FDA's Drug Review Process

Question 1

often, a drug is developed to do what?

Answer 1

treat a specific disease

Question 2

drugs can also be discovered….

Answer 2

by accident

Question 3

give an example of a drug whose purpose was discovered “on accident”

Answer 3

retrovir was first studied as an anticancer drug with no success.

twenty years later, researchers discovered that it could treat AIDS

Question 4

preclinical testing is also known as ___ testing

Answer 4

animal

Question 5

true or false

most drugs that undergo preclinical testing never even make it to human testing and review by the FDA

Answer 5

true

Question 6

before submitting an IND, the pharmaceutical company may seek advice from whom?

Answer 6

the FDA

Question 7

Explain what happens at the submission of an IND

Answer 7

the sponsor (company, research institution, or other organization that takes responsibility for developing a drug) must show the FDA results of preclinical testing in lab animals AND propose what they plan to do for human testing

Question 8

drug studies in humans (clinical trials) can only begin when?

Answer 8

after an IND is reviewed by both the FDA and an IRB (localized institutional review board)

Question 9

is is the “local institutional review board” that reviews an IND along with the FDA?

Answer 9

a panel of scientists and non scientists in hospitals and research institutions.
they oversee clinical research

Question 10

who approves the clinical trial protocols?

Answer 10

IRBs

Question 11

what do the clinical trial protocols explain?

Answer 11

-type of people who can participate
-schedule of tests and procedures
-medications and dosages to be studied
-length of study
-objectives of study

Question 12

_____ ensures that the proposed clinical trial is acceptable, that the participants have given consent and are fully informed of the risks, and that the researchers take appropriate steps to protect the patient from harm

Answer 12

IRBs

Question 13

phase 1 studies are conducted in what kind of volunteers? how many?
what is the purpose of phase 1?

Answer 13

HEALTHY volunteers 20-80 of them
goal: determine what the drug’s most frequent side effects are, how the drug is metabolized and excrete

Question 14

phase 2 studies only begin if……..

Answer 14

phase 1 studies don’t reveal unacceptable toxicity

Question 15

the emphasis of phase 1 is on ___ while the emphasis of phase 2 is on _____

Answer 15

phase 1 = safety
phase 2 = efficacy

Question 16

phase 2 aims to obtain preliminary data on what?

Answer 16

whether the drug works in people who HAVE the disease or condition

Question 17

phase 2 is conducted on what 2 groups?

Answer 17

-patients with the condition receiving the experimental drug

-patients with the condition who are receiving a different treatment — either a placebo or a different real drug

Question 18

how many subjects are typically in phase 2 clinical trials?

Answer 18

few dozen - 300

Question 19

what happens at the end of phase 2?

Answer 19

FDA and sponsors come up with an agreement on how large-scale studies in phase 3 should be done

Question 20

name the 2 common meeting points of the sponsor with the FDA

Answer 20

-before submitting an IND
-after phase 2 to discuss phase 3

Question 21

phase 3 studies only begin if…….

Answer 21

evidence of effectiveness is shown in phase 2

Question 22

explain what is happening in phase 3 and how many subjects there are

Answer 22

gather more info about safety and effectiveness
studies DIFFERENT POPULATIONS, DOSAGES, AND USING THE DRUG IN COMBO WITH OTHER DRUGS

number of subjects ~3000

Question 23

what is the “formal step” a drug sponsor takes to ask that the FDA consider approving a new drug for marketing in the United States?

Answer 23

NDA (new drug application)

Question 24

when an NDA is submitted, the FDA has how long to decide whether to file it so it can be reviewed?
what would be a reason to NOT file it

Answer 24

60 days
if the application is incomplete (ex – required studies missing)

Question 25

in accordance with _________, the CDER expects to review and act on at least _______% of NDAs for standard drugs, no later than ____ months after the application has been receive

Answer 25

PDUFA (prescription drug user fee act)
90%
10 months

Question 26

though the FDA expects to review and act on at least 90% of NDAs for standard drugs no later than 10 months after the applications are received, what is the REVIEW GOAL for priority drugs

Answer 26

6 months

Question 27

what’s the longest part of the drug review process, taking several years

Answer 27

clinical trials

Question 28

what is the “accelerated approval program”

Answer 28

allows earlier approval of drugs that treat serious diseases that fill an UNMET medical need

Question 29

HOW is approval faster in the “accelerated approval program”

Answer 29

FDA can base the drug’s effectiveness on a “surrogate endpoint” such as blood tests or X-ray results, rather than waiting for results from a clinical trial

Question 30

what is the “fast track” program

Answer 30

helps reduce the time for FDA’s review of products that treat serious or life-threatneing diseases AND those that have the potential to address unmet medical need

Question 31

explain how drug approval works in the fast track program

Answer 31

drug sponsors can submit portions of an application AS THE INFO becomes available (called rolling submission) instead of having to wait until all info is available

Question 32

under MedWatch, when important new risks are uncovered, what is done?

Answer 32

the risks are added to the drug’s labeling and the public is informed through letters, public health advisories, and other education

in some cases, the use of the drug can be substantially limited

in RARE cases, the drug needs tp be withdrawn

Question 33

who runs medwatch?
is it mandatory?

Answer 33

the FDA
not mandatory

Question 34

since _____ was passed in 1992, more than 1000 drugs and biologics have come into the market, including new medicines to treat cancer, aids, cardiovascular disease, and life-threatening infections

Answer 34

PDUFA

prescription drug user fee act
drug co pays fees that boost FDA resources and FDA agrees to time frames for its review of NDAs