Regulatory Science Symposium: Patient-Centered Drug Development and Real World Evidence/Data Session 4: Rare Diseases (2019)

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

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