PM433 Drug Development & Emerging Therapies Assignment Sample NUIG Ireland
PM433 Drug Development & Emerging Therapies is a new drug candidate that is currently being developed as a treatment for age-related macular degeneration (AMD), diabetic retinopathy, and glaucoma.
AMD is a leading cause of vision loss in adults, affecting approximately 10 million people in Ireland. Diabetic retinopathy is the most common form of retinopathy and can lead to blindness in people with diabetes. Glaucoma is a leading cause of blindness worldwide, and it is estimated that there are over 60 million cases of glaucoma globally.
PM433 was discovered through the use of phenotypic screening, which allows for the identification of potential drug candidates by assessing their ability to improve or protect a cell or tissue from a specific disease or condition.
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In this section, we are describing some assigned briefs. These are:
Assignment brief 1: Use literature databases (e.g. PubMed) to find published manuscripts relevant to the main stages in the development of a CNS drug, and identify and extract the key information from the relevant manuscripts.
The main stages in the development of a CNS drug are preclinical testing, clinical trials, and then commercialization. To find manuscripts relevant to each stage, you can search literature databases such as PubMed.
In preclinical testing, scientists research and develop a potential new drug. This process starts with basic research to identify a target for the new drug. Then, researchers conduct animal studies to see if the drug is safe and effective. If the results of these studies are promising, the new drug moves on to clinical trials.
Clinical trials are conducted in phases. Phase I studies assess safety in a small group of people (20-80). Phase II studies evaluate whether the drug works in a larger group of people (100-300). Phase III studies compare the new drug to existing treatments in a large group of people (1,000-3,000). After a drug is approved by the FDA, it is available for commercialization.
You can use the following search terms to find relevant manuscripts on PubMed: preclinical testing AND “new drugs”, clinical trials AND “new drugs”, commercialization AND “new drugs”.
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Assignment brief 2: Compile and present the extracted data in clearly accessible formats (tabulated and essay).
Over the past few years, data extraction has become increasingly important as companies strive to make better use of the data they have available. Data extraction allows companies to get at the information they need quickly and easily, without spending hours sifting through irrelevant data.
There are several different ways to extract data, but one of the most popular methods is using an online tool like Google Sheets or Microsoft Excel. These tools allow you to quickly and easily filter and sort through large amounts of data, making it simpler to find the information you need.
Once you have extracted the data you need, it is important to present it in a clear and accessible format. This could involve creating graphs or charts to visualize the data, or simply tabulating the data in a way that is easy to understand.
When presenting the data, it is also important to consider your audience. For example, if you are presenting the data to a group of scientists, you will need to use language and terminology that they will understand. On the other hand, if you are presenting the data to the general public, you will need to use language that is easy to understand.
Assignment brief 3: Synthesise and critically assess the published literature to make an evidence-based judgment on the development of a CNS drug.
Critical assessment of the published literature is required to make an evidence-based judgment on the development of a CNS drug. The goal of this assessment is to determine the potential efficacy and safety of a drug in its proposed indication.
One important factor to consider is how well the drug performs in preclinical studies. For example, it is necessary to evaluate whether a drug demonstrates adequate efficacy and safety in animal models before progressing to human trials. Furthermore, it is also important to look at how a drug behaves in vitro (e.g., receptor binding, metabolism, etc.).
Another key factor is the clinical data that is available for a drug. This includes results from phase I, II, and III trials. It’s also important to look at the safety data that is available for a drug. This includes information on adverse events, as well as any other safety concerns that have been raised about a drug.
Finally, it is also important to consider the commercial viability of a drug. This includes factors such as patent protection and market size.
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Assignment brief 4: Demonstrate an awareness of the complexity of, and challenges inherent within, the CNS drug development process.
The CNS drug development process is complex and fraught with challenges. The brain is a uniquely complex organ, and many factors—including the blood-brain barrier, variations in gene expression, and the presence of multiple receptor subtypes—can make it difficult to develop drugs that effectively target the brain.
Another challenge is that the effects of drugs can vary significantly from one individual to another, making it difficult to predict how a drug will behave in humans. Preclinical studies in animals are essential for assessing a drug’s safety and efficacy, but they often do not accurately reflect what will happen when the drug is used in people. Clinical trials are essential for determining a drug’s safety and efficacy in humans, but they are expensive and time-consuming, and the results are often not definitive.
Due to the challenges inherent in CNS drug development, many drugs that show promise in early studies fail to meet their full potential. It is estimated that only 1 in 10 drugs that enter clinical trials are ultimately approved by regulatory agencies such as the FDA.
Assignment Brief 5: Make an evidence-based, critical judgment on the potential of an emerging therapy/novel target to address the unmet clinical needs of, and limitations of existing therapies for, human CNS conditions.
The potential of an emerging therapy/novel target to address the unmet clinical needs of a patient population is always an exciting prospect. However, it is important to temper this excitement with a critical evaluation of the available evidence. To make an evidence-based, critical judgment of the potential of an emerging therapy/novel target, one must consider the limitations as well as the promising aspects of the therapy/target in question.
One must first consider what unmet clinical needs the therapy/target aims to address. Is there a significant population of patients who stand to benefit from this therapy/target? What are the risks and side effects associated with the use of this therapy/target? How does this therapy/novel target compare to existing therapies?
It is also important to consider the evidence that is available for the therapy/target in question. This includes preclinical data, as well as any clinical data that may be available. Does the evidence support the efficacy and safety of this therapy/target? Are there any major concerns that have been raised about the use of this therapy/target?
Taking all of these factors into consideration, one can then make an evidence-based, critical judgment on the potential of an emerging therapy/novel target. If the therapy/target in question shows promise in addressing the unmet needs of a patient population and has a favourable risk-benefit profile, then it may be worth further investigation. However, if there are significant concerns about the safety or efficacy of the therapy/target, then it may not be worth pursuing.
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Assignment Brief 6: Design and prepare an informative and visually accessible poster using PowerPoint software that highlights the evidence for progression (or otherwise) of an emerging therapy/novel target to the clinical marketplace.
Using PowerPoint, you can create an informative and visually appealing poster on anything from the progression of a disease to the steps of a scientific process. In this example, we’ll highlight the evidence for (or against) the progression of a particular disease.
First, select your slide size. We recommend using a landscape format for posters. Then, add a title and subtitle if desired.
Next, insert your images and text. For each image, be sure to include a brief caption and alternate text for those who cannot see the image. In addition, be sure to use clear and concise language throughout your poster so that it is easy to understand at a glance.
Finally, add any necessary finishing touches such as your name, contact information, and the date.
Assignment Brief 7: Engage in a scientific discussion regarding the potential of an emerging therapy/novel target for the treatment of human CNS disease.
The potential of an emerging therapy will always beam an intriguing topic for scientific discussion. A novel target for the treatment of human CNS disease opens up a new avenue of exploration and research. The hope is that this new target will eventually lead to more effective treatments with fewer side effects. While we don’t yet know all the possible benefits or risks associated with this therapy, it’s important to keep an open mind and continue learning more about it. Exciting discoveries often come from unexpected places, so we should never lose sight of the potential for new therapies to make a big impact.
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