Medidata Supports Patients on Rare Disease Day by Working to Address Challenges of Rare Disease Trials

February 28, 2022— Medidata, a Dassault Systèmes company, is honoring patients on Rare Disease Day by working to expedite critical rare disease clinical trials and supporting the work of key organizations to improve patient outcomes.

From helping locate eligible patients for trials, to discovering biomarkers, accelerating diagnosis, and delivering insights into complex patient populations, Medidata advances rare disease trials by expediting outcomes and improving experiences,” said Sastry Chilukuri, co-CEO of Medidata. “Our patient-centric focus on product development and leadership on technologies like Acorn AI’s Synthetic Control Arm help CROs and sponsors address rare disease trial challenges like patient enrollment and retention.”

Medidata has supported more than 1,800 rare disease clinical trials with nearly 300,000 enrolled patients. Rare diseases affect over 400 million people worldwide—over 7,000 rare diseases have been identified in the U.S. alone, but only 500 therapies have been approved. 

Medidata also enables the commercial activities of launching a rare disease therapy. Navigating the real world challenges of a drug launch are daunting, and that journey requires a trusted partner who understands the patient journey and the realities that patients, physicians, and caregivers go through.

Through navigating the complexities of diagnosis, access, reimbursement, and the intricate dosing regimens that often come with rare disease treatments, it’s all about getting the drug to the patient faster. And that can’t happen without the fast, accurate, aggregation and delivery of data.

To further rare disease research, Medidata partners with the Castleman Disease Collaborative Network (CDCN), the National Organization for Rare Disorders (NORD), and Project ALS.

“At Project ALS, we know complicated problems are solved faster when people work together. Our partnership with Medidata on research and identifying biomarkers has generated important insights into better understanding and diagnosing ALS,” said Margot Shanahan, associate director of research operations at Project ALS. “Rare disease day is an opportunity to both support people who are living with these diseases and to appreciate all those in the life sciences community who are working towards meaningful therapeutic strategies.”

To join Medidata in supporting patients on Rare Disease Day, visit our website and to learn how Medidata is incorporating the patient voice in rare disease research.


Tom Paolella
Senior Director, Corporate Communications & Affairs

Paul Oestreicher
External Communications Director