The State Of Rare Disease Clinical Trials

The technologies and supporting procedures that researchers use to gather, manage, and analyze clinical trial data will have a significant impact on how quickly and effectively they can get new medications to patients who need them as they look for novel approaches to treat rare diseases.

Rare disease trials are clinical studies designed to evaluate the safety and efficacy of treatments for rare diseases, also known as orphan diseases. In the United States, rare diseases are defined as conditions that affect fewer than 200,000 people.¹ The European Union defines a rare disease as affecting fewer than 1 in 2,000 people.² However, according to the National Center for Advancing Translational Sciences, 1 in 10 people are affected by a rare disease, 1 in 2 diagnosed are children, and their estimated yearly medical care costs total about $400 billion.³

Gain insight from the challenges, recent progress, and study needs in the rare disease sector to better determine the right unified platform to support rare disease research goals in clinical trials.