UNIT 1 Flashcards by Kat Catacutan

defined as an agent intended for use in the
diagnosis, mitigation, treatment, cure, or prevention of disease in humans or in other animals

DRUG

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may be derived from plant or animal sources, as
by-products of microbial growth, or through chemical synthesis, molecular modification, or biotechnology

New Drugs

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only difference between a drug and a
poison is the _____

Dose

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art, practice, or profession of preparing, preserving, compounding, and dispensing medical drugs (according to merriam-webster)

Pharmacy

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a facility that is licensed to dispense prescription medications and provide pharmaceutical care to patients

Pharmacy

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responsible for ensuring the safe and effective use of medications

Pharmacies

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defined as the physical form in which a
medication is produced and dispensed. This includes
various forms such as tablets, capsules, liquids, creams, ointments, and injections.
the study of this includes physical, physiochemical, and clinical discussions

Dosage Form

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defined by the US FDA (Food and Drug Administration), are instruments, machines, implants, or other similar articles that are intended for use in the diagnosis, cure, mitigation, treatment, or prevention of disease. They can also be used to affect the structure or any function of the body.

Medical Devices

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What are the Steps for Drug Development and Approval Process

Discovery and Development
Preclinical Research
Investigational New Drug (IND) Application
Clinical Trials
New Drug Application (NDA)
FDA Review and Approval
Post-Market Surveillance

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Drug Development and Approval Process

__________: Understanding the biological basis of a disease and identifying potential drug targets.
- is a key step in drug discovery,
where a biological target is identified that a drug can bind to and cause a physiological change. The target can be a protein, nucleic acid, RNA, or gene.

Discovery and Development
Target Identification

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Drug Development and Approval Process

_________: Designing and testing compounds for activity against the target.
•_________: Selecting promising compounds for further development.
- a chemical compound that shows promise as a treatment for a disease and may lead to the development of a new drug.

Discovery and Development
Drug Design and Screening
Lead Compound Identification

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Drug Development and Approval Process

Vitro and Vivo Studies

Preclinical Research

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________: Laboratory tests to assess biological activity.

In Vitro Studies

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__________: Animal testing to evaluate safety, efficacy, and pharmacokinetics

In Vivo Studies

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how the drug is absorbed, distributed, metabolized, and excreted

pharmacokinetics

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Drug Development and Approval Process

• Submission to the FDA including preclinical data, proposed clinical trial plans, and manufacturing information.
• FDA review to ensure safety for human testing.

Investigational New Drug (IND) Application

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Drug Development and Approval Process

Phase 1 - Phase 4

Clinical Trials

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Clinical Trials

Small group of healthy volunteers (20-100) to assess safety, dosage, and side effects.

Phase I

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Clinical Trials

Larger group of patients (100-300) to evaluate efficacy and side effects

Phase II

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Clinical Trials

Large-scale testing (1,000-3,000 patients) to confirm efficacy, monitor side effects, and compare with commonly used treatments.

Phase III

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Takes place AFTER the FDA has approved the treatment, and involves thousands of participants over many years. This phase aims to gather
more information about the treatment’s long-term safety and effectiveness, as well as any other benefits or side effects.

Phase 4

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Drug Development and Approval Process

• Comprehensive submission to the FDA including all data from preclinical and clinical studies, proposed labeling, and manufacturing process
• FDA review to assess the drug’s safety, efficacy, and manufacturing

New Drug Application

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Drug Discovery and Development Process

________: A team of physicians, statisticians, chemists, pharmacologists, and other experts reviews the NDA

_________: An external panel may be convened to provide recommendations.

___________: The FDA approves the drugs if it determines that the benefits outweighs the risks

FDA Review and Approval Process
Review Team
Advisory Committee
Decision

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Drug Development and Approval Process

________: Post-approval studies to gather additional information on long-term effectiveness and safety.
•_______: Monitoring of adverse effects through the FDA’s MedWatch program

Post-Market Surveillance
Phase IV Trials
Adverse Event Reporting

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Fill in the blanks: Number of patients: Length: Phase I ________ Several months Purpose: Mainly safety

20-100

Fill in the blanks: Number of patients: Length: Phase II ________ Several months to 2 years Purpose: some short-term safety but mainly effectiveness

100 to 300 (up to several hundreds)

Fill in the blanks: Number of patients: Length: Phase III ________ 1-4 years Purpose: Safety, Effectiveness, dosage

1,000-3,000 (Several hundreds to several thousands)

Sources of New Drugs

1. Natural Products 2. Biotechnology and Genetic Engineering 3. Peptides and Proteins 4. Small Molecule Drugs 5. Nanotechnology 6. Regenerative Medicine 7. Microbiome-Based Therapies 8. RNA-Based Therapies

Sources of New Drugs: ______: Marine environments are a rich source of novel compounds. For example, Trabectedin (Yondelis) is derived from the sea squirt Ecteinascidia turbinata and is used in cancer treatment. •______: Many drugs are derived from plants. For instance, Artemisinin from the sweet wormwood plant (Artemisia annua) is used to treat malaria. •_______: Antibiotics like Penicillin, derived from the mold Penicillium notatum, and Streptomycin, from the bacterium Streptomyces griseus, are classic examples.

Natural Products Marine Organisms Plants Microorganisms

Sources of New Drugs •________: These are engineered to target specific cells. An example is Adalimumab (Humira), used to treat autoimmune diseases like rheumatoid arthritis. •________: This involves modifying or manipulating genes to treat diseases. For example, Luxturna is a gene therapy for a specific type of inherited vision loss. •________: Human insulin (e.g., Humulin) produced by recombinant DNA technology is a well-known example.

Biotechnology and Genetic Engineering Monoclonal Antibodies Gene Therapy Recombinant Proteins

Sources of New Drugs •__________: Peptides are increasingly being developed for therapeutic use. Exenatide (Byetta), derived from the saliva of the Gila monster, is used to treat type 2 diabetes. •__________: Enzymes and other proteins are being used as drugs, such as Dornase alfa (Pulmozyme), used in cystic fibrosis treatment to break down mucus.

Peptides and Proteins Peptide-Based Drugs Protein Therapeutics

Sources of New Drugs • ___________: Advances in _________ allow for the creation of new small molecule drugs. For example, Sofosbuvir (Sovaldi) is a synthetic antiviral drug used to treat hepatitis C.

Small Molecule Drugs Synthetic Chemistry

Sources of New Drugs _________: These can be used for targeted drug delivery. Doxil is a liposomal formulation of doxorubicin used in cancer treatment, designed to reduce toxicity and improve efficacy. •________: Structures like micelles and dendrimers are being explored to improve drug solubility and delivery.

Nanotechnology Nanoparticles Nanocarriers

Sources of New Drugs ______: These are being investigated for their potential to regenerate damaged tissues. Prochymal, an adult stem cell therapy, is used for treating graft-versus-host disease •______: Combining cells, scaffolds, and biologically active molecules to repair or replace damaged tissues, such as in the development of bioengineered skin

Regenerative Medicine Stem Cells Tissue Engineering

Sources of New Drugs __________: These are used to modulate the gut microbiome for therapeutic purposes. Fecal microbiota transplantation (FMT) is used to treat recurrent Clostridium difficile infections •__________: Viruses that target bacteria, known as bacteriophages, are being explored as alternatives to antibiotics. For example, phase therapy is being investigated for treating antibiotic-resistant infections

Microbiome-Based Therapies Probiotics and Prebiotics Bacteriophages

Sources of New Drugs: •____________: This technology can silence specific genes involved in disease. Patisiran (Onpattro) is an RNAi therapeutic for hereditary transthyretin-mediated amyloidosis. •_____________: The COVID-19 vaccines developed by Pfizer-BioNTech and Moderna use mRNA technology to elicit an immune response against the virus

RNA-Based Therapies RNA Interference (RNAi) mRNA Vaccines

Methods of Drug Discovery

Traditional Methods Modern Methods Biotechnology-Based Methods Phenotypic Screening Network Pharmacology

Traditional Methods

Natural Product Screening Serendipity

Methods of Drug Discovery Traditional Methods • Description: Involves extracting and testing compounds from natural sources such as plants, microorganisms, and marine organisms. • Advantages: Rich source of bioactive compounds, many drugs have been successfully derived this way (e.g., penicillin, paclitaxel). • Limitations: Time-consuming, complex extraction and purification processes, variability in natural sources.

Natural Products Screening

Methods of Drug Discovery Traditional Methods Description: Involves the unexpected discovery of drugs, often while researching something else. • Advantages: Can lead to breakthrough medications (e.g.,penicillin, sildenafil). • Limitations: Unpredictable, relies heavily on chance and observation.

Serendipity (Accidental Discovery)

Methods of Drug Discovery Modern Methods

High-Throughput Screening (HTS) Structure-Based Drug Design (SBDD) Ligand-Based Drug Design (LBDD) Computational Drug Discovery

Methods of Drug Discovery Modern Methods • Description: Uses automated equipment to rapidly test thousands of compounds for biological activity against a target. • Advantages: Efficient, can screen large libraries of compounds quickly, high potential for discovering novel drug candidates. • Limitations: Requires substantial investment in technology and infrastructure, high rate of false positives/negatives

High-Throughput Screening (HTS)

Methods of Drug Discovery Modern Methods • Description: Involves designing drugs based on the 3D structure of the target protein, often determined by X-ray crystallography or NMR spectroscopy. • Advantages: Rational design process, can optimize binding affinity and specificity, reduces trial and error. • Limitations: Requires detailed structural information, not all targets have known structures

Structure-Based Drug Design (SBDD)

Methods of Drug Discovery Modern Methods •Description: Uses knowledge of molecules that bind to the target to design new compounds. Often employs computational methods to identify key features. •Advantages: Useful when the target structure is unknown, leverages existing ligand information. •Limitations: Dependent on the quality and quantity of known ligands, can be less precise than SBDD

Ligand-Based Drug Design (LBDD)

Methods of Drug Discovery Modern Methods •Description: Utilizes computer algorithms and models to predict the interaction between drugs and targets, often through virtual screening. •Advantages: Cost-effective, can screen vast compound libraries, speeds up the initial phases of drug discovery. •Limitations: Models may not accurately predict real-world interactions, dependent on the quality of input data.

Computational Drug Discovery

Methods of Drug Discovery Biotechnology-Based Methods

Monoclonal Antibody Technology Gene Therapy and RNA-Based Approaches

Methods of Drug Discovery Biotechnology-Based Methods • Description: Involves creating antibodies that specifically target antigens on diseased cells. • Advantages: High specificity, can target previously "undruggable" targets, successful in treating cancers and autoimmune diseases. • Limitations: Expensive production process, potential for immunogenicity.

Monoclonal Antibody Technology

Methods of Drug Discovery Biotechnology-Based Methods • Description: Involves modifying genetic material to treat diseases. Includes techniques like RNA interference (RNAi) and CRISPR gene editing. • Advantages: Can target genetic causes of disease, potential for long-lasting effects, highly specific. • Limitations: Delivery challenges, potential off-target effects, ethical and regulatory concerns.

Gene Therapy and RNA-Based Approaches

Methods of Drug Discovery • Description: Involves screening compounds based on their observable effects on cells, tissues, or whole organisms, without prior knowledge of the specific target. • Advantages: Can discover drugs with novel mechanisms of action, useful for complex diseases. • Limitations: Identifying the exact mechanism can be challenging, may require extensive follow-up studies.

Phenotypic Screening

Methods of Drug Discovery • Description: Examines the complex interactions between drugs and multiple targets within biological networks. • Advantages: Can provide insights into multi-target drugs, helps in understanding polypharmacology, useful for complex diseases. • Limitations: Computationally intensive, requires comprehensive biological network data.

Network Pharmacology

Organizational Structure Required for cGMP

Quality Control Unit (QCU) Production Department Engineering and Maintenance Research and Development (R&D) Regulatory Affairs

This is a critical component within any pharmaceutical manufacturing organization. ____ is responsible for the oversight and enforcement of cGMP regulations, ensuring that all procedures are followed and that products meet quality standards.

Quality Control Unit (QCU)

Responsible for the actual manufacturing processes, including formulation, processing, packaging, and labeling.

Production Department

Ensures that all manufacturing equipment and facilities are properly maintained and function correctly.

Engineering and Maintenance

Conducts studies to develop new products and improve existing ones, ensuring that they comply with cGMP standards.

Research and Development (R&D)

Ensures that the company complies with all regulatory requirements and handles submissions to the FDA and other regulatory bodies.

Regulatory Affairs

• Standardization: Written cGMP guidelines ensure that manufacturing processes are standardized. This consistency helps in producing pharmaceutical products that meet predetermined quality criteria every time. • Reproducibility: Clear, documented procedures allow for reproducibility of processes, which is crucial in maintaining product quality across different batches.

Consistency in Manufacturing Processes

•_________: Compliance with written cGMP guidelines is mandatory for regulatory approval. The FDA and other regulatory bodies require detailed documentation of manufacturing practices to ensure products are safe and effective. •_________: Written cGMP records provide the necessary documentation for regulatory audits and inspections, demonstrating adherence to legal and regulatory standards.

Compliance with Regulatory Requirements Regulatory Adherence Audit and Inspection

•___________: Documented procedures reduce the likelihood of errors by providing clear instructions for each step of the manufacturing process. •___________: Written cGMP guidelines include protocols for handling deviations and non-conformances, ensuring that any issues are promptly addressed and rectified.

Quality Assurance and Control Error Reduction Deviation Management

•_________: Written procedures serve as a training tool for new employees, ensuring they understand and follow the correct methods. •_________: Clear documentation assigns responsibility and accountability to specific individuals or teams, promoting a culture of quality and responsibility.

Training and Accountability Employee Training Accountability

________: Written cGMP guidelines help identify and mitigate risks associated with the manufacturing process, ensuring product safety. •________: Consistent adherence to written procedures ensures that each product meets its intended quality standards providing effective and reliable medications to patients

Product Safety and Efficacy Risk Management Consistency in Quality

•__________: Documented processes can be reviewed and analyzed for continuous improvement. This helps in refining manufacturing practices and enhancing product quality over time. •__________: Written records allow for the collection and analysis of data, which can be used to make informed decisions about process improvements.

Continuous Improvement Process Improvement Feedback Loop

•__________: Written cGMP guidelines provide a record of compliance with regulatory requirements, which can be crucial in legal matters or disputes. •__________: Detailed records ensure that every step of the manufacturing process is traceable, which is essential for investigations in the event of product recalls or quality issues.

Legal Protection Documentation of Compliance Traceability

Written cGMP guidelines provide a structured framework for ensuring the quality and safety of pharmaceutical products.

1. Consistency in Manufacturing Processes 2. Compliance with Regulatory Requirements 3. Quality Assurance and Control 4. Training and Accountability 5. Product Safety and Efficacy 6. Continuous Improvement 7. Legal Protection

Various Types of Tamper-Evident Packaging

Shrink Bands Tamper-evident tape Tamper-evident labels Flexible pouches Breakable caps and seals Blister packs Foil seals Film wrapper Bubble pack Aerosol Container

Sealed tubes, specifically _____, are tampered resistant, since it has sealed bottoms

Plastic tubes

Made of PVC plastic, ______ can wrap around the neck, cap, or cover of a container. If the product is tampered with or damaged, the ______ will show that it has been compromised. Perforated _____can improve the user experience.

Shrink Bands

A strip of material with a perforated edge that's applied to the outside of a package. It can be easily torn off to reveal if the package has been opened. _______ is often used to secure large items like cartons, crates, snd boxes during shipping.

Tamper-evident tape

These labels can tear or leave a residue if someone tries to open the package, indicating that the product may have been compromised. Hershey's is one company that uses _____ on some of its packaging.

tamper-evident labels

A popular choice in the food industry, _______can often be resealed so that users can continue to use the package until all the product has been consumed.

Flexible pouches

These______ break or show visible evidence when opened

Breakable caps and seals

encase individual doses in a plastic or foil material, which must be punctured or peeled away to access the product.

Blister packs

These seals are usually found under caps and provide a barrier that must be peeled or punctured to access the product.

Foil Seals

Scale and Volume Large-Scale Production: Medications are produced in large quantities to meet the needs of a wide patient population. Batch Consistency: Ensures uniformity and consistency across all produced units within a batch.

Pharmaceutical Manufacturing

Scale and Volume Small-Scale Production: Medications are prepared in small quantities, often on a per- patient basis. Customization: Tailored to the specific needs of an individual patient, often when commercially available drugs are unsuitable.

Extemporaneous Compounding

Regulatory Oversight: Regulated by Agencies: Subject to strict regulations and oversight by agencies such as the FDA (Food and Drug Administration) in the U.S. and EMA (European Medicines Agency) in Europe. cGMP Compliance: Must comply with current Good Manufacturing Practices (cGMP) to ensure quality, safety, and efficacy.

Pharmaceutical Manufacturing

Regulatory Oversight Less Stringent Regulation: While still regulated, compounding pharmacies have different and often less stringent oversight compared to manufacturing facilities. USP <795> and <797>: Must comply with standards set by the United States Pharmacopeia (USP) for non- sterile and sterile compounding, respectively.

Extemporaneous Compounding

Product Formulations Standardized Formulations: Medications are produced with standardized dosages and formulations to ensure consistency.

Pharmaceutical Manufacturing

Product Formulations: Customized Formulations: Dosages and formulations can be customized based on patient- specific needs, such as allergies, age, or specific medical conditions.

Extemporaneous Compounding

Quality Control testing and quality control measures are implemented to ensure each batch meets predefined standards. Documentation and Traceability: Detailed documentation for every step of the manufacturing process for traceability and accountability

Pharmaceutical Manufacturing

Quality Control Pharmacist’s Expertise: Quality relies heavily on the skill and expertise of the compounding pharmacist. Limited Testing: Quality testing is more limited compared to large-scale manufacturing.

Extemporaneous Compounding

Manual Processes/Automation and Technology Automation and Technology: Advanced Technology: Utilizes advanced machinery and technology for efficient production. Automation: Many processes are automated to increase efficiency and reduce human error.

Pharmaceutical Manufacturing

Manual Processes/Automation and Technology: Manual Processes Manual Preparation: Medications are often prepared manually or with minimal automation. Individualized Care: Focuses on providing personalized care rather than mass production.

Extemporaneous Compounding

•____________: This technology allows the rapid testing of thousands of synthetic compounds to identify potential drugs.

High-Throughput Screening