Drug Discovery and Development
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Questions and Answers

Which of the following is NOT a characteristic of a good drug target?

  • High-throughput screening assays available (correct)
  • Involvement in the pathophysiology of the disease
  • Primarily expressed on tissues of interest
  • On-target adverse effects manageable

What is the approximate number of drug targets in the human genome?

  • 3,000 (correct)
  • 600-1,500
  • 20,000-22,000
  • 3,000-5,000

Which phase of clinical trials involves post-marketing surveillance?

  • Phase III
  • Phase IV (correct)
  • Phase I
  • Phase II

What does the term 'druggable genome' refer to?

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

Which phase of clinical trials involves testing the compound in humans to determine tolerability, effectiveness, and adverse effects?

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

In which phase of clinical trials is the drug tested on a larger group of healthy volunteers or a small group of diseased patients to assess its safety and effectiveness?

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

Which phase of clinical trials involves a large number of patients with the disease and aims to determine the effectiveness of the drug in the specific target population?

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

During which phase of clinical trials is the drug tested in the general population to evaluate its effectiveness and monitor safety and adverse effects?

<p>Phase IV (C)</p> Signup and view all the answers

Which of the following is a key step in drug discovery?

<p>Clinical assessment of efficacy and safety (C)</p> Signup and view all the answers

What are the major differences between phenotype-based drug discovery and target-based drug discovery?

<p>Phenotype-based drug discovery relies on information about the disease mechanism, while target-based drug discovery relies on lead compound identification. (A)</p> Signup and view all the answers

What factors contribute to the feasibility of drug development?

<p>Economic viability (B)</p> Signup and view all the answers

Which of the following is a key step in drug discovery?

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

Which phase of clinical trials involves testing the compound in humans to determine tolerability, effectiveness, and adverse effects?

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

What factors contribute to the feasibility of drug development?

<p>Lower risk (A)</p> Signup and view all the answers

What is the main purpose of target identification and validation in drug discovery?

<p>To identify potential drug targets that are involved in the pathogenesis of the disease (B)</p> Signup and view all the answers

What is the primary goal of lead optimization in drug discovery?

<p>To improve the drug-like properties of lead compounds (C)</p> Signup and view all the answers

What is the purpose of preclinical development in drug discovery?

<p>To evaluate the safety and toxicity of lead compounds (A)</p> Signup and view all the answers

What is the main goal of toxicity testing in drug development?

<p>To evaluate the safety and toxicity of lead compounds (C)</p> Signup and view all the answers

Which phase of clinical trials involves testing the compound in humans for the first time, typically in a small group of healthy volunteers?

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

What is the purpose of Phase II clinical trials?

<p>To determine the optimal dose and dosing frequency of the drug (A)</p> Signup and view all the answers

What is the duration of Phase III clinical trials?

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

What is the purpose of Phase IV clinical trials?

<p>To assess the safety and effectiveness of the drug in the general population (B)</p> Signup and view all the answers

Flashcards

Target Identification and Validation

Focuses on identifying and validating potential drug targets that are involved in the disease process.

Lead Optimization

Involves refining the chemical structure of potential drug candidates to enhance their effectiveness, safety, and drug-like properties.

High Throughput Screening (HTS)

A large-scale effort to identify and validate promising drug targets. It involves screening libraries of compounds against a specific target to identify those that show potential therapeutic activity.

Druggable Genome

The number of drug targets in the human genome that are feasible for drug discovery.

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Preclinical Development

Involves testing the safety and efficacy of preclinical candidates in animals to evaluate their potential for human use.

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Phase III

A phase of clinical trials that involves testing a drug candidate in a large group of patients with the disease to evaluate its effectiveness and safety.

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Phenotype-Based Drug Discovery

Focuses on identifying potential drug candidates based on observed effects on the disease process, rather than targeting specific molecular targets.

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Phase II

This phase is crucial in determining the optimal dose and dosing frequency of the drug for maximum efficacy and minimal adverse effects.

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Phase I

A phase of clinical trials where the drug candidate is tested in healthy volunteers to determine its safety and evaluate its pharmacokinetic properties.

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Target-Based Drug Discovery

A type of drug discovery that focuses on identifying and validating molecular targets that are associated with the disease process.

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Toxicity Testing

The process of characterizing the safety and toxicity of lead compounds in various preclinical animal models.

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Phase IV

A post-marketing surveillance phase that involves monitoring the drug's long-term effects and safety in the general population.

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Feasibility of Drug Development

Factors that contribute to the feasibility of drug development, such as economic viability, regulatory approval processes, and the potential market for the drug.

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Target Identification and Validation

The process of identifying and validating potential drug targets that are associated with the disease process.

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Phase II

A phase of clinical trials designed to determine the optimal dose of the drug and how it works in a larger group of patients.

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Preclinical Development

The process of conducting experiments in animals to evaluate the safety and efficacy of preclinical candidates.

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Phase III

A critical component of drug discovery that examines the safety and efficacy of the drug in a large number of patients with the disease.

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Target Identification and Validation

The process of identifying and validating potential drug targets that are associated with the disease process.

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Phase III

A key step in drug discovery that involves testing the drug on a larger group of patients with the disease to assess its effectiveness and safety.

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Phase II

A phase of clinical trials designed to determine the optimal dose and dosing frequency of the drug for maximum efficacy and minimal adverse effects.

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Phase I

An essential step in drug discovery that involves testing the drug on a small group of healthy volunteers to determine its safety and pharmacokinetic properties.

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Phase IV

A phase of clinical trials that involves monitoring the drug's long-term safety and effectiveness in the general population after it has been approved for use.

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