Unique Challenges and Opportunities in Orphan Diseases

Looking back, the 1983 Orphan Drug Act was a pivotal moment for healthcare, not just in the US but worldwide too. Research on orphan diseases has since flourished, paving the way in 2017 for the first US-approved gene therapy launch, and to date, hundreds of gene therapies are in the pipeline. Jeremy Edwards of Raremark, a patient community online specialist in rare conditions, outlines the challenges and opportunities in the orphan space.