Development and Preclinical Studies

5 Animal Tests, In Vitro Assays and In Silica Methods 

In earlier chapters, we discussed the discovery of new drugs. After a lead compound has been identified, it is subjected to a development process to optimize its properties. The development process includes pharmacological studies of the lead compound and its effects on toxicity, carcinogenicity, mutagenicity and reproductive development. These data are important for determining the safety and effectiveness of the lead compound as a potential drug. 

An ideal drug is potent, efficacious and specific, that is, it must have strong effects on a specific targeted biological pathway and minimal effects on all other pathways, to reduce side effects. In reality, no drugs are perfectly effective and absolutely safe. The aim of pharmacological studies is to obtain data on the safety and effectiveness of the lead compound. Many iterations of optimization of the lead compound may be necessary to yield a potential drug for clinical trial. 

Exhibit 5.1 FDA 21 CFR Part 58 Good Laboratory Practice for Nonclinical Laboratory Studies Table of Contents 

Applicability to studies performed under grants and contracts 

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Before entering into a clinical evaluation program, a drug candidate is subjected to a number of preclinical studies to further define and characterize its safety profile. The results from the pharmacology, developability, and preclinical studies are documented in technical reports or scientific publications and used to prepare the IND submission. All of the preclinical studies, described in the section below, and nonclinical studies, discussed in the following section, need to be conducted according to GLP regulations. 

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