The Need for Clinical Studies in Rare Disease
Check the Stats
Here’s a staggering statistic: only 5 percent of rare diseases have approved treatments. But that doesn’t mean there isn’t extensive work and research being done to find therapies, procedures, and eventually, cures for these conditions.
Clinical studies are essential stepping stones to medical advances, but only one in ten patients have participated in one. That’s understandable: clinical studies can seem—and in fact be—intimidating, time-consuming, complicated, or all of the above. Furthermore, 88 percent of patients say their doctor didn’t refer them to a clinical trial, 42 percent of patients lack information about logistics such as clinical study locations and requirements, and 74 percent of patients simply aren’t aware of available clinical studies.
However, half of patients say studies can help their experience after diagnosis, and eight out of every ten patients want studies to be available for new treatment options (about two-thirds want to participate in studies related to quality of life, while over a third are interested in studies about medical devices, and over half want to take part in studies supporting gene therapy research). Clinical studies can also help patients get access to leading experts for their conditions, and contribute to advancing scientific and medical research.
To find out more about options for clinical studies, talk to your healthcare professional and visit the National Institute of Health’s resources on clinical trials.
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