5.4.1 Drug Development

• Stages in the drug development process

  1️⃣ Drug Discovery

  2️⃣ Pre-clinical Testing

  3️⃣ Clinical Trials

  4️⃣ Regulatory Approval
• What is the purpose of target identification and validation in drug development?

  Relevance of biological targets
• Steps in target identification and validation

  1️⃣ Target Selection

  2️⃣ Target Validation

  3️⃣ Modulation Assessment
• The FDA and EMA are organizations that ensure drug safety and efficacy
• Steps in lead compound discovery and optimization

  1️⃣ Lead Compound Discovery

  2️⃣ Lead Optimization
• What is evaluated in in vitro preclinical studies?

  Toxicity, mechanism of action
• Phase 1 clinical trials are relatively short in duration, lasting weeks to months.


  True
• Regulatory approval ensures that the drug is safe and effective
• Match the steps in lead compound discovery and optimization with their descriptions:

  Lead Compound Discovery ↔️ Screening chemical libraries to identify drug candidates

  Lead Optimization ↔️ Improving compound properties through testing
• In vitro studies use cell cultures and test tubes to assess toxicity
• What is the primary purpose of Phase 1 clinical trials?

  Assess safety and tolerability
• What is the main goal of Phase 2 clinical trials?

  Assess efficacy
• Which regulatory agencies approve drugs in the United States and Europe?

  FDA and EMA
• What is the purpose of post-market surveillance?

  Detect and evaluate adverse events
• Pre-clinical testing is conducted on humans to assess safety.

  False
• Genetic and biochemical studies are used to validate the target
• What are initial drug candidates identified in lead compound discovery called?

  Lead compounds
• Lead compound discovery and optimization aims to identify promising drug candidates.


  True
• Modulation assessment evaluates the drug's mechanism of action and safety profile.

  True
• Lead compound discovery involves screening large libraries of chemical compounds to identify initial drug candidates
• In Phase 1 clinical trials, the participants are typically healthy volunteers
• Stages of the drug development process

  1️⃣ Drug Discovery

  2️⃣ Pre-clinical Testing

  3️⃣ Clinical Trials

  4️⃣ Regulatory Approval
• Lead optimization aims to improve properties such as potency, selectivity, pharmacokinetics, and safety
• Preclinical testing helps identify potential issues with drug candidates before human trials.


  True
• Preclinical testing is optional in the drug development process.

  False
• Phase 1 trials typically involve a small number of healthy volunteers
• Phase 2 trials often use a randomized, placebo-controlled design
• Regulatory approval ensures a drug is safe and effective for public use.


  True
• The drug development process involves several key stages
• Clinical trials involve three phases: safety, efficacy, and large-scale safety and efficacy
• Target identification and validation is optional for drug discovery.

  False
• Lead optimization aims to improve properties such as potency, safety, and selectivity
• What is the primary focus of Phase 1 clinical trials?

  Safety
• What are the properties of a lead compound that are improved during lead optimization?

  Potency, selectivity, pharmacokinetics, safety
• Preclinical testing aims to assess a drug's safety and efficacy before human trials.

  True
• What is the main focus of dosage in Phase 1 clinical trials?

  Determine maximum tolerated dose
• What are the primary goals of the three phases of clinical trials?

  Phase 1: safety, Phase 2: efficacy, Phase 3: large-scale efficacy and safety
• Target validation confirms the target's role in the disease process using in vitro and in vivo experiments.


  True
• What is the focus of in vivo preclinical studies?

  Efficacy, dosage, toxicity
• What are the two types of studies involved in preclinical testing?

  In vitro and in vivo