• Drug safety and dose range
• Determine PK properties
• 20-100 healthy volunteers
• 6 - 12 months

• Drug efficacy within safe range
• Drug vs placebo
• 100 - 500 subjects
• 1 - 2 years

• Drug effectiveness vs competition
• Drug interactions
• Extrapolate for population
• 1000s of patients at multiple sites
• 1 - 4 years

investigational new drug

sent to FDA prior to trials

New rug application

Sent to FDA after all 3 trials

must show safety and efficacy

Supplimental new drug application

used for approved drugs to change a label. strength, use

abbreviated new drug application

used to gain approval for generic after brand patent expires

Doesn't need to prove safety and efficacy, only bioequivalence.

Risk Evaluation and Mitigation Strategy

required by FDA if drug is dangerous

cost minimization analysis

Cost benefit analysis

of a single item

cost effectiveness analysis

cost vs consequences

cost utility analysis

humanistic (intangible unmeasurable)

"quality years"

intention to treat

all participants (even dropouts)