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.
Part 4: Inflation Reduction Act: The indirect effects and implications of IRA price negotiations
Since passage of the Inflation Reduction Act (IRA) in August 2022, the focus of many industry analysts and pharmaceutical companies has been on two important...