Being "One in a Million" Just Got Better

Being "One in a Million" Just Got Better

Pediatricians are known for saying that children aren’t just small adults; and their healthcare needs shouldn’t be treated that way.  Giving children less of the same medicine or ordering a smaller MRI just doesn’t work. 

With that in mind, the population of patients with rare diseases isn’t just a smaller version of patient populations with more common illnesses.  Treating them that way would be just as ineffective.  We understand that patients with rare diseases lead healthier lives when drug support programs are built around their specific needs.  The Safety and Innovation Act, signed into law this week, also recognizes the needs of rare disease patients and provides them with greater access to new therapies. 

Of particular interest for our industry and the patients we serve are the following provisions, noted by the National Organization for Rare Disorders (NORD):

  • Accelerated patient access to new medical treatments
  • Resolution of conflict-of-interest provisions introduced in the previous PDUFA reauthorization
  • Accelerated development of “breakthrough therapies” that show early promise
  • Enhanced FDA consultation with rare disease medical experts
  • A rare pediatric disease priority review voucher incentive program
  • Development of Humanitarian Use Devices (medical devices for small patient populations)

At HealthBridge Pharma & Biotech, more than 90% of products we support in our specialty pharmacies are classified as orphan or ultra orphan therapies. We fully support this legislation and recognize its benefits to our manufacturer customers and their patients.