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Rare Disease

Rare Disease

Advancing Rare Disease trials, one patient at a time.

Affecting 400+ million people worldwide, over 7,000 rare diseases have been identified in the U.S. alone. Only 500 therapies have been approved.

From identifying and retaining patients, discovering biomarkers, accelerating diagnosis, and delivering insights into complex patient populations, Medidata is advancing Rare Disease trials by expediting outcomes and improving experiences.

Powered by the Medidata Rave Clinical Cloud™, experts, researchers, and clinicians are globally connected, using technology that delivers seamless end-to-end clinical operations, data integration, quality control, and analysis.

Blog Post

Understand the multiple challenges of Rare Disease trials across:

The complexity of patient enrollment and retention
The impact of limited data
The definition of clinical targets
The clinical trial execution

Affects less than 200,000 individuals in the U.S.

50% begin in childhood

~7.6 years for correct Rare Disease diagnosis

5x average annual cost per patient, Rare Disease vs. non-Rare Disease

73 new therapies in the last 3 years

1/3 R&D pipeline by 2024

Medidata has the most extensive Rare Disease trial experience

Based on U.S. Rare Disease Definition

1,240+
Rare Disease studies

170+
unique sponsors with Rare Disease trials

65K+
sites set up to conduct Rare Disease trials

190K+
enrolled Rare Disease patients

Infographic

Opportunities to conduct Rare Disease clinical trials are scarce.

How to “get it right” from the start?

Case Study

"We're using omics to double rare disease patients' chance to respond to therapy."

Learn how the Castleman Disease Collaborative Network (CDCN) designed the first study to accelerate diagnosis and leveraged Medidata’s machine learning technology capabilities to identify differentiated patient responses.