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Midterm 2 Development Flashcards

1
Q

What is the primary objective of drug development?

A

High number of approved drugs with competitive ‘quality’ drug label claims

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2
Q

True or False: CMC is unlikely to be critical path to First Patient Dosed in any clinical study

A

True

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3
Q

What is the objective of Registration stage in Drug Development?

A

Gain regulatory approval of drug label claims

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4
Q

What is critical path in the preclinical development stage?

A

Guideline safety & tolerability studies supporting safety for IND submission

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5
Q

What general criterion does not define a project

A

No defined outcome or outcome specifications

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6
Q

True or False: BLA is submitted for regulatory review & approval of a biological drug

A

True

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7
Q

What clinical study milestone best represents the actual treatment start?

A

First Patient Dosed

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8
Q

True or False: The global pharmeceutical industry is highly regulated

A

True

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9
Q

What is the cycle time for regulatory review under ‘standard’ review conditions

A

12-18 Months

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10
Q

In the US, what regulatory action occurs before regulatory approval, or any other regulatory action?

A

FDA Advisory Board Meeting

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11
Q

How is drug development defined (start->finish)?

A

Drug candidate identified -> approved drug

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12
Q

What is a key event (milestone) in preclinical development?

A

IND submission

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13
Q

What stage is NOT considered a drug development stage?

a) Phase 2 b) Phase 3 c) Preclinical Development d) Commercialization e) Registration

A

Commercialization

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14
Q

Scientific & Technical Analysis, Commercial Analysis and Valuation are the 3 major parts determining risk & value of an R&D program

A

True

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15
Q

During what stage is the drug candidate produced at laboratory scale?

A

Lead Optimization

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16
Q

What is the definition of a pipeline in the pharmeceutical industry?

A

Totality of all R&D programs operated by a pharmeceutical company

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17
Q

CMC product development is mainly about answering the following set of key questions?

A

What’s the product and whats the process to make it? What quantities are required & with what grade? When is it needed? Where can the product be manufactured?

All of the above

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18
Q

Which Non-US Regulatory milestone (key event) is similar to Pre-IND meeting in the US?

A

Scientific Advice Meeting with EMA, Scientific Advice Metting with PDMA, Scientific Advice Meeting with Health Canada,

All of the above

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19
Q

What is made first in the manufacturing process of small (organic) molecule drug e.g. NEXAVAR?

A

Drug Substance (= Active Pharmeceutical Ingredient, API)

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20
Q

Which disease areas (clinical indications) have increasingly been using expedited regulatory pathways provided in the US?

A

Cancer indications e.g. kidney, liver, and ovarian cancer

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21
Q

The manufacturing process of RITUXAN (Rituximab), a monoclonal antibody, is more complex than the manufacturing process leading to NEXAVAR, true or false

A

True, because it is an NBE and NBE’s take longer than NCE’s to manufacture

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22
Q

The global development strategy of NEXAVAR (Sorafinib) was mainly determined by which of the following criteria?

A

Predominantly unmet medical need & expedited regulatory pathways, and clinical cancer indications best-suited to multi-kinase inhibitory drug action

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23
Q

The CMC Manufacturing concept of “Product=Process” applies only to NCE’s

A

False

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24
Q

Which US regulatory pathway to approval did NEXAVAR NOT use?

A

Standard BLA submission

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25
RITUXAN (RITUXIMAB) selectively binds the CD20 receptor of cancerous and non-cancerous B-cells
True
26
The overall global development time of NEXAVAR was reported as
62 months
27
RITUXIMAB is currently standard-of-care treatment for several hematological (blood) cancers
True
28
NEXAVAR is currently marketed as member of which cancer drug class?
Potent Multi-Kinase Inhibitor incl. RAF-1 Kinase
29
Development value chain objectives
* New Chemical Entities; New Biological Entities; First-In-Class; Best-In-Class * High # of approved drugs * Competitive 'quality' of drug label claims
30
What is the starting point in Drug Development?
'Drug-Like' Clinical drug candidate
31
What is bioavailability?
* The degree to which a drug or other [bioactive] substance becomes available to the target tissue after administration * The physiological availability of a given amount of a drug, as distinct from its potency
32
What is PK and PD?
Pharmacokinetics: The process by which a drug is Absorbed, Distributed, Metabolized, and Eliminated (ADME) by the body Pharmacodynamics: The study of how a drug acts on a living organism (duration and magnitude) of response
33
Drug Development stages
Preclinical --\> Phase 1, 2, 3 --\> Registration
34
Key milestone in Preclinical development
IND submission (Investigational New Drug Application)
35
Milestones for Phase 1
* FIH single dose * FIH multiple dose * Safety parameters * PK: Bioavailability * What drug levels needed? Biomarkers of efficacy? * Low dose --\> highest dose
36
Key milestones of Phase 2 (I)
First Patient Enrolled Top Line Results (safety, efficacy: PE, biomarkers)
37
Key milestones Phase 2 (II)
FDA EOP2 Meeting
38
Phase 3 objective
to establish clinical safety & efficacy for registration
39
Key Milestones Phase 3 (I)
First Patient Enrolled (protocol Z) Top Line Results
40
Key Milestones Phase 3 (II)
FDA Pre-NDA Meeting
41
Objective of Registration Phase
gain regulatory approval of grug label claims
42
Key Milestones of Registration
New Drug Application (NDA) Submission (FDA) Decision: Commitment-To-Launch: 1st Geography NDA Approval (US) Launch in 1st Geography
43
major parts determine risk & value of an R&D program
* Scientific & technical analysis * Commercial analysis * Valuation
44
Risks in Drug Discovery/Development
* How much risk, how many uncertainties? * What price are you willing to pay? * For What outcome, and how important is it to you?
45
Project Management is about managing the 4 P's
Product People Process Playground
46
Guideline safety & toxicology is critical path to....
IND
47
IND includes...
specific data to understand long-term safety
48
True or false: Guideline PK program is NOT critical path to IND
true
49
Primary goal of Preclinical Development is
to establish safety for First-In-Human (FIH) administration
50
What is the objective of Phase 2 in drug development?
Establish safety & efficacy in patients and demonstrate clinical proof-of-biological activity
51
What's an IND?
Investigational new drug application
52
Which event (milestone) in phase 2 of clinical development marks the start of actual patient treatment?
First Patient Dosed
53
CMC is not critical path to IND submission?
False
54
What is the objective of preclinical development?
Establish safety and tolerability in animals for first in humans
55
CMC is unlikely to be critical path in First Patient dosed in any clinical study?
True
56
Demonstration of Phase 2 Proof-of-biological concept is a major inflection point for risk/value analysis
True
57
What stage is not considered a drug development stage?
Commercialization
58
What is the objective of Phase 3 in Drug Development?
Establish safetfy and efficacy in patients for registration
59
Demonstration of an approved, commercially competitive drug label is not a major inflection point for risk/value analysis
False
60
What is the objective of Registration stage in drug development?
Gain regulatory approval of drug label claims
61
Scientific & technical analysis, Commercial analysis and valuation are the 3 major parts determining risk & value of an R&D program
true
62
What set of key regulatory milestones represent the US?
IND submission, EOP2 FDA, NDA submission
63
During what stage is the drug candidate produced at laboratory scale?
Lead Optimization
64
NDA stands for what?
New Drug Application
65
What is critical path in the preclinical development stage?
Guideline safety & tolerability studies supporting safety for IND submission
66
What does not describe project management in the Pharmaceutical industry
Make decisions which R&D programs to move forward or not
67
What combination best describes pharmaceutical R&D?
\>$800M and \<10% success and 10-15 years cycle time
68
Which kg range best reflects clinical scale manufacturing, assuming early clinical stages (FIH -\> Phase 2B) ?
10-100kg
69
What general criterion does NOT define a project
No defined outcome or outcome specifications?
70
What final drug product quality is required to enter first in human, and other phase 1, 2 and 3 clinical studies?
GMP
71
How is successful outcome of a phase 3 study defined?
Meeting the primary clinical endpoint in a statistically significant manner (p\<0.05)
72
BLA is submitted is submitted for regulatory review & approval of a biological drug
True
73
GLP grade Final Drug Product is Not required to conduct safety & toxicology studies required for IND submission
False
74
CMC product development is mainly about answering the following set of key questions
* What's the product and whats the process to make it? * What Quantities are required & with what grade? * When is it needed? * Where can the product be manufactured?
75
What is the 'End-Product' of Preclinical Development?
IND submission
76
Is achieving statistical significance in meeting the phase 3 primary clinical endpoint necessary for gaining regulatory acceptance & approval?
Yes
77
During what phase of clinical development is clinical proof-of-concept, of clinical Proof-of-Biologcial Activity established?
Phase 2B
78
What is included in the Clinical Trial Protocol, a key document in Clinical Drug Development?
Clinical objective, clinical success criteria & primary clinical endpoint, patient inclusion & exclusion criteria, safety & efficacy criteria
79
Which functional area (area of specific expertise) is not a CMC functional area?
clinical R&D
80
What is the key objective of regulatory authorities worldwide?
Ensure sufficient evidence is provided demonstrating efficacy, safety, therapeutic benefit and risk/benefit in the studied patient population
81
What are the objectives in the Registration stage of development?
Approval of drug label claims re: clinical safety; approval of drug label claims re: clinical efficacy; approval of drug label claims re: disease (therapeutic) area and indication; approval of drug label claims re: studied patient population
82
What is the cycle time for regulatory review under 'standard' review conditions
12-18 months
83
Which US regulatory meeting will review phase 3 clinical efficacy and safety data?
Pre-NDA, or Pre-BLA meeting with FDA
84
Which Non-US regulatory milestone (key event) is similar to the Pre-IND Meeting in the US?
* Scientific Advice meeting with EMA * Scientific Advice Meeting with PMDA * Scientific Advice Meeting with Health Canada
85
Which scale is mostly used in the Preclinical
pilot scale
86
Assuming successful Phase 3 and Pre-NDA Meeting outcome, which regulatory action is the most likely to occur?
Conditional approval, including defined post-approval commitments to be fulfilled by the company
87
Which expedited regulatory pathway provides 1) additional 7-year market exclusivity post approval, and 2) tax deductions for R&D expenses incurred in the US?
Orphan Drug Status
88
Which parameters are key drivers of clinical study size (patient N), costs and duration?
* Primary clinical endpoint * Study power * Statistical significance (p-value)
89
In general, major manufacturing process optimization occurs during preclinical development, and minor process improvements occur until the manufacturing process is locked-in prior to phase 3 start
True
90
Which disease areas (clinical indications) have increasingly been using expedited regulatory pathways provided in the US?
Cancer indications e.g. kidney, liver, and ovarian cancer
91

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