Drug Regulation and Monitoring

Question 1

How can subject bias be countered during a clinical trial?

Answer 1

placebo

Question 2

How can observer bias be countered in a clinical trial?

Answer 2

blinding, especially double blinding

Question 3

What type of design can detect natural fluctuation of disease?

Answer 3

Cross-over design

Question 4

________ of subjects counters the bias of selection.

Answer 4

Randomization

Question 5

Phase I studies:

Answer 5

20-80 healthy patients
Intended to establish safety and tolerability
Focus included dosing and PK parameters
Often non-blinded=all subjects get drug

(if high levels of toxicity are expected patients with target condition may be used in place of healthy volunteers)

Question 6

Phase II studies:

Answer 6

50-300 patients with the medical condition of interest
Objective= determine effectiveness of drug for a particular disease
Capable of detecting some AE
Dose response and dosing regimens
Usually involves a single-blind study, where drug is evaluated against placebo or standard of care
Several dosing options to obtain optimum doe range and toxicity info
May pinpoint data that must be collected in phase III

Question 7

Phase III trials:

Answer 7

300-3000 patients and are conducted at multiple sites
Studies are based on specific clinical or surrogate endpoints
Randomized, controlled, double-blind trials with multiple arms
Results should provide adequate basis for extrapolating results to the general population

Question 8

Accelerated development and approval of a drug for serious/life-threatening disease

Answer 8

Fast Track Status

Question 9

Sponsor formally proposes that FDA approve new drug for sale in the US

Answer 9

New Drug Application

Question 10

Sponsor submits preclinical research data to FDA to proceed with human clinical trials

Answer 10

Investigational New Drug Application

Question 11

For disease that affects <200,000 pts in US, 7yrs exclusivity patent

Answer 11

Orphan Drug

Question 12

Ensure the rights and welfare of clinical trial participants before and during the trial

Answer 12

Investigational Review Board

Question 13

Meds in phase III development, gives access to patients who may not survive

Answer 13

Compassionate Use

Question 14

Reviews safety/efficacy data on interim basis

Answer 14

Data and Safety Monitoring Board

Question 15

Phase IV Studies:

Answer 15

Post drug approval studies
More info about side effects and safety of the drug when used in the general population
Long term risk/benefits
Efficacy when used in the general population
May be required by the FDA

Question 16

True or False: A phased trial is needed for a generic drug to be approved for use.

Answer 16

False

Question 17

Generic drugs must prove _________ to innovator drugs.

Answer 17

Bioequivalence = same absorption, distribution, metabolism, and excretion

(Same batch requirements for identity, strength, purity, and quality as the innovator drug)

Question 18

Generic drugs must have the same _______ as the innovator drug.

Answer 18

Active

Question 19

What is the purpose of MEDWatch?

Answer 19

To capture serious AE data post drug approval and report them.
Any person can report to MEDWatch

Question 20

What are required risk management plans that use risk minimization strategies beyond the professional labeling to ensure that the benefits of certain Rx drugs outweigh their risk?

Answer 20

REMS(Risk Evaluation and Mitigation Strategies)