
Medidata Supports Rare Disease Day
Advancing Rare Disease trials, one patient at a time.
Rare Disease Day:
February 28
Medidata is proud to join the official Rare Disease Day initiative raising awareness and generating change for the 300 million people worldwide living with a rare disease, their families and carers.
Listening to Rare Disease Patients and Caregivers
A wish list for effective, expedient clinical trials.
Medidata has the most extensive Rare Disease trial experience
Based on U.S. Rare Disease Definition
2,040+
Rare Disease studies
355+
unique sponsors with
Rare Disease trials
103K+
sites set up to conduct
Rare Disease trials
322K+
enrolled Rare Disease
patients
Affecting 300+ 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.

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
Elevate Awareness for Rare Disease
Scientific breakthroughs in Rare Disease therapy with Car T-cell therapy and CRISPR hold promise for the future.

Opportunities to conduct Rare Disease clinical trials are scarce. How to “get it right” from the start?
