The life sciences community has created a long list of medical breakthroughs for people with rare diseases. Many additional significant advances are likely during this new decade. However, the U.S. health care system has been unable to keep pace to make these innovations widely available to patients.
“Maisie’s Journey” – Maisie suffers from Spinal Muscular Atrophy (Type 1). See her incredible story here:
As many as 30 million Americans currently live with a rare disease. The U.S. Food and Drug Administration (FDA) defines an orphan (rare) disease as a condition that affects fewer than 200,000 people nationwide. Some rare diseases have patient populations of fewer than one hundred people.
Facts about rare diseases
“Action” is a key component of who we are and what we do. While think tanks play important roles for advancing science, medicine and policy, the Rare Access Action Project (RAAP) focuses on specific, achievable and measurable campaigns designed to support the rare disease community and its various stakeholders.
The Rare Access Action Project (RAAP) announced results of a devastating report conducted on the impact of proposed change to Medicare D to companies that develop treatments for Rare Diseases,...Read More