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.
Rare Disease Clinical Development:
Novel Approaches to Overcoming Operational Challenges
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 fewer 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
Medidata has the most extensive Rare Disease trial experience
Based on U.S. Rare Disease Definition
Rare Disease studies
unique sponsors with Rare Disease trials
sites set up to conduct Rare Disease trials
enrolled Rare Disease patients
Opportunities to conduct Rare Disease clinical trials are scarce.
How to “get it right” from the start?
"We're using omics to double rare disease patients' chances 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.
Insights from the Medidata Institute
Dr. Miganush Stepanians
President and CEO, PROMETRIKA