lecture 2 Flashcards

1
Q

how are compounds in development named

A

the company developing them gives them a name and a number code

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2
Q

how are compounds that show promise as a therapeutic drug named

A

they are names referring to the disease theyre effective against

saquinavir + ritonavir for antiviral drugs (hiv)

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3
Q

what are marked medicines

A

medicine with a proprietry (owned by someone)

they have a name that only the owner company can use

nurofen, calpol etc

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4
Q

what is a proprietry name

A

a name given by the company that owns the medicine

the name is specific to the drug formulation

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5
Q

what is generic medicine

A

a drug that isnt covered by a patent application

many companies can sell, formulate and make profit from it (paracetamol)

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6
Q

similarity between generic medicine vs marketed medicine

A

same active principle
diff trade name

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7
Q

european law on generic drugs

A

generic medicines must have a international, non-proprietary name (rINN)

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8
Q

rINN

A

international
non proprietry name

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9
Q

what is the european, international non proprietary name

A

usually the name of the drug

paracetamol, ibuprofen

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10
Q

british approved names had to be changed to what

A

the international
non proprietry
names

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11
Q

what is a drug class

A

medicine with:

similar chemical structures

same mechanism of action (binds to the same target)

used to treat similar diseases

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12
Q

what is a drug class defined by

A

a prototype drug

usually the first developed drug in that class

used as a reference to allow comparison

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13
Q

what is the ATC

A

anatomical
therapeutic
chemical
classification
system

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14
Q

how does ATC classify drugs

A

classifies the active ingredient based on the organ or system that its therapeutic, pharmacological or chemical properties act upon

aka what does this drug affect!!

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15
Q

what is the purpose of classifying drugs under ATC

A

allows us to monitor its use for research + improve quality medication use

eg: oh wow this is classified under cardiovascular system so it is probably mostly used for the heart<3

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16
Q

how many levels of ATC are there

A

5

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17
Q

what is the first level of ATC system

A

anatomical group it effects

1letter (a,b,c,d)

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18
Q

2nd level of ATC system

A

therapeutic subgroup

2 digits

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19
Q

what is therapeutics

A

treatment + healing

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20
Q

what is pharmacological

A

uses + effects

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21
Q

third level of ATC system

A

indicated the therapeutic/pharmacological subgroup

1 letter

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22
Q

forth level of ATC system

A

chemical/therapeutic/pharmacological subgroup

1 letter

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23
Q

5th level of ATC system

A

chemical substance

2 digits

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24
Q

drug definition

A

raw material from animal/vegetable origin that contains active principles that alterates the normal functioning of the CNS when introduced to the b ody via any administration

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25
drug || definition
a biologically active, chemically pure substance that has a therapeutic action. - reacts with biological environment + has the ability of curing, mitigating or preventing disease in humans/animals)
26
mitigating meaning
reducing severity of something
27
medicine definition
drug in its proper dosage form, used in medication
28
drug / medicine definition difference
drug: compound medicine: tablet: its proper dosage
29
drug target
biological entity (protein/gene) that interacts + has its activity modulated by a particular compound
30
modulated meaning
controlling somethings influence
31
druggable meaning
smt is druggable if its activity, behaviour or function, can be modulated by a therapeutic therapeutic: small molecule/ biologics
32
biological target example
nucleic acids proteins
33
7 stages from finding a drug to getting it on the market
- early stage research + discovery - preclinical studies in animals - phase 1 - phase 2 - phase 3 - fda review + approval - post approval
34
what is phase 1 clinical development
focuses on safety 20-80 healthy ppts 1-2 years
35
what is phase 2 of the clinical development
efficacy + safety 100-300 patients 1-2 years
36
what is phase 3 clinical development
efficacy + safety 1,000-3,000 patients 2-3 years
37
how long does approval from the fda last
1 - 2 years
38
how many compounds are are lost during the process
from 5,000 - 10,000 (drug discovery + preclinical) to 1 fda approved drug (fda review)
39
how much does drug discovery cost + take from the original idea to launching the finished product
1.5 billion dollars 12 - 15 years
40
what must u do before identifying a target
find an unmet medical need we must identify a disease in order to identify targets
41
who is the concept of unmet medical need important to
stakeholders and regulators
42
what is a stakeholder
rich companies that have interests in 'x'
43
what is a regulator
payers academics pharma industries health technology assessment agencies (HTA)
44
whats so important about unmet medical needs
theyre personal to everyone everyones unmet need is different should focus on all unmet needs as theyre all uncomforatble (not just unmet needs for critical or debilitating conditions)
45
whats a debilitating condition
a condition that makes someone weak or infirm
46
how and where do UMN occur
- when current therapies that are available do not address a condition - when there are no approved molecules - when there are a few molecules in the late part of clinical studies (not out yet) - existing molecules are compromised by taking the effective dose of 'x'.
47
development of new medicine is driven by what ambition
treating unmet medical needs the UMN drives ppl to want to meet these needs.
48
list of therapeutic areas
-cancer -cardiovascular diseases -diabetes -hiv , aids - immune system diseases - neurodegenerative diseases - viral diseases
49
what is a drug target
a biomolecule that is directly associated with the disease + is responsible for the cellular or molecular function
50
drug target examples
enzymes // proteins receptors metabolic pathways / apoptosis lipid membrane ion channles dna/rna
51
what is the benefit of discovering a drug target
allows researchers to optimize the drug for a particular disease or condition
52
optimise meaning
make it the best identify its most effective use
53
drug target characteristics
- confirmed role in pathophysiology of a disease/ is disease modifying - expression is not evenly distributed throughout the body - its 3d structure is availabe to assess druggability - easily assayable/tested allowing screening - has a promising toxicity profile, adverse affects can be predicted using phenotypic data - favourable intellectual property status (IP) (relavent for big pharma as it shows how its marketed and sold)
54
what is the modern drug discovery process
target identification target charactisation target validation
55
initial hypothesis in drug discovery
that a particular molecule is the key or causes a pathogenic or symptomatic mechanism in a disease. eg: hypothesising that this molecule causes the inflammation etccc.
56
target identification in drug discovery
identifying the function of a possible therapeutic target (gene/protein) and its role in the disease eg: identifying 'x' is the drug target bc iit causes the inflammation
57
in drug discovery what is target identification followed by
target characterisation of the molecular mechanism addressed by the target - u found the target, now u need to see its mechanism for how it causes the inflammation
58
target validation in drug discovery
shows that modulation of the target is likely to have a therapeutic effect
59
modulation meaning
controlling its influence
60
what is the goal in target identification
to find a biological target (protein//nucleic acid) whos activity can be regulated with a therapeutic
61
target identification techniques
in silico laboratory
62
in silico target identification technique
silico screening machine based methods experimentation performed by a computer
63
laboratory target identification technique
biological assays such as expression profiling of rna or proteins aka measuring which genes from the genome are being expressed in particular cells at any given time or functional screening: gene knockdown, knockout, overexpression
64
what is gene knockdown
reducing the genes expression
65
what is gene knockout
eliminating the gene
66
what is gene overexpression
turning certain genes on
67
what is target validation
ensuring that engagement of the target has potential therapeutic benefits aka if we do smt to the target, will it lead to a positive effect.
68
2 approaches to target validation
chemical + genetic approach
69
chemical approach to target validation
involves the use of drugs to show that inhibition of the target results in disease inhibition
70
genetic approach to target validation
target gene knockdown (reduced expression) - rna target gene knockout (gene elimination) - gene
71
target validations can be divided into what 2 groups
in vivo in vitro lab tests
72
in vivo division of target validation
use of disease models (animals) use of animal alternatives: worms, microorganisms, computer models, 3d tissue cultures.
73
in vitro division of target validation
lab tests - cell/ tissue based experiments - parameters are studied (ionic conc, enzyme activity, protein expression profiles) -study of the targets biological function when bound to different ligands (diff drug candidates)
74
what is a parameter
a measurable factor that defines a system
75
stages of early stage research + discovery
target identification hit generation lead generation
76
what is found in the hit generation
%wegotahit! finding compounds that engage with the bio target + have good properties
77
what is the lead generation in the early research and discovery stage of drug discovery
%theyretakingthelead from the possible hits analysis + improvement of compounds with pharmacological activity, the correct potency, solubility + stability
78