Testing Models, Safety and Efficacy of the Drug ZL105

Answer

Testing Models, Safety and Efficacy of the Drug ZL105

            The purpose of this paper is to discuss the approach for testing the efficacy and toxicity of the experimental Drug ZL105.  The models to be used in testing the drug will include cell cultures, animal and human participants.  The efficacy, lethality, and toxicity should be tested when determining the safety and effectiveness of the drug. Important issues to be gathered from the tests include efficacy range in different types of cancers, safety, lethality, and adverse effects.

Models

Cell Cultures

            Wild-type cells lines and cancerous cells lines can be used for testing the safety, efficacy of the anti-cancer agent. Cell lines can provide valuable information since aspects such as copy number variations, methylation, gene expression, and single nucleotide variants can be known beforehand (Parca et al., 2019).  Cell cultures are characterized by a significant degree of reliability in informing human health in terms of reproducibility and repeatability (Piccinini et al., 2017). Cell cultures create an environment which mimics the conditions that are found in tumors in vivo. In addition, cell lines simulate physiologic cellular microenvironment that allow for reconstruction of tissue-like cytoarchitecture with cell-to-extracellular matrix interactions, cell-to-cell interactions, and exhibit differentiation. Besides, therapeutic response can be observed similar to those that are observed in vivo. Cell cultures can be used for determining the effectiveness, safety, and lethality of the drug (Ryan et al., 2016). The advantages of this method include cost effectiveness, it is a more accurate representation of the in vivo scenario, and provides a good model for determining drug resistance. The disadvantages include complex downstream processing and the endpoint assay is dependent on the culture method utilized (Ryan et al., 2016).  The disadvantage is that the test can be performed on cancerous cell lines, which have abnormal function; hence, may not completely portray physiological functions (Saeidnia, Manayi, & Abdollahi, 2015).

Animals Models

            Animal models provide excellent in vivo physiological conditions for testing lethality, effectiveness, safety and toxicity of the Drug ZL105. Animal models are more reliable compared to in vitro tests. Animal models are characterized by some drawbacks like the need to extrapolate the results to humans and that there are differences in biokinetics parameters between animal models and humans (Saeidnia, Manayi, & Abdollahi, 2015). The other disadvantage is that the use of animal models raises ethical and moral challenges. Some proportion of the population considers the practice as amounting to cruel treatment of animals. In addition, it is expensive to keep and use laboratory animals (Moran et al., 2016). The other limitation is that ethical approval is needed before the study is conducted (Payab et al., 2018). Animal studies are unreliable in informing human health since there exists differences in human and animal disease as well as species differences in genetics and physiology (Aktah, 2015).

Human Participants

            Human participants provide the ultimate model for testing Drug ZL105 owing to the fundamental reason that the drug is intended for use in human beings for the treatment of cancers.  Reliability of findings can be achieved through the use of appropriate research design and control of bias (Noble & Smith, 2015). The research should be conducted in accordance with human rights law, ethical principles and values, universally accepted set rules, and legislations. Informed consent is a prerequisite before human participants are recruited into the study (Constantin, 2018). The advantages of using human participants in studying the safety and efficacy of Drug ZL105 is that participants can gain access to novel agents in trial, gaining of focused health care for cancer, and that the investigator can gain insight on the drugs pharmacokinetic and pharmacodynamics, identify effects, efficiency, and adverse effects. The disadvantage of using human participants is that it may lead to harm and adverse events, may pose ethical challenges, especially if vulnerable persons are used or if participants are paid to participate in the study (Wikler, 2015). 

Determining the Safety and Effectiveness of the Drug

            In determining the safety and effectiveness of Drug ZL105, it would be necessary to test for efficacy, lethality, and toxicity. Efficacy refers to therapeutic effect of the drug. The range of cancers that can be treated using the drug need be identified. Efficacy includes identification of an effective dose and route of administration (Brunetti et al., 2016). Lethal dose of the drug (expressed as LD₅₀) is referred to as the estimated dose necessary to kill 50% of a given group of experimental animals. Other measures such as no toxic effect level, no observed adverse effect level, maximum tolerated dose, and no observed effect level should be studied for this drug (Wypych & Wypych, 2015). Toxicity of the drug will include development of an understanding about its harmful or poisonous effects. It includes identification of adverse effects. Toxicity assessment can be done through large scale animal studies. Caenorhabditis elegans can be used for rapid assessment of the acute toxic effects, reproductive and developmental effects (Xiong, Pears, & Woollard, 2017).

Other Important Information

            The other important information about the drug needs to be gathered. Usability of the drug in different age groups, in pregnancy, and in people with various existing acute and chronic conditions needs to be determined (Jackson, 2016). Additional drug information that is also needed include dosages, route of administration, dosage frequency, adverse effects, drug interactions, impact of existing chronic and acute conditions, and short-term and long-term effects.

 Different protocols should be used when studying for the safety and efficacy of different products. Thus, different protocols are required for studying different substances such as cosmetics, drugs, and herbal medicines, since cosmetics may pose relatively lower risk to human health and environment as opposed to drugs and herbal medicines (Wypych & Wypych, 2015).