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 NEXT Global

Rare Disease

Tech adopters, clinical research innovators, and patient champions discuss solutions and new methodologies suited to rare disease challenges and how to improve the patient experience.

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

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

Rare Disease studies

unique sponsors with Rare Disease trials

sites set up to conduct Rare Disease trials

enrolled Rare Disease patients


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.


Opportunities to conduct Rare Disease clinical trials are scarce.

How to “get it right” from the start?


Learn from the experts.

Novel Challenges in Rare Disease Clinical Development: Perspectives from the Experts

Improving Access to Clinical Trials for Rare Disease Patients through Registries

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.

Phase Bio

"Medidata Rave has the capability to scale with us. We can monitor multiple studies in parallel and potentially merge data sets as needed."

John Lee, MD, PhD
Chief Medical Officer, PhaseBio


"Rare disease sponsors day to day challenges are managing many measurements in a small number of patients that are very hard to find, and very hard to keep. So the more integrated and unified the data collection approach, the better you will fare in a complex trial.""

Dr. Miganush Stepanians
President and CEO, PROMETRIKA