Rare Disease Research: Finding Participants And Driving Progress

By Ian Davison, RTSM Subject Matter Expert; Melissa Newara, Vice President of Subject Matter Expertise; Rod McGlashing, Data Science Subject Matter Expert; and Tina Caruana, eClinical Solutions (Digital & Decentralized Trials) Subject Matter Expert

In the United States alone, there are currently 7,000 recognized rare diseases, many of which lack effective treatments. However, research into these conditions is gaining momentum, as evidenced by the fact that over half of the FDA’s novel drug approvals in 2022 were for rare or "orphan" diseases.

The unique characteristics of participants and their families significantly drive progress in rare disease research. Individuals affected by these diseases, along with their loved ones, often show a remarkable eagerness to participate in clinical research and actively contribute to raising awareness and funding for new treatments.

This unparalleled determination and commitment can accelerate research efforts considerably. In this article, you will discover where to find participants for rare disease studies, how to leverage ongoing funding and advocacy for rare disease cures, and strategies to enhance recruitment and engagement in your trials.