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Drug Discovery > Midterm 2 Development > Flashcards

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
Q

RITUXAN (RITUXIMAB) selectively binds the CD20 receptor of cancerous and non-cancerous B-cells

A

True

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26
Q

The overall global development time of NEXAVAR was reported as

A

62 months

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27
Q

RITUXIMAB is currently standard-of-care treatment for several hematological (blood) cancers

A

True

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28
Q

NEXAVAR is currently marketed as member of which cancer drug class?

A

Potent Multi-Kinase Inhibitor incl. RAF-1 Kinase

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29
Q

Development value chain objectives

A
  • New Chemical Entities; New Biological Entities; First-In-Class; Best-In-Class
  • High # of approved drugs
  • Competitive ‘quality’ of drug label claims
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30
Q

What is the starting point in Drug Development?

A

‘Drug-Like’ Clinical drug candidate

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31
Q

What is bioavailability?

A
  • 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
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32
Q

What is PK and PD?

A

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

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33
Q

Drug Development stages

A

Preclinical –> Phase 1, 2, 3 –> Registration

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34
Q

Key milestone in Preclinical development

A

IND submission (Investigational New Drug Application)

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35
Q

Milestones for Phase 1

A
  • FIH single dose
  • FIH multiple dose
  • Safety parameters
  • PK: Bioavailability
  • What drug levels needed? Biomarkers of efficacy?
  • Low dose –> highest dose
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36
Q

Key milestones of Phase 2 (I)

A

First Patient Enrolled

Top Line Results (safety, efficacy: PE, biomarkers)

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37
Q

Key milestones Phase 2 (II)

A

FDA EOP2 Meeting

38
Q

Phase 3 objective

A

to establish clinical safety & efficacy for registration

39
Q

Key Milestones Phase 3 (I)

A

First Patient Enrolled (protocol Z)

Top Line Results

40
Q

Key Milestones Phase 3 (II)

A

FDA Pre-NDA Meeting

41
Q

Objective of Registration Phase

A

gain regulatory approval of grug label claims

42
Q

Key Milestones of Registration

A

New Drug Application (NDA) Submission (FDA)

Decision: Commitment-To-Launch: 1st Geography

NDA Approval (US)

Launch in 1st Geography

43
Q

major parts determine risk & value of an R&D program

A
  • Scientific & technical analysis
  • Commercial analysis
  • Valuation
44
Q

Risks in Drug Discovery/Development

A
  • How much risk, how many uncertainties?
  • What price are you willing to pay?
  • For What outcome, and how important is it to you?
45
Q

Project Management is about managing the 4 P’s

A

Product

People

Process

Playground

46
Q

Guideline safety & toxicology is critical path to….

A

IND

47
Q

IND includes…

A

specific data to understand long-term safety

48
Q

True or false: Guideline PK program is NOT critical path to IND

A

true

49
Q

Primary goal of Preclinical Development is

A

to establish safety for First-In-Human (FIH) administration

50
Q

What is the objective of Phase 2 in drug development?

A

Establish safety & efficacy in patients and demonstrate clinical proof-of-biological activity

51
Q

What’s an IND?

A

Investigational new drug application

52
Q

Which event (milestone) in phase 2 of clinical development marks the start of actual patient treatment?

A

First Patient Dosed

53
Q

CMC is not critical path to IND submission?

A

False

54
Q

What is the objective of preclinical development?

A

Establish safety and tolerability in animals for first in humans

55
Q

CMC is unlikely to be critical path in First Patient dosed in any clinical study?

A

True

56
Q

Demonstration of Phase 2 Proof-of-biological concept is a major inflection point for risk/value analysis

A

True

57
Q

What stage is not considered a drug development stage?

A

Commercialization

58
Q

What is the objective of Phase 3 in Drug Development?

A

Establish safetfy and efficacy in patients for registration

59
Q

Demonstration of an approved, commercially competitive drug label is not a major inflection point for risk/value analysis

A

False

60
Q

What is the objective of Registration stage in drug development?

A

Gain regulatory approval of drug label claims

61
Q

Scientific & technical analysis, Commercial analysis and valuation are the 3 major parts determining risk & value of an R&D program

A

true

62
Q

What set of key regulatory milestones represent the US?

A

IND submission, EOP2 FDA, NDA submission

63
Q

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

A

Lead Optimization

64
Q

NDA stands for what?

A

New Drug Application

65
Q

What is critical path in the preclinical development stage?

A

Guideline safety & tolerability studies supporting safety for IND submission

66
Q

What does not describe project management in the Pharmaceutical industry

A

Make decisions which R&D programs to move forward or not

67
Q

What combination best describes pharmaceutical R&D?

A

>$800M and <10% success and 10-15 years cycle time

68
Q

Which kg range best reflects clinical scale manufacturing, assuming early clinical stages (FIH -> Phase 2B) ?

A

10-100kg

69
Q

What general criterion does NOT define a project

A

No defined outcome or outcome specifications?

70
Q

What final drug product quality is required to enter first in human, and other phase 1, 2 and 3 clinical studies?

A

GMP

71
Q

How is successful outcome of a phase 3 study defined?

A

Meeting the primary clinical endpoint in a statistically significant manner (p<0.05)

72
Q

BLA is submitted is submitted for regulatory review & approval of a biological drug

A

True

73
Q

GLP grade Final Drug Product is Not required to conduct safety & toxicology studies required for IND submission

A

False

74
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?
75
Q

What is the ‘End-Product’ of Preclinical Development?

A

IND submission

76
Q

Is achieving statistical significance in meeting the phase 3 primary clinical endpoint necessary for gaining regulatory acceptance & approval?

A

Yes

77
Q

During what phase of clinical development is clinical proof-of-concept, of clinical Proof-of-Biologcial Activity established?

A

Phase 2B

78
Q

What is included in the Clinical Trial Protocol, a key document in Clinical Drug Development?

A

Clinical objective, clinical success criteria & primary clinical endpoint, patient inclusion & exclusion criteria, safety & efficacy criteria

79
Q

Which functional area (area of specific expertise) is not a CMC functional area?

A

clinical R&D

80
Q

What is the key objective of regulatory authorities worldwide?

A

Ensure sufficient evidence is provided demonstrating efficacy, safety, therapeutic benefit and risk/benefit in the studied patient population

81
Q

What are the objectives in the Registration stage of development?

A

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
Q

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

A

12-18 months

83
Q

Which US regulatory meeting will review phase 3 clinical efficacy and safety data?

A

Pre-NDA, or Pre-BLA meeting with FDA

84
Q

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

A
  • Scientific Advice meeting with EMA
  • Scientific Advice Meeting with PMDA
  • Scientific Advice Meeting with Health Canada
85
Q

Which scale is mostly used in the Preclinical

A

pilot scale

86
Q

Assuming successful Phase 3 and Pre-NDA Meeting outcome, which regulatory action is the most likely to occur?

A

Conditional approval, including defined post-approval commitments to be fulfilled by the company

87
Q

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?

A

Orphan Drug Status

88
Q

Which parameters are key drivers of clinical study size (patient N), costs and duration?

A
  • Primary clinical endpoint
  • Study power
  • Statistical significance (p-value)
89
Q

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

A

True

90
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

91
Q
A

Drug Discovery (3 decks)

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