BRIEF -- Latest QuintilesIMS Institute analysis on Orphan drugs

In the USA, the National Institutes for Health estimates that between 25 million and 30 million people suffer from rare diseases - defined as those affecting fewer than 200,000 people.

Rare diseases are serious, chronic illnesses that can become progressively disabling and can limit life expectancy. There are approximately 7,000 rare diseases and over 25 million patients affected by them in the USA. These patients and their families form a distinctive community, with half of patients being children, and with many caretakers and patients actively participating in groups to raise awareness.

Although there has been a high level of interest in rare disease medicines among healthcare stakeholders, there still remain significant gaps in patient support, challenges around timely and accurate diagnosis and no treatment options for many patients. This report provides perspective on rare diseases and their treatments, including an in-depth analysis on orphan drug volume and pricing in the context of specialty therapies