How long does it typically take to develop a drug from target identification to approval?
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>10 years
2
What are the stages of drug development?
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1. drug discovery
2. preclinical research
3. IND app
4. Clinical trials (SEAL)
3
What are the two stages of drug discovery?
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Pre-discovery: uncover disease mechanisms and possible targets
Discovery stage: search for molecules to inhibit disease mechanism
4
What is the final aim of drug discovery?
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Identify lead compounds.
5
What is the purpose of preclinical research in drug development?
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• test efficacy and safety of lead compounds
• in cell culture and small animal studies
6
What are the steps involved in preclinical research?
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• Confirm drug mechanism of action and efficacy
• construct dose-response curves
• evaluate toxicity and lethal doses
• assess carcinogenic and teratogenic effects
• confirm the best route of administration
7
What is the purpose of an IND application?
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To submit data to the FDA for review before clinical trials can start.
8
What data does an IND application include?
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• Animal efficacy data
• toxicity data
• dosing schedule
• administration protocol
• intended use
9
What is the purpose of Phase I clinical trials?
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SAFETY
• Safety testing in healthy volunteers
• 20-80 people
10
What is the purpose of Phase II clinical trials?
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EFFICACY
• small group of patients, 100s
• placebo is also given
11
What is the purpose of Phase III clinical trials?
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APPROVAL
• Efficacy and safety testing in a large group of patients
• 1000s
• can test dosages and combos with other drugs
12
What happens at the end of Phase III trials?
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Data are submitted to the FDA as a New Drug Application (NDA).
13
What is phase IV of clinical trials
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LONG TERM
• after FDA approves
• post marketing surveillance
• continue to track unidentified or delayed effects
14
What is the FDA's MedWatch system?
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A voluntary system for reporting adverse events
15
What is the United States Pharmacopeia (USP)?
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An independent, scientific, and nonprofit organization founded in 1820.
16
USP Function
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Set standards for medications and dietary supplements (like, purity, strength, manufacturing quality)
17
What is the significance of the USP standards for medications in the USA?
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Medications in the USA follow the USP standards.
18
What is the Food and Drug Administration (FDA) responsible for?
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enforce standards set by USP
19
What is the Drug Enforcement Administration (DEA) responsible for?
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enforces controlled substance laws and regulations
20
What must healthcare providers do to prescribe controlled substances?
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must be registered with the DEA and include the DEA number in each controlled substance prescription
21
How many classes does the Controlled Substances Act categorize drugs into?
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Five
22
What are Schedule I drugs?
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Highest potential for abuse and no medical use
23
examples of schedule I drugs
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heroin, LSD, and marijuana
24
What are Schedule II drugs?
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High potential for abuse leading to extreme psychological or physical dependence
25
Examples of schedule II drugs
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morphine, codeine, and fentanyl
26
What are Schedule III drugs?
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moderate to low potential for physical and psychological dependence
27
What are some examples of Schedule III drugs?
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Drugs containing less than 90 milligrams of codeine, barbiturates, and ketamine
28
What are Schedule IV drugs?
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Low potential for abuse and low risk of dependence
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examples of schedule IV drugs
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benzodiazepine sedatives, antianxiety, and antidepressants
30
what are schedule V drugs
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lowest potential for abuse; cough syrups
31
What are the types of drug categories
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• conventional
• protein based - biologics
• oligonucleotide therapies
• cell and gene therapies
32
What are conventional drugs?
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meds with defined chemical structure that are chemically synthesized
33
Types of conventional drugs
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prescription and over the counter
34
What are biologics/biosimilars?
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meds made in living cells
• hormones, therapeutic antibodies, vaccines, growth factors, via the parenteral route
35
What are antisense oligonucleotides and siRNA?
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Short chain nucleic acids that can alter gene expression, via the parenteral route.
36
What is gene therapy?
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Repairing a faulty gene via viral vectors and others.
37
What is the definition of a chemical name in drug nomenclature?
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A chemical name describes the chemical structure of a drug.
38
What is the purpose of a generic name in drug nomenclature?
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A generic name is a shortened version of the chemical name used by health professionals.
39
What is the purpose of a brand/trade name in drug nomenclature?
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A brand/trade name is given by pharmaceutical companies that make the medication.
40
What is the purpose of an Abbreviated New Drug Application (ANDA)?
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To approve a generic drug that is bioequivalent to the brand-name version.
41
What must manufacturers submit to demonstrate bioequivalence for a generic drug?
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Data showing the same active ingredient(s) with the same strength, dosage form, route of administration, indications, and absorption.
42
What is the intended action of a drug?
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A therapeutic effect
43
What are side effects?
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Mild effects that can vary in intensity among individuals.
44
What is an adverse drug reaction?
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A more severe reaction
45
What is pharmacokinetics?
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How drugs get into our body
including absorption and elimination
46
What is pharmacodynamics
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What medications do to our body
focusing on individual differences in drug response.
47
what is pharmacotherapeutics
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“Indication for drug use”
48
What is pharmacogenomics?
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how genetic variations influence drug response and optimal dose
49
What is the first pharmacokinetic phase?
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Drug Absorption
50
What is the second pharmacokinetic phase?
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Drug Distribution
51
What is the third pharmacokinetic phase?
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Drug Metabolism
52
What is the fourth pharmacokinetic phase?
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Drug Excretion
53
What are absorption and distribution in drug movement?
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movement of drug from site of admin to systemic circulation and to tissues/organisms
drug → site of administration → systemic circulation
56
What are the drug-associated factors that influence absorption?
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Chemical structure, solubility, dosage form, and formulation of the drug.
57
What are the patient-associated factors that influence absorption?
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Route of administration, blood flow to the absorption site, pH at the injection site, disease state, and the biological membranes to overcome.
58
What is bioavailability?
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- fraction of a drug that is absorbed into the systemic circulation
- available to exert its pharmacological effect
59
How is bioavailability calculated?
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AUC oral / AUC IV x 100
60
What are the factors affecting bioavailability?
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First-pass metabolism, ionization state and chemical instability, total surface area available for absorption, blood flow to the absorption site, and nature of the drug.
61
Why do very lipophilic drugs have poor bioavailability?
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Because they are insoluble in aqueous body fluids.
62
why are very hydrophilic drugs poorly absorbed
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they can not maneuver lipid-rich cell membranes
63
What is the difference between intravenous (IV) and intramuscular (IM) injections?
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IV: directly into the bloodstream
IM: inject medication into the muscle tissue
64
What are the two main categories of drug administration routes?
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Local and Systemic
65
What does the term 'Local' refer to in the context of drug administration?
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Routes that deliver drugs directly to a specific site without entering the bloodstream.
66
What are the subcategories of the 'Local' route?
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Topical, ocular, intrathecal route
67
types of enteral drugs
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via GI
oral, sublingual, rectal
68
What are the factors governing the route of choice in drug administration?
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Drug stability, pharmacokinetic profile, clinical settings, and patient's condition.
69
What does the pharmacokinetic profile include?
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Impact of metabolism (GI and Liver), required site of action, and required onset of action.
70
What are the advantages of parenteral administration?
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Direct delivery to the bloodstream, rapid onset of action, and bypasses the first-pass effect.
71
What are the disadvantages of parenteral administration?
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Painful injections, risk of infection, and potential for extravasation.
72
What are the advantages of oral administration?
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Convenient, non-invasive, and can be self-administered.
73
What are the disadvantages of oral administration?
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Potential for first-pass metabolism, slower onset of action, and variable absorption.
74
What are the advantages of sublingual/buccal administration?
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Rapid absorption, avoids first-pass metabolism, and can be self-administered.
75
What are the disadvantages of sublingual/buccal administration?
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Potential for rapid degradation, limited drug stability, and variable absorption.
76
What are parenteral drugs?
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Parenteral drugs are medications that are administered directly into the body, bypassing the digestive system.