This week on The , we turn our attention to the topic of rare disease, and more specifically: how to engage and educate rare disease patients to advance development. It's estimated that nearly 30 million Americans, or nearly 1 in 10 people, are living with a rare disease - and almost 95% of rare diseases don't have any FDA-approved treatment.
One of the unique challenges and perhaps keys to rare disease development is patient engagement. Patient populations are small by definition. Roughly 50% of patients are children, and benefit-risk equations are completely different from those of more traditional diseases. To learn more about engaging rare disease patients, we sit down with Dr. Lorna Speid, to discuss her stories of rare disease drug development, outlines the unique challenges of engaging the rare disease population, and her advice to developers.