Patient-Centered Drug Development and Real World Evidence/Data Boot Camp Session 4: Rare Diseases (2019)


Topics

  1. Agenda
  2. Rare Diseases & Orphan Drugs: What are they?
  3. Current Trends in Rare Diseases and Orphan Drug Development
  4. What focus on the diagnostic journey?
  5. Charting the path to shorten the journey to diagnosis
  6. Technology pilots
  7. Current Trends in Rare Diseases and Orphan Drug Development
  8. Number of Orphan Drug Designation Requests by Year
  9. Number of Orphan Drug Designations by Year
  10. Orphan Drug Approvals
  11. Current Trends in Rare Diseases and Orphan Drug Development
  12. Legislative Landscape in the US
    1. Orphan Drug Act (ODA) – Great Success Story
    2. Rare Diseases Act
  13. Orphan Drug Designation Incentives
  14. Approvals Before and After ODA and EU Legislation
  15. FDA’s Commitment to Rare Diseases
  16. Progress has been made…
  17. Orphan Drug Development Challenges: Just a Few Examples
  18. Value and Opportunities in Bringing an Orphan Drug to Market
  19. Value and Opportunities in Bringing an Orphan Drug to Market: Orphan Pricing Rationale
  20. Value and Opportunities in Bringing an Orphan Drug to Market
  21. Rare Disease Advocacy
    1. Approach
    2. List of some Organizations

Acknowledgement

Accompanying text created by Khyati Ashtekar | Graduate Student, Regulatory Science, USC School of Pharmacy | ashtekar@usc.edu  


Instructors

NIH Funding Acknowledgment: Important - All publications resulting from the utilization of SC CTSI resources are required to credit the SC CTSI grant by including the NIH funding acknowledgment and must comply with the NIH Public Access Policy.