two lists. The big difference is Belgium and Poland. However, were we to expand the table to include the top 10 site locations, Poland would be in the 11th spot for our Rare Oncology trials whereas Belgium would rank 11th in our Rare Non-oncology trials. 

Our work in rare oncology and rare non-oncology trials 

Precision for Medicine is proud to have supported some of the most groundbreaking rare disease trials. From rare hematological oncology to pediatric CNS, we have helped shepherd drugs to market, through multiple trials for the same drug, across different regulatory requirements and supporting the needs of patients to reduce the burden of participation for them as well as their caregivers. 

Precision’s experience in rare oncology and rare non-oncology trials 

Moving rare disease research forward 

Rare oncology indications are incredibly different from non-oncology rare diseases. Patient profiles, protocol requirements, data points—nothing is the same. Sponsors need to make sure their chosen CRO can handle what each indication requires. Simply having experience in any rare disease should not be enough of a qualifier to support such critical research. 

Precision for Medicine has extensive rare disease experience; over 56% of our trial experience has involved at least one rare indication.