Drug Discovery and Clinical Trials Flashcards

1
Q

Drug Discovery

A
  • Disease characterization
  • Target selection & identification of “drug hits”
  • Lead optimization
  • Pharmacological profiling
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2
Q

Pre-Clinical Development

A

In vitro and in vivo Animal models

  • Pharmacokinetics & Toxicology
  • Formulation & Synthesis scale up
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3
Q

Clinical Development

A

Drug tested in human volunteers & patients

  • Safety & Efficacy
  • Pharmacokinetics & Toxicity
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4
Q

Compound-centered approach to Drug Discovery

A

Compound with interesting activity and/or chemistry

Screen biological systems for interesting biological functional effects

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5
Q

Target-centered approach to Drug Discovery

A

Identify protein target with known disease association/activity

Screen large chemical libraries to Identify drug hits that interact with target and modify activity

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6
Q

Lead Optimization

A

Identified new drug lead may not have best properties for a drug. Develop chemically modified drug variants.

Screen for improved pharmacological profile.

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7
Q

What is the goal of pre-clinical development?

A

To provide evidence that a drug is safe for future testing in humans

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8
Q

What is safety pharmacology?

A

Evaluate drug using in vivo animal testing for the presence of any obvious acute systemic toxic effects
- two different species and two routes of administration

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9
Q

No-effect dose

A

The maximum dose at which toxic effects are not seen

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10
Q

LD50

A

The dose that kills 50% of animals

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11
Q

What are the major steps in preclinical development?

A

A. Safety Pharmacology

B. Toxicology

C. Pharmacokinetic testing
- fully characterize pharmacokinetic parameters: ADME

D. Drug Interaction studies

  • determine metabolism by CYP450 family members - identify any possible inhibitors of CYP450
  • determine specificity for Drug Transporter proteins

E. Chemical & Pharmaceutical Development

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12
Q

Investigational New Drug Application (IND)

A

Vehicle for providing evidence to the FDA that a new drug drug is a viable candidate for further development and appropriate for initial limited use in humans (i.e. “reasonably safe”)

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13
Q

What are the components of the IND?

A
  1. Animal Pharmacology and Toxicology data
  2. Manufacturing information
  3. Clinical Protocols and Investigator information
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14
Q

Investigator IND

A

Request to study an unapproved drug

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15
Q

Emergency use IND

A

Allows authorization of an experimental drug in an emergency situation for use in a single patient that has a serious or immediately life-threatening condition

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16
Q

Treatment IND

A

Allows promising experimental drugs that have not yet been approved to be used in patients with serious or immediately life-threatening conditions where no other therapy is available and death is likely

17
Q

Institutional Review Board

A

Minimize potential risk to human subjects

Determine whether potential risk is reasonable relative to anticipated benefit

18
Q

Superiority Trial

A

Trial designed to demonstrate that one treatment is clinically superior to either placebo or another drug

19
Q

Non-inferiority Trial

A

A trial designed to demonstrate that one drug is not appreciably less effective than the standard

20
Q

Phase I Clinical Trial

A

20- 100 Healthy Volunteers

Is it safe? Tolerability PK

21
Q

Phase II Clinical Trial

A

100-200 Patients
Does it work in patients?
Dosing

22
Q

Phase III Clinical Trial

A

1,000-6,000 Patients

Does it work in large patient populations

23
Q

Phase IV Clinical Trial

A

Post- marketing Surveillance
Adverse effects
Interactions Compliance

24
Q

New Drug Application

A

NDA contains all pre-clinical and clinical data collected during a drug’s research & development and must be FDA approved to enter market

25
Q

What information must appear on the label?

A
Approved indications 
Clinical Pharmacology
- Dosage
- Adverse Reactions
- Contraindications
- Special warnings and precautions
e.g. not for pregnancy/specific condition
26
Q

What are the 3 classes of drug recalls and their meanings?

A

Class I: Reasonable probability that use of drug will cause serious adverse health consequences or death e.g. microbial contamination

Class II: Use of drug will cause temporary adverse health consequences, although probability of serious health consequences is remote

Class III: Use of drug is unlikely to cause adverse health consequences e.g. quantity packaging error

27
Q

How does marketing of a generic drug work once the patent is expired?

A

Once a patent has expired any company may submit an Abbreviated New Drug Application (ANDA) to allow marketing of a GENERIC VERSION of the drug with NO need to provide evidence of efficacy & safety