Animal Efficacy Studies

Toxicology studies must be performed in at least two animal species. If the toxicity profile of the compound is acceptable, then it joins the hit or lead list of compounds to proceed. The metabolism of the compound must be understood and pharmacokinetic studies must be performed in small and large animals. Efficacy studies must be performed in relevant animal models, especially in chimpanzees when more than one candidate is identified and a choice has to be made before proceeding to studies in humans. The ultimate preclinical steps include various studies testing drug combinations in vitro and in vivo, selection of resistant viruses, viral fitness, pyrophosphorolysis, and others. 

Human or animal efficacy studies Chemical assays for quality control of commercial products... 

The use of AAAs as feed supplements appears an attractive approach either in itself or in combination with pro-, pre- and synbiotics, but the lack of animal feeding studies in which the efficacy of this approach could be determined makes it difficult to assess to what extent a two-barrier (targeting control at both the stomach and intestinal level) approach is commercially feasible. Also, since the viability of probiotics may also be affected by the use of AAA to increase the disinfection activity of the stomach, probiotics may need to be formulated in a way that protects them during stomach transfer. However, its potential should be determined in future research. 

Diaminoindane 4 has an IC50 of 0.7 nM in the in vitro MTP inhibition assay, and has shown in vivo efficacy in various animal model studies [15]. No clinical trials of 4 have been reported. 

Many of the issues relating to the definition of nonchni-cal laboratory study were addressed in the discussion of GLP 58.1 (Scope). Field trials in animals includes all efficacy studies of new animal drugs. Such studies are outside the scope of the GLP regulations. This is consistent with the GLP exemption for human clinical trials. The exemption for basic exploratory studies carried out to determine whether a test article has any potential utility would extend to early screening studies of a test article, the results of which are used to determine whether a test article merits further development or not. 

There are myriad clinical issues that can benefit from a forward quality perspective and commitment. The entire process—from synthesizing a compound in the laboratory and establishing toxicology in animal studies to subsequent pharmacological and efficacy studies in human trials—offers opportunities for and legitimately requires QA. This is an enormously costly process (typically over 100 million) therefore, embracing a forward quality mindset from the onset of clinical development and throughout clinical trial activities is extremely beneficial. 

Although extensive in vitro testing is performed on each immunotoxin to assess its cytotoxicity and specificity, it is animal model studies that ultimately determine the serum half-life, serum stability, as well as the efficacy of an immunotoxin. A compilation of the in vitro and in vivo data enables a decision to be made on the potential of each immunotoxin to be an effective and safe therapeutic and to determine whether human clinical trials are warranted. 

The results of any efficacy studies in experimentally infected animals should be summarized. 

Development of a Mixture of Dietary Carotenoids as Cancer Chemopreventive Agents C57BI./6J Mice as a Useful Animal Model for Efficacy Studies with Carotenoids... 

Rosenberg MP, Bortner D. Why transgenic and knockout animal models should be used (for drug efficacy studies in cancer). Cancer Metastasis Rev 1998 17 295-9. 

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