Drug Discovery Process Flashcards
1
Q
Is it better to provide distilled sterile water or normal saline solution (sodium chloride NaCl- 0.90%) for people to do drugs?
A
We would recommend water as we don’t know what drug they will be taking and both are safer then dirty water, the cost of normal distilled sterile water is cheaper so we can affect the biggest population
2
Q
What is disease
A
A condition which interferes with an organisms normal state (baseline)
3
Q
Aetiology of disease
A
- The science of causes of diseases
- Can be caused by genetic or environmental factors which causes dysfunction
- Dysfunction causes diagnostic signs, symptoms and disabilities or prognosis
4
Q
What is a drug
A
An agent intended for use in diagnosis, treatment, cure or prevention of disease in humans or animals
5
Q
What happens if a drug is given to a healthy individual?
A
It will cause an effect in the body which alters biological functions
Every drug has symptoms whether they are noticeable or not
6
Q
What is a lead compound?
A
It is the prototype chemical compound which has a desired biological or pharmacological activity
It is not the complete drug and doesn’t possess all desired features of a drug
7
Q
What is a prodrug?
A
A compound that needs metabolic biotransformation after being administered to be active within the body
8
Q
Why use Prodrugs
A
- For Solubility, site specificity (targeting), permeability, extended effect, reduced toxicity, stability on shelf
9
Q
Dosage form
A
- The types of preparations in which the drug is presented to the patient
- Drug delivery systems (pill, liquid…)
10
Q
Rationale for different dosage forms?
A
- Safe and convenient means of administering an accurate dose of a drug (important for potent drugs requiring very low doses)
- To protect the drug against chemical decomposition (both external such as atmospheric oxygen and humidity, and internal such as gastric acid)
- To conceal unpleasant odor or taste
- Control the release of the drug
11
Q
What is needed to know for prep and formulation of a drug
A
- Active ingredient- drug
- Non- medicinal additives/ excipients (substances added that are not for therapeutic action but rather aid the manufacturing process)
- Dosage form
- Method of preparation
12
Q
What is the order of drug naming
A
- When discovered initially represented by empirical formula (NaCl)
- The “ new compound will then have a chemical name (heptane-2- carboxylic acid)
- When the new chemical entity shows sufficient promise as a drug –> non-proprietary name is proposed
- When a drug product is developed and marketed, a proprietary name is given (trademark: only the inventor can use this name such as tylenol)
13
Q
What is a proprietary name
A
- Brand name
A trademark officially registered by a company
14
Q
What is a Non proprietary name
A
- Generic name
The generally recognized or common name for a drug
15
Q
Who is Standardized nomenclature established by
A
- United states adopted names (USAN) council
World health organization (WHO)
16
Q
Example of a drug product Tylenol
A
- Drug: acetaminophen (generic name)
- Dosage form: tablet
Drug product: tylenol tablet (brand name)
- Dosage form: tablet
17
Q
What is Pharmaceutics definition
A
The study of the physical, chemical and biological factors which influence the stability and effectiveness of pharmaceutical products and which should be considered in their formular and manufacture
18
Q
What is Pharmaceutics concerned with 4 things
A
- *formulation
- Manufacture
- Stability
Effectiveness
19
Q
What is biopharmaceutics (Definition)
A
- The study of the properties of the drug and dosage form after administration into the body
E.g: pharmacokinetics (ADME)
20
Q
What are the 4 parts of pharmacokinetics
A
- Absorption
- Distribution
- Metabolism
Excretion
21
Q
What is pharmacokinetics concerned with
A
What does the body do to the drug
22
Q
What is pharmacodynamics concerned with
A
What does the drug do to the body
23
Q
What is the difference between pH value and pKa value?
A
- pH is related to the acidity or basic properties of a compound
pKa determines if the compound is protonate or unprotonated ( it is unprotonated if the pH is above the pKa
24
Q
in relation to pka is deprotonated or protonated above pH
A
Deprotonated: Above pKa
Protonated: Below pKa
25
What is the Henderson-Hasselbalch equation
Equation used to calculate pKa
26
Explain Hydrogen bonding
- A type of dipole-dipole attraction and is not covalent
- Attractive force between a hydrogen atom covalently bonded to a very electronegative atom such as N, O, or F and another very electronegative atom
- Weaker then an ionic or covalent bond but stronger then van der waal forces (attraction force between atoms or non polar molecules caused by a temporary change in dipole moment, this creates a similar shift in nearing atoms or molecules)
27
What is a new drug
- Any drug that has not been generally recognized as safe and effective yet
- Need to be new chemical entity
- A change in previously approved drug products formulation or manufacture
28
What are the Regulatory issues (USA and Canada)
- USA: food, drug and cosmetic act
Food and drug administration (FDA)
New drug application (NDA)
- Canada: Food and drug act
Health Canada
New drug submission (NDS)
29
How long is the drug discovery process?
10-12 year
30
Out of 10,000 compounds in pre clinical testing how many enter human testing, how many are approved
- 5 testing
- 1 approved
31
30. Organization of drug discovery process
- Pre-discovery--> drug discovery--> pre clinical--> IND submitted (CTA, canada)--> clinical trials (phase1, phase 2, phase 3)--> NDA submitted (NDS canada)--> FDA review--> scale up to MFG--> post marketing surveillance
32
Explain Phase 1,2,3 trials
- Human testing
- Phase 1: small group of healthy individuals (for safety and pharmacokinetics
- Phase 2: patients with the disease 100-500 ( efficacy of drug and right dosing)
- Phase 3: large amounts of patients (safe and effective for wide variety of people
33
When does the formulation of the drug take place
In general it is in the pre clinical trials
34
what is IND (CTA)
Investigational new drug: the sponsor of the new drug will show all testing and why it should be tested in humans
Clinical trial application: Canadian version
35
What is NDA (NDS)
New drug application
Takes almost a year of reviewing
NDS is Canadian version: new drug submition
36
What is CADTH
makes recommendations to the provincial formulary decisions to allow for specific drugs to be covered
37
What is PMPRB
sets a reasonable price that companys can charge
38
What is SAP
Special access program
- if a drug is in human testing a physician can make a special request for their patient to take this specific drug if they are resistant to other treatments
39
Why is the number of months to approve new "traditional drug" is 18 months while for biologics is 25 months
- The complexity of biologics means there is fewer experts within the field
-Biologicals is anything that is biologically driven
40
What happens if the new drug is designed to treat life threatening illness
- There is a fast track program (priority) for approval
41
What happens when a drug goes to the market
- It is continually is monitored
- We still don’t know everything about its safety
-Can still be removed from the market
42
What is post marketing, why is it done
- Phase IV clinical studies
- They may need to revise labels ( adverse reaction reporting)
- Product defect reporting
- The company is looking for ways to extend there product line: which is modified versions of existing drugs as it is cheaper and can extend the patent
43
When does a company usually file a patent for a new drug
pre clinical stage
44
What are patent Protection Strategies
- New clinically superior formulations, weekly rather then daily…
- New routes of administration
-New uses of the drug (chiral switches)
45
what are innovator products
- A brand name of product discovered/developed by an innovator company for the for the first time
- New active ingredients
- Patent protection
-Company's sole right to sell the product
46
what is a generic name, how is it gotten
- A copy of the brand name pharmaceutical product
-Identical (bioequivalent) to the brand name drug
- Can happen when patents or other exclusivity expire
- Abbreviated new drug application (ANDA)
-Lower costs (as there is no preclinical/ clinical trials)
47
What is bioequivalence of drug products, what is not important within
- They are pharmaceutical equivalents (alternative)
-Rate and extent of absorption does not show significant differences
48
How is bioequivalence of drug products shown
- Lab testing
- Simple cross study (only 12-24 healthy subjects
-Patient testing
49
How many years will the drug dominate market the market without competition
7-12 years, patent is in preclinical phase and it still takes time to get it to market
50
Use of post marketing clinical studies
For the fda/ health Canada adverse reaction reporting (revise labeling), Product defect reporting, company looks for new uses
51
How many years will the drug dominate market the market without competition
7-12 years, patent is in preclinical phase and it still takes time to get it to market
52
When does the formulation work take place during the drug development process
preclinical
53
How can a patient access a drug that is not approved yet
Emergency release through special access program
54
Name patent extension strategies?
-anything that changes efficacy/ safety.
-New formulation( daily to weekly),
-route,
-new uses (different indication)
-chiral switching
55
When the generic name is given to a drug? Vs when a brand name is given
-Generic name is given preclinical, brand name is given during new drug application
-when the new chemical entity shows sufficient promise it is gets a non proprietary name,
-when the drug is developed and marketed it is given a proprietary name (brand)
56
Do we look at pharmacokinetics or pharmacodynamics first in a new drug
Pharmacokinetics: what the body does to the drug
57
What is the difference between pharmaceutics and biopharmaceutics
-Pharmaceutics is concerned with the production of a drug product (formulation, manufacturing)
-biopharmaceutics is concerned with the interaction between the drug product and the body
58
How long does it take for the patent to expire and can a specific patent be extended
20 years and no it cant
59
During IND process, FDA may approve the drug for clinical trails despite deficiencies
Not serious enough to justify delays, CDER informs the sponsor that it can proceed but additional info is needed
60
What is a prodrug and why is it used
-Drug that is activated from metabolism,
-used for permeability, site specificity, solubility, extended, rerelease, reduce toxicity, stability
61
Use of Bioequivalence studies
To ensure generic is equal to brand
62
Which route of drug administration is desired when developing new drug product
Oral dosage form, easiest way to take a drug
63
What is CTA
Clinical trial application(Canadian, IND is American)
64
What is a dosage form
The types of preparations in which the drug is presented to the patient,
-drug delivery system
65
The uses of different dosage forms
-safe and convent
-to protect the drug -
to conceal unpleasant taste odor,
-control the release
66
What is the CDER
Center for drug evaluation and research, reviews the IND
