Regulatory Science Symposium: Patient-Centered Drug Development and Real World Evidence/Data Session 2: History, Terms and Definitions (2019)

1. Topics covered
   1. Patient Focused Drug Development (PFDD)
   2. Real World Evidence/Data
   3. Goals for today
2. Terminology
   1. Patient Focused or Centered Drug Development
3. Prescription Drug User Fee Act
4. History of Patient Focused Drug Development
5. 21^st Century Cures Act
6. FDA (Draft) Guidance
7. What else is in the CURES Act?
8. Definitions
   1. Real World Data
   2. Real World Evidence
   3. Randomized Controlled Trials (RCT)
9. Real World Data/Evidence
10. Why Real World Data/Evidence?
11. Current use of Real World Evidence/Data
12. (MORE) Definitions
    1. Large Simple Trials (LST)
    2. Pragmatic Clinical Trials (PCT)
13. RCT vs PCT – Internal and External Validity
14. Future Use of RWD/E – FDA Framework
15. Use of RWD/E in Decision Making
16. FDA Investing in RWD/E
17. Real World Data Possibilities
18. Key Elements of Real World Data
    • Provenance
    • Lineage
    • Integrity
19. Topics for Today

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