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However, LASV vaccine development has been hindered by the lack of appropriate small animal models for efficacy evaluation independent of biosafety level four (BSL-4) facilities.
It is desirable to test the selected AOs targeting human dystrophin exons in animal models for efficacy as part of AO drug development.
Figure 2 presents simulation results to evaluate the impact of changing the priors for the parameters of the logistic regression models for efficacy and toxicity.
Some suggest animal models can be skipped if dosing remains constant in repurposing; however, the case is often not so simple and new indications can require different dosages which will require mouse models for efficacy testing.
Although, the performance of the Braun model suffered more than the performance of the Gumbel model when vague priors were placed on the slope parameters in the logistic regression models for efficacy and toxicity.
While the use of preclinical animal models for efficacy assessment will probably also persist, it is less clear that current preclinical animal models are entirely adequate or appropriately used (even when adequate), or that an optimal panel of such models to predict efficacy with sufficient accuracy currently exists.
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Marzi, A. et al. Stat1-deficient mice are not an appropriate model for efficacy testing of recombinant vesicular Stomatitis virus-Based Filovirus Vaccines.
Generally, several factors should be considered for an efficient and safe mucosal vaccine development, including the antigen, adjuvant, formulation, administration route and animal model for efficacy and safety evaluation.
Extra caution should be taken when using ED50's from rat LPS model for efficacy and safety evaluation for inhale ICS with regard to the outcome, most importantly, the translatability to human.
In addition, these methods require that we specify a joint probability model for efficacy and toxicity, which is often accomplished using a copula model.
In the context of a phase I-II clinical trial, we specify parametric models for the dose-response relationship for toxicity and efficacy and a copula model is used to specify a joint model for efficacy and toxicity.
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