1. Drug Development Stages
IRB continuing education
Walter K. Kraft
Fall 2018

4. Sage words from an olde tyme drug developer
When the molecule is born, its fate is determined. It's up to us to find out what the truth is, but in the end we can't change its fate.
The more skilled we are, the sooner we know the fate.

5. Preclinical Identifying diseases Understanding biology of the system
Formulating molecules
Test tube engagement
Animal testing in at least 2 species

6. Investigational New Drug (IND)
FDA approval to test in humans
30 days to place on hold
Very high standards for manufacturing, stability, purity, proof of concept, animal

7. Phase 1 Generally healthy volunteers Characterize 50-200 volunteers
Pharmacokinetics
Safety
Drug interactions
Pharmacodynamics
volunteers
In oncology, patients used

8. Phase I Oncology Trials
Usually no benefit to subjects
Common therapeutic misconception among patients
More recently, in healthy volunteers for non- cytotoxic drugs

9. Simvastatin Phase II dose range study in 43 individuals
Phase III patient 4S trial linked simvastatin to reductions in CV in events.

11. As IRB reviewer
FDA approval does not mean everything is known about a drug
We assess risk and ensure it is conveyed to subjects
We do not seek the fool’s errand of eliminating risk
What stage is a drug trial?
What is the specific question being asked by the study?
What is the uncertainty around drug safety and efficacy?