Lec 4 Drug Approval and Development

Question 1

amendment passed as a result of the thalidomide disaster

a. Sherley Amendment
b. Durham-Humphrey Amendment
c. Kefauver-Harris Amendment

Answer 1

c. Kefauver-Harris Amendment

Question 2

which of the following is NOT part of the Kefauver-Harris Amendment?

a. medications must demonstrate both safety and efficacy
b. authorized factory inspections
c. transferred jurisdiction of prescription drug advertising from the FTC to the FDA
d. added informed consent and reporting of ADRs for clinical investigations

Answer 2

b. authorized factory inspections

(this is from the Federal Food, Drug, and Cosmetic Act)

Question 3

any manufacturer for drugs used in the U.S. must be registerd with the FDA and undergo an inspection at least once every ___ years

a. one
b. two
c. three
d. four

Answer 3

b. two

Question 4

if you don’t comply with cGMP, the product is

a. adulterated
b. misbranded
c. both
d. neither

Answer 4

a. adulterated

Question 5

if a manufacturer doesn’t register with the FDA, the product is

a. adulterated
b. misbranded
c. both
d. neither

Answer 5

c. both

Question 6

in law, making drugs falls into what 3 general categories?

Answer 6

1. pharmacy or traditional compounding (503a)
2. outsourcing facilities (503b)
3. manufacturing

Question 7

compounding according to prescriptions specific to particular patients on an as needed basis.

a. pharmacy or traditional compounding (503a)
b. outsourcing facilities (503b)
c. manufacturing

Answer 7

a. pharmacy or traditional compounding (503a)

Question 8

manufacturing large batches with or without prescriptions to be sold to facilities for office use only.

a. pharmacy or traditional compounding (503a)
b. outsourcing facilities (503b)
c. manufacturing

Answer 8

b. outsourcing facilities (503b)

Question 9

mass production of drug products that have been approved by the Food and Drug Administration.

a. pharmacy or traditional compounding (503a)
b. outsourcing facilities (503b)
c. manufacturing

Answer 9

c. manufacturing

Question 10

meeting the requirements for 503a compounding will exempt the pharmacy from meeting regulations related to: (3 things)

Answer 10

-cGMP
-Misbranding (related to drug labeling)
-New Drug Requirements

Question 11

which of the following is FALSE about outsourcing facilities?

a. no prescription needed for a specific pt
b. must register, pay annual fees, and be inspected by FDA
c. can compound an already commercially available product

Answer 11

c. can compound an already commercially available product

Question 12

product labels for outsourcing facilities must contain: (11 things, it’s a lot)

Answer 12

-statement “this is a compounded drug”
-name, address, phone number of outsourcing facility
-lot number or batch number
-established name of the drug
-dosage or strength
-quantity or volume
-beyond use date
-storage and handling instructions
-NDC
-statement “not for resale”
-list of active and inactive ingredients

Question 13

which laws are applicable to pharmacy compounding (503a)?

a. misbranding
b. cGMP
c. new drug requirements
d. all of the above
e. none of the above

Answer 13

e. none of the above

Question 14

which laws are applicable to outsourcing facilities (503b)? SELECT ALL THAT APPLY

a. misbranding
b. cGMP
c. new drug requirements
d. all of the above
e. none of the above

Answer 14

a. misbranding (labeling/packaging)
b. cGMP

Question 15

which laws are applicable to drug manufacturers?

a. misbranding
b. cGMP
c. new drug requirements
d. all of the above
e. none of the above

Answer 15

d. all of the above

Question 16

Kefauver-Harris amendment: what happened to drugs still on the market that came out BEFORE 1938?

Answer 16

assumed to be safe and effective, “grandfathered” in

Question 17

Kefauver-Harris amendment: what happened to drugs that came out AFTER 1938?

Answer 17

they are investigated for efficacy, any issues recommended to the FDA (DESI = drug efficacy study implementation)

Question 18

T or F: for DESI, products must be pulled from the market until a decision is made

Answer 18

F

Question 19

T or F: some DESI decisions have yet to be made, even today

Answer 19

T

Question 20

in vivo animal testing

a. preclinical research
b. phase I
c. phase II
d. phase III
e. post-marketing safety monitoring

Answer 20

a. preclinical research

Question 21

the Investigational New Drug (IND) must contain what 3 things?

Answer 21

-animal pharmacology and toxicology studies
-manufacturing information
-clinical protocols and investigator information

Question 22

Once the IND is submitted, the sponsor must wait ___ days before initiating any clinical trials to allow the FDA to review the IND for safety and unreasonable risk.

a. 2
b. 5
c. 30
d. 180

Answer 22

c. 30

Question 23

evaluates safety and dosage of the compound; healthy human volunteers; baseline human PK and PD properties reviewed

a. preclinical research
b. phase I
c. phase II
d. phase III
e. post-marketing safety monitoring

Answer 23

b. phase I

Question 24

determine the effectiveness of therapy; several hundred human volunteers with the disease

a. preclinical research
b. phase I
c. phase II
d. phase III
e. post-marketing safety monitoring

Answer 24

c. phase II

Question 25

Goal is to demonstrate efficacy at a higher power, and to expand information around AEs; hundreds/thousands of pts in several geographic locations a. preclinical research b. phase I c. phase II d. phase III e. post-marketing safety monitoring

Answer 25

d. phase III

Question 26

new drug application (NDA) must include everything from preclinical data to phase 3 trial data. It also must include: (7 things)

Answer 26

-proposed labeling -safety updates -drug abuse information -pt information -data from studies outside the U.S. -institutional review board compliance information -directions for use

Question 27

-FDA reviews MedWatch -routine manufacturer inspections a. preclinical research b. phase I c. phase II d. phase III e. post-marketing safety monitoring

Answer 27

e. post-marketing safety monitoring (phase 4)

Question 28

NDA timeline: how much total time does it take? (range)

Answer 28

3.5-13.5 years

Question 29

law enacted to encourage the development of drugs for rare disease states

Answer 29

Orphan Drug Act

Question 30

Hatch-Waxman act purpose

Answer 30

to reduce the requirements for approval of generic prescription drugs

Question 31

look at Orphan Drug Act (slide 24)

Answer 31

you got it

Question 32

which of the following is TRUE about the Hatch-Waxman Act? a. before this act, generics can be approved without impinging on patent b. after this act, generic companies had to perform own safety and efficacy studies c. before this act, approval for generics can't be started until the patent expires d. before this act, generic companies had to prove bioequivalence and proof of acceptable manufacturing

Answer 32

c. before this act, approval for generics can't be started until the patent expires

Question 33

difference between NDA and abbreviated NDA

Answer 33

abbreviated NDA does not need animal or human clinical trial date to establish safety and effectiveness

Question 34

What term is being described?: demonstration that the rate of absorption of the generic drug is equivalent to that of the innovator drug. Must deliver the same amount of ingredient in the same amount of time

Answer 34

bioequivalence

Question 35

what document needs to be submitted after preclinical research in the drug development process?

Answer 35

IND (investigational new drug application)