Table 2

Information needed to support request for clinical testing of a drug candidate (IND filing to FDA).



Chemistry, Manufacturing and Control (CMC)

Compound with acceptable stability and formulation

Controlled production under cGMP (current Good Manufacturing Processes)

Absorption, distribution, metabolism and excretion (ADME)

Route of administration, half-life

Metabolic pathways

Potential drug-drug interactions (including effects on cytochrome P450 enzymes)


Systemic and organ toxicity: gross and microscopic changes; two animal species, covering time periods of intended human exposure

Estimated safety window between efficacious dose and 'no observed adverse effect level' (NOAEL)

Initial data on potential genotoxicity and cardiotoxicity

Mechanism of action and pharmacology

Effects on receptor in vitro

Efficacy in animal models in vivo

Clinical development plans

Detailed protocol of initial studies

FDA, Food and Drug Administration; IND, Notice of Claimed Investigational Exemption for a New Drug.

Hefti BMC Neuroscience 2008 9(Suppl 3):S7   doi:10.1186/1471-2202-9-S3-S7

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