Therapeutic Area

Rare Disease

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.

Learn more about Rare Disease Day and the work Medidata is doing to continue advancing rare disease trials below.

Medidata Supports Rare Disease Day

February 29, 2024

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 careers.

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


Rare Disease Studies


Unique sponsors with Rare Disease trials


Sites set up to conduct Rare Disease trials


Enrolled Rare Disease patients

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.