Drug Discovery and Development Overview

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Questions and Answers

What is the primary focus of Phase 2 clinical trials?

Dosage optimization

Cost-effectiveness of the drug

Effectiveness and short-term side effects (correct)

Long-term side effects

What is the FDA's time frame to decide whether to file a New Drug Application (NDA)?

30 days

90 days

120 days

60 days (correct)

What is included in a New Drug Application (NDA)?

Patient testimonials

Animal and human data, analyses, and manufacturing information (correct)

Only human trial data

Marketing strategy and sales projections

What is the purpose of the FDA's facility inspection during the drug approval process?

To ensure the manufacturing facility meets safety regulations Signup and view all the answers

What distinguishes Adverse Drug Reactions (ADRs) from side effects?

ADRs are moderate to severe and unpredictable Signup and view all the answers

Which of the following is a requirement after a drug has been approved for marketing?

Periodic safety updates Signup and view all the answers

What is the typical review goal for priority drugs by the FDA?

Six months Signup and view all the answers

Which action can the FDA take if significant adverse events are confirmed post-marketing?

Change directions for product use or provide warnings Signup and view all the answers

What is the primary purpose of post-marketing requirements (PMRs)?

To conduct sponsor-required studies mandated by law Signup and view all the answers

Which statement accurately describes post-marketing commitments (PMCs)?

PMCs are optional studies that sponsors agree to conduct voluntarily. Signup and view all the answers

What factor primarily influences the cost of drug development?

Contract studies in clinical and preclinical research Signup and view all the answers

What is the typical timeframe for developing a new pharmaceutical agent?

8 to 15 years Signup and view all the answers

Which type of drug is defined as an identical copy in active ingredients, formulation, and strength produced by another company?

Generic drug Signup and view all the answers

The FDA's standard for non-prescription drugs is a reflection of what?

An equal standard of safety and efficacy as prescription drugs Signup and view all the answers

What is the typical patent duration granted to an innovator for newly approved drugs?

20 years Signup and view all the answers

How has the human genome project impacted drug discovery processes?

By altering the way drugs are discovered and developed Signup and view all the answers

What is the primary focus during Phase 1 studies of drug clinical trials?

Ensuring safety of the drug Signup and view all the answers

Which document must be filed with the FDA before a company can begin testing a drug in humans?

Investigational New Drug (IND) Application Signup and view all the answers

What information is NOT typically included in the IND application?

Global marketing strategy Signup and view all the answers

What triggers a clinical hold issued by the FDA?

Identified risks to study subjects Signup and view all the answers

Which of the following compounds is NOT typically used as an active ingredient in drug development?

Heavy metals Signup and view all the answers

What is a key factor that influences the cost and timeline of drug development?

The complexity of the clinical trials involved Signup and view all the answers

During which phase are acute and sub-acute toxicities typically determined?

Pre-clinical phase Signup and view all the answers

Why is post-marketing surveillance crucial in drug approval?

To detect any adverse effects not identified in earlier trials Signup and view all the answers

Study Notes

Drug Discovery

Drugs are extracted from various sources, including plants, mammals (hormones and microorganisms), and semisynthetic/synthetic compounds.
Plant poisons, like morphine from opium poppies and digitalis from foxgloves, are sources.
Inorganic chemicals, such as potassium, sodium, chloride, and calcium, are also used.
Biologics, including insulin, estrogen hormones, epinephrine, and diphtheria antitoxin, are used.
Drugs from the sea, like algae, invertebrates, and sponges, are also a source.

Drug Research & Development Team

A team of scientists from diverse disciplines, such as pharmaceutical chemistry, pharmaceutics, clinical toxicology, clinical pharmacology, biopharmaceutics, and clinical pharmacokinetics, work together.

FDA Drug Approval Process

Drug Discovery and Development: Takes 2-10 years.
Pre-clinical Research and Development: Includes initial substance synthesis, lab studies, and Institutional Review Board (IRB) meetings. Takes 3-6 years.
Clinical Trials:
- Consists of Phase 1, 2, and 3 clinical trials.
- Phase 1: Healthy volunteers (20-80). Focuses on safety.
- Phase 2: Patients with target disease. Few dozens to few hundreds of patients. Focuses on effectiveness and short-term side effects.
- Phase 3: Larger scale trials. Hundreds to thousands of patients. More populations, different dosages, and combinations. Focuses on safety, effectiveness, and combination therapies.
FDA Drug Application
- IND (Investigational New Drug): submitted after pre-clinical trials. Includes clinical protocol (IRB Approved). Introductory and investigative plan. Chemistry, Manufacturing, and Control (CMC) information. Animal pharmacology and toxicology studies data.
- NDA (New Drug Application): submitted after clinical trials. Includes animal and human data, data analysis, drug behaviour, and manufacturing details.
FDA Review:
- FDA has 30 days to review IND. If no response within 30 days, sponsor can begin clinical trials.
- FDA reviews NDA, reviews for 60 days, may refuse incomplete application.
- FDA decides whether it is safe for human testing.
- May issue a clinical hold (when risk to subjects identified).
FDA Approval and Manufacturing:
Post-Market Surveillance: Takes 8-9 months, FDA reviews the NDA, reviews for 60 days, may refuse incomplete application
- FDA performs facility inspections.
- FDA reviews drug labels for appropriate information.
- FDA approves, or issues complete response letter.
- Post-market surveillance begins.
- Drug sponsors must submit periodic safety updates to the FDA.

Drug Manufacturing

Once FDA approves NDA, companies can market and produce the drug.
Newly Approved Drug (Patent Drug): Extremely novel product developed by the company holding patent for 20 years to market.
Generic Drug: Identical copy (ingredients, formulation, strength), manufactured by companies other than the innovator.
Non-Prescription Drug: FDA applies same standards to non-prescription drugs.

Future of Drug Discovery

Human Genome Project: Changed drug discovery and development methods dramatically. Aims to decipher the human genome and identify and characterize genes.
Surrogate Markers: Laboratory measurements or physical signs that predict long-term treatment results.
Metabolomics: Study of small molecules (metabolites).
New Methods of Drug Discovery:
- Rational drug design: Using known small molecules with tailored properties to target known biomolecules (proteins or nucleic acids).
- Powerful computers: Computer simulations to aid in solving complex chemistry problems.
- Computational chemistry: Theoretical chemical methods to calculate molecular interactions and properties using efficient computer programs.
- X-ray crystallography: Determining atomic and molecular structures of crystals.
- Nuclear magnetic resonance (NMR) spectroscopy: Characterization technique for determining molecular structure at atomic level.
- 3-Dimensional Structure-Activity Relationship (SAR): Correlations between chemical structure, biological function, and target activity using diverse molecular structures.

Time and Cost of Drug Development

Developing a new pharmaceutical drug usually takes 8-15 years (some take longer, some faster).
Clinical and preclinical contract studies, like toxicology studies and analytical sample costs, drive up development costs.

Timeline of Drug Development

Key milestones and estimated timelines for drug discovery and development (Target Selection, Discovery and Screening, Lead Selection, Lead Optimization, etc.)

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Description

This quiz covers the extensive process of drug discovery, highlighting sources of drugs and the multidisciplinary nature of drug research and development teams. Explore the stages involved, from initial extraction to the FDA drug approval process, and understand the collaboration between various scientific fields. Test your knowledge on the timeline and critical aspects of bringing a drug to market.