Every year, thousands of people face a diagnosis of one of the six to eight thousand identified rare disease conditions.
The majority of rare diseases are genetic, and over half of patients suffering from them are children. Today, less than five percent of rare diseases have even a single approved treatment, and accessing the right medical treatments can be a significant challenge for many.
Diagnosis for adults with a rare disease can take up to five years, and half will receive misdiagnoses along the way. The need for increased awareness, research and expedited treatment pathways in rare diseases remains critical.
Rare diseases challenge traditional ways of doing business. They require the industry to identify target patient populations for trials and then keep them engaged during the course of the study, even across geographies.
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