Clinical Trial Landscape: Rare Pediatric

Rare diseases are a global issue. While each rare disease impacts fewer than 50 people in 100,000, over 9000 have been identified.¹ Taken in total, this means that 1 in 16 people are affected by a known rare disease. Of these, roughly 70-75% present in childhood, with 2 out 3 appearing before the child turns two. Some 30% will die before the age of 5.²

In this overview, we explore the clinical trials currently underway for rare pediatric disorders, examining their distribution by phase, status, country, and patient populations.

Exploring the Rare Pediatric Clinical Trial Landscape        

Despite the advent of new therapies, rare pediatrics continues to present significant challenges, necessitating detailed clinical studies to forge effective treatments. 

Rare Pediatric Clinical Trials by Status  

Analysis of Citeline data reveals that rare pediatric conditions are the subject of 1,593 active clinical trials, from early to late stages, reflecting a broad spectrum of research activity.³

Rare Pediatric Trials by Status

Citeline Trialtrove® – 28MAY202416

Rare Pediatric Trials by Start Date

Yearly analysis of rare pediatric trial start dates indicates growing research momentum, with an increase in trial starts each year.

Planned & Ongoing Rare Pediatric Trials by Start Date

Citeline Trialtrove® – 28MAY2024

Rare Pediatric Trials by Country  

Clinical trials for rare pediatrics extend across 105 countries, with a high concentration of studies in China (516) and the United States (502), illustrating the truly global nature of this research.

Planned & Ongoing Rare Pediatric Trials by Site Country

Citeline Trialtrove® – 28MAY2024

Rare Pediatric Trials by Indication

The scope of patient care in rare pediatric trials is wide, encompassing a wide range of therapeutic areas. Blood cancers top the list.

Top Planned & Ongoing Rare Pediatric Trials by Indication

Citeline Trialtrove® – 28MAY2024

Rare Pediatric Trials by Phase     

Early-phase clinical trials dominate the rare pediatric landscape, with 240 Phase 1, 194 Phase 1-2, and 518 Phase 2 trials, aimed at establishing a solid foundation for future efficacy studies.

Planned & Ongoing Rare Pediatric Trials by Phase

Citeline Trialtrove® – 28MAY2024

Precision's Expertise in Rare Pediatric Research          

Our track record in rare pediatrics includes 31 clinical trials, reflecting our deep scientific expertise and strategic research focus.

Rare Pediatric Trial Starts  

Since 2019, Precision for Medicine has overseen the start of 13 new clinical trials in rare pediatric research.

Precision for Medicine's Rare Pediatric Trials by Start Date

Citeline Trialtrove® – 28MAY2024

Precision's Reach in Rare Pediatric Research 

Precision for Medicine’s rare pediatric trials reach across 51 countries, engaging a wide range of healthcare systems.

Precision for Medicine's Rare Pediatric Trials by Site Country

Citeline Trialtrove® – 28MAY2024

Rare Pediatric Trials by Phase     

Our experience in rare pediatric research includes a range of trial phases, including 14 early phase trials and 17 late phase studies.

Precision for Medicine's Rare Pediatric Trials by Phase

Citeline Trialtrove® – 28MAY2024

Precision's Experience by Indication     

Precision for Medicine’s trials in rare pediatrics cover various indications, including Pulmonary Hypertension and Rett Syndrome most notably.

Precision for Medicine's Rare Pediatric Trials by Indication

Citeline Trialtrove® – 28MAY2024

Conducting Rare Pediatric Trials with Precision

Rare pediatric diseases span every therapeutic area and there are currently trials taking place in almost half of all the countries in the world. In this article, we discussed the clinical trial landscape around this space and detailed some of our recent work, highlighting phases, site locations, and indications.

Moving forward, Precision for Medicine is dedicated to playing a critical role in this research and the hope it brings. Visit our rare disease CRO services page to learn more about how we are working to create a healthier world.

References

1. Pollard S, Weymann D, Dunne J, et al. Toward the diagnosis of rare childhood genetic diseases: what do parents value most? [published correction appears in Eur J Hum Genet. 2021 Oct;29(10):1589. doi: 10.1038/s41431-021-00925-7]. Eur J Hum Genet. 2021;29(10):1491-1501. doi:10.1038/s41431-021-00882-1.
2. Muntañola, A.C. (2021). Rare Diseases in the Pediatric Population. In: Huml, R.A. (eds) Rare Disease Drug Development. Springer, Cham. https://doi.org/10.1007/978-3-030-78605-2_15.
   
3. Citeline Trialtrove.