FDA drug approval process

Question 1

what is a drug?

Answer 1

-substance recognized by a formulary
-used to treat, cure, prevent, diseases that is not a food

Question 2

prescription drugs

Answer 2

require a doctor authorization to purchase

Question 3

generic drugs

Answer 3

product “=” to brand name drugs in terms of preformace

Question 4

OTC drugs

Answer 4

available to buy w/o doctor prescription

Question 5

what is overseen by CDER and then CBER?

Answer 5

CDER: prescription, generic, OTC drugs
CBER: biologics, vaccines, cell/gene therapy

Question 6

development of drug process

Answer 6

-discovery and early development
-pre-clinical
-clinical (phase I, II, III, IV for post approval)
-submit to FDA
-post marketing safety monitoring

Question 7

preclinical steps in drug development

Answer 7

-understand disease
-identify potential targets
-in vivo and in vitro studies
-initial work to determine formulation

Question 8

US regulations allow for human drug testing only after ___ is filed

Answer 8

IND (investigational new drug)

Question 9

types of IND

Answer 9

investigator
emergency
treatment

Question 10

primary goal of phase 1

Answer 10

establish the overall safety

Question 11

IND includes data from

Answer 11

-pre-clinical studies
-human experience from foreign studies
-protocols
-investigators brochure
-informed consent commitment

Question 12

2ndary goal of phase 1

Answer 12

-establish max/min dose
-find serious side effects
-determine metabolism of drug

Question 13

primary goal of phase 2

Answer 13

establish activity of a drug for group of pt w/ a disease

Question 14

2ndary goal of pahse 2

Answer 14

determine common short term side effects

Question 15

primary goal of phase 3

Answer 15

confirm safety and effectiveness for drug for pt with a disease

Question 16

2ndary goal for phase 3

Answer 16

evaluate overall risk benefit ratio

Question 17

priamary goal of phase 4

Answer 17

monitor ongoing safety in large populations and uncontrolled drug use

Question 18

2ndry goal for phase 4

Answer 18

-identify unusual side effects
-identify additional uses of drug

Question 19

phase 1 # of participants

Answer 19

10-75

Question 20

phase 2 # of participants

Answer 20

50-300

Question 21

phase 3 and 4 # of participants

Answer 21

300-5k

Question 22

phase 1 # of participating institutions

Answer 22

1

Question 23

phase 2 # of participating institutions

Answer 23

1-5

Question 24

phase 3 and 4 # of participating institutions

Answer 24

5-100

Question 25

phase 1 length of time to complete

Answer 25

1-6 months

Question 26

phase 2 length of time to complete

Answer 26

6 months-2 yrs

Question 27

phase 3 length of time to complete

Answer 27

1-10yrs

Question 28

phase 4 length of time to complete

Answer 28

6 months- 5yrs

Question 29

typical cost phase 1

Answer 29

100k-1m

Question 30

typical cost phase 2

Answer 30

10m-100m

Question 31

typical cost phase 3

Answer 31

10m-500m

Question 32

typical cost phase 4

Answer 32

10-100m per trial

Question 33

what is the top 3 most expensive clinical phase by therapeutic area?

Answer 33

(most)= pain and anesthesia ophalmology anti-infective

Question 34

if application for drug is deemed fileable: complete vs incomplete

Answer 34

incomplete= FDA issues a refuse to file letter complete=application is forwarded for review

Question 35

during review of drug application: the FDA will contact the sponsor on ______

Answer 35

correctable deficiencies

Question 36

major scientific issues regarding the review of a drug application

Answer 36

-requires consideration of entire application agency -addressed by agency in form of an action letter at initial review process

Question 37

during review of drug application, what are the areas: (3)

Answer 37

-easily correctable deficiencies -major scientific issues -advisory committee

Question 38

not approved letter

Answer 38

lists the deficiencies in the application and explains why it wont be approved

Question 39

approvable letter

Answer 39

signals the drug can be approved and lists minor deficiencies like labeling changes or post-approval studies

Question 40

approval letter

Answer 40

states the drug is approved

Question 41

drug labeling must be justified by data in the

Answer 41

NDA (new drug application)

Question 42

the labeling outlines the

Answer 42

benefits and risks of the product

Question 43

labeling: description

Answer 43

drug name, dosage, ingredients, formula

Question 44

labeling: clinical pharmacology

Answer 44

action of drug in human and in vivo and vitro actions, pharmacokinetics

Question 45

labeling: indications and usage

Answer 45

situations where drug should not be used

Question 46

labeling: warnings

Answer 46

serious adverse reactions or safety hazards

Question 47

labeling: precautions

Answer 47

describes undesirable effects associated with drug

Question 48

labeling: drug abuse/dependence

Answer 48

types of abuse that can occur with the drug and adverse reactions

Question 49

labeling: over dosage

Answer 49

signs, symptoms, and lab findings of over dosage and general treatment

Question 50

labeling: dosage/administration

Answer 50

recommendation of dosage use and upper limit dose

Question 51

labeling: how supplied

Answer 51

info on dosage forms

Question 52

FDA claims advertisements and promotional fall under regulations of labeling, does this include social media?

Answer 52

yes

Question 53

post approval curveillance

Answer 53

-monitor safety and product complaints -monitor publicaitions

Question 54

post approval complaince

Answer 54

-notify and submit labeling changes to gain approval -submit all advertisements to FDA first -notify all manufacturing changes

Question 55

class 1 recall

Answer 55

high risk that the use of product will cause serious health problems or death

Question 56

class 2 recall

Answer 56

lower risk the product can cause adverse health reactions, reversible

Question 57

class 3 recall

Answer 57

minimal risk of the product will cause adverse health reactions