Drug Development, Regulation, Safety

Question 1

GCP

Answer 1

Clinical trail research must meet good clinical practice (GCP) standards
-set of rules that dictate key aspects of clinical trials from study design, facilities, and personnel to proper documentation of data

Question 2

What do clinical trails require review and approval of?

Answer 2

The IND and the IRB

Question 3

Which phase of studies has an emphasis on safety with small patient groups, determine adverse effects, drug metabolism and excretion?

Answer 3

phase 1

Question 4

Which phase of studies has an emphasis on effectiveness and safety?

Answer 4

phase 2

Question 5

What phase of studies involves post-marketing studies that are imposed upon a pharmaceutical firm as a condition for drug approval.

Answer 5

Phase 4

Question 6

Formal submission to the FDA to consider approving a new drug for marketing in the US.

Answer 6

NDA- new drug application- this includes all animal and human data and analyses of the data, as well as information about how the drug behaves in the body and how it is manufactured

Question 7

Which schedule has a lower potential for abuse than schedule IV and consist of preparations containing limited quantities of certain narcotics.

Answer 7

schedule 5- antidiarrheal, antitussive, and analgesic purposes

Question 8

IRB

Answer 8

Institutional Review Board- protection of human research subjects

• informed patient consent
• fully informed of their risks and have given informed consent
• informed that they have alternatives

Question 9

Which schedule has moderate to low potential for physical and physiological dependence?

Answer 9

schedule 3

Question 10

GLP

Answer 10

GLP- pre-clinical research must meet good laboratory practice standards (GLP)
-the set of rules that dictate study design, facilities, and personnel to proper documentation of data

Question 11

Lead compound discovery

Answer 11

This is the first step in pre-clinical development. Assays can be developed to screen for molecules that activate or inhibit a molecular target

• drug targets are either receptors or enzymes
• lead compounds can be discovered in a variety of screening assays

Question 12

Orphan drug status

Answer 12

An orphan drug is developed specifically to treat a rare medical condition (an orphan disease)- orphan drugs status can provide longer patent exclusivity, tax incentives, and financial incentives
-must affect less than 200,000 American citizens

Question 13

PICO

Answer 13

P- patient, population
I- intervention/indicator
C- comparator/control
O- outcome

Question 14

Which phase of studies involves larger trials power to assess safety and effectiveness?

Answer 14

phase 3

Question 15

REMS

Answer 15

Risk evaluation and mitigation strategies- another tool the FDA uses to increase drug safety

Question 16

IACUC

Answer 16

Institutional animal care and use committee- protection of animals in research

Question 17

What are pre-clinical testing?

Answer 17

• In vitro studies
• IACUC
• Animal studies address safety and efficacy

Question 18

In vitro testing

Answer 18

The pharmacological profile of the compound can be determined using in vitro assays. The goal of these experiments is to determine the potency and selectivity of the compound. Cell culture assays (activity, selectivity, toxicity) or tissue slices grown in culture (activity, selectivity, toxicity)

Question 19

Which schedule has a low potential for abuse and low risk of dependence?

Answer 19

schedule 4- Xanax and Ambien

Question 20

Medicinal chemistry

Answer 20

This is the second step in pre-clinical development. Derivatives of a lead compound are synthesized to search for a compound with the required properties of drug.
Drug properties- affinity, stability, and toxicity

Question 21

Animal studies address safety and efficacy

Answer 21

experiments in animal models are used to address efficacy of a drug candidate and other issues, pharmacokinetic properties, adverse effects and toxicity

Question 22

Which schedule has high potential for abuse, severe psychological or physical dependence?

Answer 22

schedule 2- cocaine, methamphetamine, methadone, oxycodone, methylphenidate

Question 23

IND- when is one filed?

Answer 23

Investigational New Drug Application- if pre-clinical testing, both in vitro and in animals justifies human testing an IND application is filed

Question 24

Which schedule has no accepted medical use and a high potential for abuse?

Answer 24

schedule 1- Heroin, marijuana

Question 25

What is included in the IND application?

Answer 25

plans for human testing- a review by a panel organized by the FDA determines if it is reasonably safe to move forward with human testing