Mastering Drug Discovery

<p>To test the drug in a general population</p> Signup and view all the answers

<p>Months to 2 years</p> Signup and view all the answers

<p>Phase II</p> Signup and view all the answers

<p>To evaluate the effectiveness of the drug in a specific target population</p> Signup and view all the answers

<p>Forever</p> Signup and view all the answers

<p>To determine the safety and adverse effects of a drug</p> Signup and view all the answers

<p>To evaluate the effectiveness of the drug in a specific target population</p> Signup and view all the answers

<p>False</p> Signup and view all the answers

<p>True</p> Signup and view all the answers

<p>True</p> Signup and view all the answers

<p>True</p> Signup and view all the answers

<p>True</p> Signup and view all the answers

<p>Phase III</p> Signup and view all the answers

<p>To evaluate the effectiveness of the drug in the general population</p> Signup and view all the answers

<p>Months to 2 years</p> Signup and view all the answers

<p>Phase I</p> Signup and view all the answers

<p>To evaluate the effectiveness of the drug in a specific target population</p> Signup and view all the answers

<p>True</p> Signup and view all the answers

<p>To determine the safety and adverse effects of the drug</p> Signup and view all the answers

<p>Forever</p> Signup and view all the answers

<p>Phase II</p> Signup and view all the answers

<p>False</p> Signup and view all the answers

<p>Clinical need and commercial potential</p> Signup and view all the answers

<p>The number of drug targets in the human genome</p> Signup and view all the answers

<p>To understand the molecular mechanisms of disease</p> Signup and view all the answers

<p>To confirm hits</p> Signup and view all the answers

<p>The re-examination of PD, PK, toxicity, selectivity, etc.</p> Signup and view all the answers

<p>All of the above</p> Signup and view all the answers

<p>Toxicity testing</p> Signup and view all the answers

<p>Nephrotoxicity, hepatotoxicity, immunotoxicity</p> Signup and view all the answers

<p>To evaluate the safety and effectiveness of the drug in a large number of patients with the disease</p> Signup and view all the answers

<p>To evaluate the safety and effectiveness of the drug in a large number of patients with the disease</p> Signup and view all the answers

<p>Target identification and validation</p> Signup and view all the answers

<p>Phenotype-based drug discovery and target-based drug discovery</p> Signup and view all the answers

<p>Rationale drug design</p> Signup and view all the answers

<p>To determine the safety and adverse effects of the drug</p> Signup and view all the answers

<p>Phase I, Phase II, Phase III</p> Signup and view all the answers

<p>Phase III</p> Signup and view all the answers

<p>Unmet medical need and economic viability</p> Signup and view all the answers

<p>To determine the effectiveness of the drug in a specific target population</p> Signup and view all the answers

<p>A few years</p> Signup and view all the answers

<p>To evaluate the safety and adverse effects of the drug in the general population</p> Signup and view all the answers

<p>Drug Discovery, Preclinical development, Clinical trials, Post-marketing surveillance</p> Signup and view all the answers

<p>To determine the safety and adverse effects of a drug</p> Signup and view all the answers

<p>To determine the safety, effectiveness, and dose of a drug in a specific target population</p> Signup and view all the answers

<p>To evaluate the effectiveness of a drug in a large number of patients with the disease</p> Signup and view all the answers

<p>To assess the effectiveness of a drug in the general population and monitor safety and adverse effects</p> Signup and view all the answers

<p>Several months</p> Signup and view all the answers

<p>Months to 2 years</p> Signup and view all the answers

<p>1-3 years</p> Signup and view all the answers

<p>1-2 years</p> Signup and view all the answers

<p>To identify and validate a potential target for the drug</p> Signup and view all the answers

<p>Target identification and validation, phenotype-based drug discovery, target-based drug discovery, lead discovery and optimisation, preclinical assessment of toxicity and safety, clinical assessment of efficacy, toxicity and safety.</p> Signup and view all the answers

<p>Phenotype-based drug discovery involves screening compounds for their ability to produce a desired phenotype, while target-based drug discovery involves identifying and validating a specific target for the drug.</p> Signup and view all the answers

<p>Lead optimisation is the process of refining and improving the properties of a lead compound to make it more effective and safe as a drug candidate.</p> Signup and view all the answers

<p>Unmet medical need, economic viability, size of target population, feasibility of drug development, and information about the disease.</p> Signup and view all the answers

<p>The purpose of preclinical assessment is to evaluate the toxicity and safety of a drug candidate before it is tested in humans.</p> Signup and view all the answers

<p>Phase I, Phase II, and Phase III.</p> Signup and view all the answers

<p>The purpose of Phase I clinical trials is to evaluate the safety and dosage of a drug candidate in a small group of healthy volunteers.</p> Signup and view all the answers

<p>The purpose of Phase II clinical trials is to evaluate the effectiveness and side effects of a drug candidate in a larger group of patients.</p> Signup and view all the answers

<p>The purpose of Phase III clinical trials is to confirm the effectiveness and monitor side effects of a drug candidate in a larger population.</p> Signup and view all the answers

<p>The purpose of Phase IV clinical trials is to monitor the long-term safety and effectiveness of a drug after it has been approved for marketing.</p> Signup and view all the answers

<p>Clinical need and commercial potential, involvement in disease pathophysiology, target-related safety, primarily expressed on tissues of interest, manageable on-target adverse effects, druggable, availability of assays for screening</p> Signup and view all the answers

<p>The subset of the human genome that can be modulated by low molecular weight compounds with 'drug-like' properties</p> Signup and view all the answers

<p>It involves understanding the molecular mechanisms of the disease, which can sometimes be straightforward for monogenic diseases but often complex for polygenic diseases. It can also involve the use of big data and translational relevance.</p> Signup and view all the answers

<p>To identify 'hits' by developing an assay amenable to high-throughput screening and using compound libraries to screen for compounds that elicit a desired phenotype.</p> Signup and view all the answers

<p>Lead optimisation is the process of evaluating and refining leads. It involves medicinal/synthetic chemistry, generating structural analogues, evaluating structure-activity relationships, and re-examining pharmacodynamics, pharmacokinetics, toxicity, and selectivity.</p> Signup and view all the answers

<p>An IND dossier includes information about the drug's toxicity profile, pharmacological profile (in vitro and in vivo), pharmacodynamic and pharmacokinetic data, dose information, chemical/manufacturing information, formulation, proposed clinical plans/protocols, and toxicity testing.</p> Signup and view all the answers

<p>The types of toxicity assessed in preclinical assessments include nephrotoxicity, hepatotoxicity, cell death (necrosis/apoptosis), hypersensitivity/immunosuppression, acute toxicity, genotoxicity, chronic toxicity, immunotoxicity, carcinogenicity, mutagenicity, and teratogenicity.</p> Signup and view all the answers

<p>The factors considered when choosing a disease for drug discovery include clinical need, commercial potential, evidence that the target is involved in disease pathogenesis, and evidence that modifying the target alters the disease state.</p> Signup and view all the answers

<p>Preclinical development involves assay development and screening, hit and lead identification, lead optimisation, and preclinical assessments such as toxicity testing and pharmacological characterisation.</p> Signup and view all the answers

<p>The rationale behind drug design during lead discovery and optimisation is to generate or obtain structural analogues of leads, evaluate their structure-activity relationships, and re-examine their pharmacodynamics, pharmacokinetics, toxicity, and selectivity.</p> Signup and view all the answers