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.
Medidata Supports Rare Disease Day
February 28, 2022
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.
Video
Rare Disease Experts on Patient Engagement in Clinical Development
Learn from the experts on engaging patients before, during, and after the trial.
White Paper
Trends and Challenges in Rare Disease Clinical Development
Understand how to address and solve key common themes in rare disease trials including accessing, collecting, and analyzing data
Video
Commercial Data Solutions for Rare Disease
Clinical trials are all about proving the science works. Commercialization is about delivering on the promise of that science to the rest of the world. And that’s where Medidata Acorn AI Commercial Data Solutions joins the fight against rare disease.
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 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
1/3 R&D pipeline by 2024
Medidata has the most extensive Rare Disease trial experience
Based on U.S. Rare Disease Definition
1,975+
Rare Disease studies
345+
unique sponsors with Rare Disease trials
95K+
sites set up to conduct Rare Disease trials
305K+
enrolled Rare Disease patients
Video
Advancing Rare Disease Trials
Glen de Vries, Medidata Co-Founder and Co-CEO, on how Medidata is advancing Rare Disease trials, one patient at a time.
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' 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.
John Lee, MD, PhD
Chief Medical Officer, PhaseBio
Dr. Miganush Stepanians
President and CEO, PROMETRIKA