Imagine you're a patientYou're a patient with an ultra-rare diagnosis, and there is a cutting-edge clinical trial available, but the nearest clinical trial treatment center is 5 states away. You and your family pack up [...]

Navigating the regulatory landscape for rare disease treatments can be challenging. One critical decision is determining the right time to seek an FDA special designation, such as Orphan Drug, Fast Track, Breakthrough Therapy, or Rare [...]