It is estimated that 400 million people suffer from a rare disease globally, with approximately 7,000 distinct types of rare and genetic diseases identified. On average, it takes eight years before rare disease patients receive an accurate diagnosis and even then, the majority of those diagnosed lack access to approved therapies for targeted treatment. Many of these rare diseases affect children, with irreversible debilitating and painful impact that is often fatal. Thus, it is essential to identify patients early and connect them to treatment as soon as possible to improve patient outcomes.
Potential impact of the CMS GENEROUS model
Reference country selection The model provided guidance on reference countries, defined by the G7 countries plus Denmark and Switzerland. Reference...