- Topics covered
- Patient Focused Drug Development (PFDD)
- Real World Evidence/Data
- Goals for today
- Terminology
- Patient Focused or Centered Drug Development
- Prescription Drug User Fee Act
- History of Patient Focused Drug Development
- 21st Century Cures Act
- FDA (Draft) Guidance
- What else is in the CURES Act?
- Definitions
- Real World Data
- Real World Evidence
- Randomized Controlled Trials (RCT)
- Real World Data/Evidence
- Why Real World Data/Evidence?
- Current use of Real World Evidence/Data
- (MORE) Definitions
- Large Simple Trials (LST)
- Pragmatic Clinical Trials (PCT)
- RCT vs PCT – Internal and External Validity
- Future Use of RWD/E – FDA Framework
- Use of RWD/E in Decision Making
- FDA Investing in RWD/E
- Real World Data Possibilities
- Key Elements of Real World Data
- Provenance
- Lineage
- Integrity
- Topics for Today
Nancy Pire-Smerkanich, DRSc, MS
Associate Director, Regulatory Knowledge and Support