Bussiness Communication

Phase
0
1
2
3
Also known as
Microdosing
Human Pharmacology
Therapeutic Exploratory

2a

2b
Therapeutic Confirmatory

3a

3b
Objective
PK /PD /Other
Enable go/no-go decisions to be based on relevant human models instead of relying on sometimes inconsistent animal data.
Safety & tolerability-Maximum tolerated dose
PK
PD safe dose range
PK
Safety

Efficacy
PK
Safety
Efficacy
Safety

Special studies (Qol, DDI, FDI)
Sub group
Efficacy
Safety

Special studies (Qol, DDI, FDI, PE)

Info for mkt
Subjects
Generally Healthy volunteer
Generally Healthy volunteer
Patients (homogenous)
Patients
(heterogeneous)
Patients
(heterogeneous)
Patients
(heterogeneous)
Site
Single, CPU
Generally Single, CPU
Generally Single,
Specialized hospital units
Generally Multiple, Specialized hospital units
Generally Multiple, Multispecialty Hospital
Generally Multiple, Multispecialty Hospital
Outcome
PK/PD/Other

Accelerator mass spectrometry (AMS)
Safe dose range
Bioavailability
Nature of adverse drug reactions
Secondary: drug activity, potential therapeutic benefits
Safe dose schedule, characterization of dose response curve, Nature of adverse drug reactions Efficacy, Nature of adverse drug reactions
Confirm efficacy Information for regulatory submission

Comparison with standard

Confirm efficacy

Information for Mkt

Comparison with competitor

Prerequisites
Preclinical data + EC & RA approval
Preclinical data + EC & RA approval
Preclinical data + Clinical+ EC & RA approval
Preclinical data + Clinical+ EC & RA approval
Preclinical data + Clinical+ EC & RA approval
Preclinical data + Clinical+ EC & RA approval
Control
Generally no control
Placebo or
Generally no control
Placebo or active
Generally active
Generally active
Types
-
SAD & MAD
Pilot & Pivotal Mandatory & optional

The description of the study population should be stated in the inclusion & exclusion criteria,