Exam 1 - Drug development and approval process (based off of objectives)

Question 1

for a new drug to be released it must
1. be discovered and developed
2. undergo preclinical research development
3. undergo clinical trials
4. have an ________ reviewed by the _______
5. be _____________ for release

Answer 1

for a new drug to be released it must
1. be discovered and developed
2. undergo preclinical research development
3. undergo clinical trials
4. have an NDA reviewed by the FDA
5. be manufactured for release

Question 2

for a new drug to be released it must
1. be _________ and __________
2. undergo ___________ ___________ development
3. undergo ________ _______
4. have an NDA reviewed by the FDA
5. be manufactured for release

Answer 2

for a new drug to be released it must
1. be discovered and developed
2. undergo preclinical research development
3. undergo clinical trials
4. have an NDA reviewed by the FDA
5. be manufactured for release

Question 3

Compliance of GLP (good lab practices)

Answer 3

Compliance of GLP (good lab practices) requires documentation of
training
study schedules
processes
status reports
that are all sent to FDA for approval of clinical investigations.

Question 4

What is the purpose of laboratory experiments and animal investigation during the preclinical investigation

Answer 4

to use pharmacodynamics, pharmacokinetics and toxicology testing to assess therapeutic effects of the NME on living organisms.

Question 5

The pre clinical research phase can take up to _____ years and at the end of this phase _____________

Answer 5

The pre clinical research phase can take up to 3-6 years and at the end of this phase the IND is submitted

Question 6

What is IND application

Answer 6

investigational new drug application. This is basically an application to move into the clinical phase. In the clinical phase the drug will be then considered an investigational new drug.

Question 7

What takes place during the preclinical research and development stage

Answer 7

the initial synthesis of drugs, animal testing of drugs, lab studies of drugs, and institutional board review of drugs takes place during the preclinical research and development stage

Question 8

the initial synthesis of drugs, animal testing of drugs, and institutional board review of drugs takes place during the _________ stage

Answer 8

preclinical research and development stage

Question 9

After the IND is submitted how does a drug company know they can continue into clincal trial

Answer 9

the FDA will not approve it, if the FDA does not object within a 30-day period, the clinical trials may begin.

Question 10

What are the ground rules for clinical trials?

Answer 10

What are the ground rules for clinical trials?
1. clinical study protocol must be developed by sponser, reviewed and aproved by an IRB (institutional review board).
2. All documents should remain open to the FDA for inspection at any time.

Question 11

What is the job of the IRB (institutional review board)
1. ___________________
2.________________________

Answer 11

What is the job of the IRB (institutional review board)
1. IRB oversees the research to protect human test subjects rights as well as maintain medical and scientific standards
2. must review and approve informed consent documents prior to commencing the trials

Question 12

The golden standard for clinical trial design is ___________ because it minimizes _________ and ___________ and also ensures that ___________

Answer 12

The golden standard for clinical trial design is double blind randomized controlled trial because it minimizes bias and confounding factors and also ensures that the given results are due to the intervention

Question 13

Randomization

Answer 13

Randomization means that there is more than one intervention and that participants are assigned to one of the intervention groups randomly.

Question 14

Blinding in the clinical trials reduces ______________________________

Answer 14

Blinding in the clinical trials reduces being influenced by knowledge, this is done by making all activities and treatment items identical.

Question 15

The key of a clinical trial is to have a _______________ to reach a _______ regarding the _____________

Answer 15

The key of a clinical trial is to have a sufficient sample size to reach a conclusion regarding the outcome

Question 16

Superiority design is __________________

Answer 16

comparison with a placebo

Question 17

Non-inferiority design is a design that ___________

Answer 17

confirms that one intervention IS NOT iinferior to a comparator

Question 18

Equivalence design determines whether __________

Answer 18

one intervention is statistically no different in effectiveness than another

Question 19

Clinical trials can take up to _______- years and only deal with about ____________ for testing. At the end of these trials, a_______________- is submitted to the FDA.

Answer 19

Clinical trials can take up to 6-7 years and only deal with about 5 drug compounds for testing. At the end of these trials, an NDA is submitted to the FDA.

Question 20

What questions should be answered by the clinical process

Answer 20

What questions should be answered by the clinical process
1. does the drug have the effect that its supposed to have
2. how much of the drug to give and how often to give it
3. what side effects are associated with the drug and can they be managed
4. how is the drug broken down and how long does it stay in the body
5. which food, drinks and other drugs can be used at the same time or should be avoided.

Question 21

what are the phases of clinical trials

Answer 21

1. testing in healthy subjects
2. testing in individuals with the disease
3. larger scale testing in individuals with disease.

Question 22

IND Phase 1
establishes

Answer 22

IND Phase 1
-Establishes PK, Safety and dosage
- uses healthy participants
- open label
- fast track conditions allow persons with dx to participate in study
- study size is less than 100 subjects
- duration is 1 year or less

Question 23

IND phase 2
establishes

Answer 23

IND phase 2
-First controlled trial to establish pk profile, safety and efficacy
- 2a consists of pilot study to determine initial efficacy
- 2b is controlled study
- single or double blinded
- fast track conditions allow persons with dx to participate in study
- study size is medium several hundered subjects
- duration is weeks to months

Question 24

IND phase 3
establishes

Answer 24

IND phase 3
- double blind
- study size large
- duration - months to years
- determines labelling, marketing claims, final formula, product stability, packaging and storage conditions

Question 25

The goals of NDA are to provide enough info to permit the FDA reviewer to determine whether:
1. ________________
2. ______________
3. _____________________

Answer 25

The goals of NDA are to provide enough info to permit the FDA reviewer to determine whether:
1. the drug is safe and effective for proposed indications and its benefits outweigh the risks
2. the package insert of the drug is appropriate
3. the manufacturing and quality control methods are appropriate to preserve the drugs identity, strength, quality and purity.

Question 26

FDA Sentinel initiative

Answer 26

FDA Sentinel initiative is designed to monitor the safety of the FDA regulated medical products and requires them to complete the adverse event reporting system (FAERS)

Question 27

FDA adverse event reporting system (FAERS)

Answer 27

FDA adverse event reporting system (FAERS) is a system that monitors any adverse events reported by healthcare professionals and consumers in the US. If a event is reported it must be sent to the FDA for further evaluation of a potential safety concern.

Question 28

What are the three types of letters the FDA will give after reviewing a drug

Answer 28

approval letter - all requirements met, company can market drug
approvable letter - some minor deficiencies which must be addressed before approval letter
non approvable letter - major concerns with application

Question 29

Drug discovery and development process includes:

Answer 29

Drug discovery and development process includes:
- identification of the active ingredient or new molecular entity (NME)
- this can take 2-10 years

Question 30

What are the two main types of discovery used in the first phase

Answer 30

compound-centered drug discovery and target centered drug discovery

Question 31

what are compound-centered drug discoveries
- __________________ that were tested on ____________________
- compounds were typically ______________ or ___________

In short: __________-

Answer 31

what are compound-centered drug dicoveries
- produced compounds that were tested on biological target receptors
- compounds were typically endogenous to the body or found in nature.

In short: blind testing, uses serendipity. compound is found first and then searches for target

Question 32

What are target-centered drug discoveries

Answer 32

What are target-centered drug discoveries
- chemical compounds created to hit a certain target

in short: target is discovered first and then a compound is created

Question 33

What are gene-based therapies

Answer 33

modifying a persons genes to treat and prevent diseases

Question 34

What is pharmacogenetics

Answer 34

A field that studies how a persons genes affect how they respond to a medication

Question 35

Describe lead optimization
- manipulations made to __________ that improve ___________
- The right match between compound and target is called ___________

Answer 35

Describe lead optimization
- hundreds of manipulations made to maximize receptor affinities and improve pharmacokinetic properties
- The right match between compound and target is called lead compound.

Question 36

How is finding the lead compound achieved

Answer 36

de-novo rational drug designs
high through put screening
combinational chemistry
serendipity

Question 37

Orphan drugs are drugs used to __________________________. because of low numbers, the manufacturer cant expect to recover the cost of developing the drug. therefore orphan drug act 1983 permits ____________________

Answer 37

Orphan drugs are drugs used to treat rare diseases that affect fewer than 200,000 people in the US. because of low numbers, the manufacturer cant expect to recover the cost of developing the drug. therefore orphan drug act 1983 permits grant assistance for clinical research, tax credits, and 7 year market exclusively to the first applicant.

Question 38

Abbreviated new drug applications (ANDA) are used to market generic drugs to provide a ______, ______ and ___________ to brand-name drugs. These do not require __________ and ________- data but must demonstrate bio equivalence through __________ of the drug. to be approved, the generic version must deliver ______________ into a patients blood stream.

Answer 38

Abbreviated new drug applications (ANDA) are used to market generic drugs to provide a safe, effective and lower cost alternative to brand-name drugs. These do not require preclinical (animal) and clinical (human) date but must demonstrate bioequivalence through rate of absorption of the drug. to be approved, the generic version must deliver the same amount of active ingredients into a patients blood stream.

20% variance acceptable and 20 years patent

Question 39

OTC status means that a medication has a wide __________________, ________________, ________________, and has adequacy of _________________

basically this means that___________________

Answer 39

OTC status means that a medication has a wide margin of safety, method of use, benefit to risk ration, and has adequacy of labeling for self medication

basically this means that the medications is safe enough and has the sufficent instructions for someone to use it without the counsel or close watch of a provider.

Question 40

OTC products will not fall under prescription status if __________________________

Answer 40

OTC products will not fall under prescription status if FDA deems it to be GRASE

Question 41

Biologics are derived from _____________ and require a ______________________________. These products are dealt with by _________and are treated and tested differently than drugs because of ___________.

Answer 41

Biologics are derived from living organisms and require a biologics license application. These products are dealt with by CBER and are treated and tested differently than drugs because of composition.

Question 42

Devices are any________________________ (which can be as simple as a toothbrush). These are dealt with by the _____________and are classified as _________________ depending on ______________

Answer 42

Devices are any device with a theraputic claim (which can be as simple as a toothbrush). These are dealt with by the CDRH and are classified as 1, 2, and 3 depending on their risk

Question 43

The classes of recall by the FDA are
class 1: if product is ______________
class 2: if product _____________________________
class 3: if product is _______________________

Answer 43

The classes of recall by the FDA are
class 1: if product is seriously harmful
class 2: if product causes temporary or medically reversible harm
class 3: if product is not likely to cause adverse effects.

Question 44

Limitations of the FDA Drug approval process include:
1. no _____________of ________________
2. rarely requires _________________ of _____________
3. does not review __________, _____________, or _____________
4. _______________ of drugs and devices maybe _________
5. pressure for ____________ may lead to ______________ concerns
6. agency is __________________________
7. ________________ may exist on FDA _______________.

Answer 44

Limitations of the FDA Drug approval process include:
1. no comparison of competing drugs
2. rarely requires clinical efficacy tests of new devices
3. does not review advertisements, cost effectiveness, or regulate surgery
4. post marketing surveillance of drugs and devices maybe inadequate
5. pressure for quick approvals may lead to safety/efficacy concerns
6. agency is slow and bureaucratic
7. conflicts of interest may exist on FDA advisory committees.