PHR 2500 Sample Questions

Describe the importance for researchers to follow ethical guidelines in human trials.

could discuss ethical travesties that occurred (WWII and Nazi experiments, Tuskegee syphilis experiments), could discuss fostering trust between the researcher and participant (less likely to quit or drop), could discuss the lowering of risk for bodily harm to occur in the participants, etc.

What incident established the need to ensure drug safety before putting medications on the market and led to the approval of the Federal Food, Drug and Cosmetic act in 1938?

the Sulfanilamide incident from 1937

Usually lasts 1/2 year and is meant to establish safety

Phase 1 clinical trials

Purpose of this trial is to determine comparative efficacy

Phase 3 clinical trials

A blockbuster drug is an extremely popular drug that generates annual sales of at least _____ for the company? answer:

$1 billion

2. Fill in the blank for the general rule of thumb for determining effectiveness by the FDA. If a _____ already exists, the _____ drug should be just as _______. If no _____ exists, the drug should _____ a patient's _____ ____ _____.

(cure, new, effective) (cure, improve, quality of life)

Which of the following are incentives of the orphan drug designation? (Note: This answer has multiple answers; please check all that are correct)

7 years market exclusivity instead of 5 years The possibility of the drug company receiving new grants NOT: Tax increases to the drug company

Name two examples of potential sponsors for clinical investigations?

- pharmaceutical companies, government entities such as the NIH, universities, or other non-profit groups

What are two of the four criteria the FDA uses to consider something a drug?

- recognized by an official pharmacopoeia or formulary - is intended for use in the diagnosis, cure, mitigation, treatment, or prevention of disease - is intended to affect the structure or any function of the body - is intended for use as a component of a medicine

Give an example of one common use of monoclonal antibodies.

-over the counter pregnancy test -covid test -recombinant DNA

How many ethical principles does the Nuremburg Code contain?

10

Which drug law establishes new expedited product development programs, such as the Breakthrough Devices program?

21st Century Cures Act

How long does the FDA have to review an NDA to determine if it is sufficiently complete to conduct a full review?

60 days

Which of the following is NOT an incentive of the orphan drug designation?

9 years market exclusivity instead of 5 years

3. What are the routes that a prescription can take to become an over the counter drug? (Select all that apply.) a. (RX-OTC), full switch, supplemental NDA b. (RX-OTC), OTC monograph, new NDA c. (RX-OTC), partial switch, new NDA d. (RX-OTC), full switch, OTC monograph

A & C

This designation allows drugs for serious conditions that filled an unmet medical need to be approved based on a surrogate endpoint.

Accelerated Approval

What are the 4 parameters that predict the pharmacological profile of a drug?

Absorption, Distribution, Metabolism and Excretion

The purpose of this expedited process is to allow drugs for serious conditions that fill an unmet need to be approved based on a surrogate endpoint.

Accelerated Approval

A biologic can be a range of product types. The following is FALSE regarding biologic?

A biologic is not a type of drug according to the FDA definition of a drug

What is a chemical library?

A collection of information on drug molecules that include their structure, purity, quantity and physiochemical characteristics

Which of the following is considered a basic element of informed consent? Select all that apply.

A description of any reasonably foreseeable risks or discomforts to the subject. An explanation of whom to contact for answers to questions about the research and research subjects' rights. A description of any benefits to the subject or to others which may reasonably be expected from the research A statement that participation is voluntary and that refusal to participate will involve no penalty.

Which of the following statements about a drug's formulation is FALSE?

A drug's formulation has NO impact on which organs the drug can reach in the body.

In which of the following situations would it be most likely that a drug with serious side effects be approved?

A new drug that cures Ebola, for which no cures exist currently

A phase 3 clinical trial has just begun for the new drug, Buckeyecillin. The study design states that this will be a randomized, two-armed study with a control group receiving a placebo and the treatment group receiving Buckeyecillin. Only the investigators will know who is receiving which medication. This study is:

A single-blind interventional study

What is an umbrella study?

A trial that enrolls patients with the same cancer, but different genetic mutations to look for potential treatments

In target identification, what is a molecule that inhibits the activity of a particular target? A. Antagonist B. Agonist C. Stopper D. Enzyme

A. Antagonist

You learned about active ingredients and inactive ingredients in the first lecture. Which of the following is TRUE?

An active ingredient is any component that provides pharmacological activity and may have a direct effect on the structure or function of the body Inactive ingredients are part of a drug product's formulation, which can influence the effects of the drug product on the body An active ingredient can be formulated and delivered in several ways such as orally or through the skin

You learned about active ingredients and inactive ingredients in the first lecture. Which of the following is FALSE?

An active ingredient is synthesized in a lab or isolated from nature to be delivered with inactive ingredients only in rare circumstances

Which of the following is NOT part of Good Manufacturing Practice (GMP)?

Assuring the integrity of the data collected

When can the fast track designation be requested by the drug company?

At any point during the drug development process

Who must formally approve the name of a branded drug before it is sent out to the market? A. The consumers B. The FDA C. The scientists D. The shareholders

B. The FDA

Written by a national commission in 1979. Defines 3 basic ethical principles: respect for persons, beneficence, and justice; These principles are applied to informed consent, assessment of risk vs. benefit, and selection of subjects

Belmont Report

A biologic can be a range of product types. The following is TRUE regarding biologic?

Biologics include proteins, which may be derived from living systems One example of a biologic active ingredient is a vaccine Biologics differ from small molecule drugs such as aspirin in both size and complexity

Which of the following examples may be awarded 3 years of market exclusivity?

Braxtocillin was originally approved to treat infections, but Buckeye Pharmaceuticals has just re-submitted the drug to be approved for a second indication, which is treatment of high blood pressure.

A drug that receives this designation gets all fast track designation features as well as intensive guidance and organizational commitment from the FDA

Breakthrough Therapy

Which of the following statements is TRUE regarding the experimentation, or testing, of unapproved drug products?

Clinical studies utilize animal testing before beginning clinical studies on humans. Sometimes the unapproved drug, in rare cases, can be given to severely ill patients when no other treatment exists. About a quarter of pharmaceutical spending is spent on clinical studies

Involves 100-500 volunteers and is the first time efficacy is evaluated

Phase 2 clinical trials

The FDA oversees which centers? Check all that apply.

Center for Drug Evaluation and Research Center for Biologic Evaluation and Research

created by the World Medical Association that put forth a set of ethical principles for medical researchers involved in human research.

Declaration of Helsinki

The Alzheimer's Association announces that it is paying for clinical trials to begin on a new drug, Remembral, that has shown promise in slowing the progression of Alzheimer's in animal models. The clinical trials will be performed at the University of Montana under Dr. Patty Potter. They are currently looking for volunteers to participate in the trial. Who is the principal investigator of this trial?

Dr. Patty Potter

What is "an expedited authorization and use of an unapproved product or the off-label use of an already approved product in a declared emergency"?

Emergency Use authorization

What are the 4 parameters that predict the pharmacological profile of a drug? Check all that apply.

Excretion Absorption Metabolism Distribution

List at least 3 of the 8 basic elements of informed consent.

Explanation of research, Description of risks, Description of benefits, Alternative treatments, Confidentiality, Compensation for injury, Who to contact, Voluntary

This designation gives the company more frequent meetings and communication with the FDA, as well as eligibility for other designations

Fast track designation

Which drug law improved patient access to already approved and experimental drugs?

Food and Drug Administration Modernization Act

_______ are not legally binding, but rather strong suggestions from the FDA. Compared to ________, which are required or authorized by the statute

Guidances, regulations

What are in silico studies?

In silico studies utilize computer models to model molecule-target binding

Which of the following is NOT included in a New Drug Application (NDA)?

Information on the profit margin of the drug

Why is it important to have good manufacturing practice?

It assures the identity, strength, quality, and purity of drug products

Which of the following statements is FALSE regarding the experimentation, or testing, of unapproved drug products?

It can take up to 5 years to get a new molecule approved by the FDA

How does the Priority Review designation speed up the drug approval process?

It decreases the time of an FDA review of an NDA from 10 months to 6 months.

What are the four steps in rational drug design?

Medicinal Chemistry Lead Discovery Target Selection in vitro & in vivo Studies

put into place by U.S. Congress in 1974 that created a national commission to develop guidelines for human subject research

National Research Act

This is sent to the FDA and includes information on the safety, efficacy and proposed labeling of a new drug, such as an antidepressant.

New Drug Application (NDA)

Which government act allowed the FDA to begin collecting fees from drug sponsors that would be utilized to expand review staff so that new drugs could be reviewed more quickly?

PDUFA

Typically involves 20-100 healthy volunteers

Phase 1 clinical trials

This designation designation means the FDA's goal is to take action on an application within 6 months (compared to 10 months under standard review).

Priority review

A group of women with breast cancer are studied and found that a majority of the women had been exposed to second-hand smoke during their lifetime, which may have contributed to their cancer development. This is known as what type of study?

Retrospective study

Which of the following was created as a DIRECT response to research atrocities in the U.S. such as the Tuskegee syphilis studies?

The National Research Act

What is the name of the code that was written as a result of WWII and the experimentation on prisoners that the Nazis performed?

The Nuremburg Code

Which of the following is considered the most important document in the history of the ethics of medical research because it serves as a blueprint for today's principles and ensures the rights of subjects of medical research?

The Nuremburg Code

Written and adopted after WWII and the Nazi experiments; requires voluntary consent and has 10 ethical principles for clinical research

The Nuremburg Code

Prescription drugs need to first undergo experimentation in preclinical and clinical studies. Before a drug can enter clinical studies:

The drug must first be approved as an Investigational New Drug (IND)

Which of the following statements about a drug's formulation is TRUE?

The formulation determines how long a drug will last. A drug's formulation is also known as the drug's dosage form. A drug's formulation determines how fast the drug will work.

What would NOT be a valid reason to exclude someone from a clinical trial?

The person is 85 years old and, in the investigators' personal opinions, older adults are not reliable patients

The FDA writes guidances and regulations for the drug industry, and takes into consideration pharmaceutical company input. If a company is found in violation of an FDA guidance, what could be their punishment?

While guidances are not legally binding, the company could be fined

Categorize each statement as either "Basic Science" or "Applied Science." a. fundamental scientific knowledge b. distinct goals and discoveries c. expanding understanding about processes in living organisms d. address specific medical issue e. development of new medication, medical device, or surgical procedure f. understanding how cells work

a. (BS) b. (AS) c. (BS) d. (AS) e. (AS) f. (BS)

Why are drug companies given market exclusivity? a. so they can try and recoup the losses from the drug discovery process b. because they are the only drug company capable of producing the drug c. because only one drug company is allowed to make a certain drug d. because they have a patent for the drug

a. so they can try and recoup the losses from the drug discovery process

3. Which of these does not fall under the FDA's jurisdiction? a. tryptophan (found in food) b. insulin c. tabaco products d. ibuprofen

a. tryptophan (found in food)

What does the acronym "GRASE" stand for?

a.) generally recognized as safe and effective

According to the Prescription Drug and User Fee Act, how much does a company have to pay to submit 1 NDA?

about $2.8 Million

Which statement is correct regarding pharmacodynamics and pharmacokinetics? a. Pharmacodynamics is the study of what the body does to the drug b. Pharmacokinetics is the study of what the body does to the drug c. Pharmacodynamics and pharmacokinetics are the same thing. d. Pharmacodynamics and pharmacokinetics are not related to the drug discovery process

b. Pharmacokinetics is the study of what the body does to the drug

14. Which of the following terms regards where the drug goes once absorbed? a. absorption b. distribution c. metabolism d. excretion

b. distribution

6. Which of the following statements regarding market exclusivity is FALSE? a. allows a company to try to recoup from losses during the drug discovery process b. is the same thing as a patent c. is specific to the United States and the FDA d. all of the above

b. is the same thing as a patent

On average it takes approximately how much money to bring a new molecule to market? a. $500 million b. $750 million c. $1.3 billion d. $2 billion

c. $1.3 billion

7. Approximately what percent of pharmaceutical spending is spent on clinical trials (phases 1-3)? a. 4% b. 15% c. 26% d. 33%

c. 26%

9. Significant changes in already approved products results in exclusivity to be extended for another...? a. 1 year b. 5 years c. 3 years d. 10 years

c. 3 years

2. Prescription drugs are all of the following except: a. Prescribed by a doctor b. Bought at a pharmacy c. Each regulated differently d. prescribed for and intended to be used by one person

c. Each regulated differently

What drug was withdrawn in 2004 due to unreported cardiovascular side effects? a. Oxycontin b. Zoloft c. Vioxx d. Adderall

c. Vioxx

Roughly 1/4 of the costs in drug discovery comes from...? a. bio screening b. toxicology c. clinical trials d. safety testing

c. clinical trials

11. Insulin was discovered using what animal's pancreas? a. horse b. goat c. dog d. pig

c. dog

12. An over the counter pregnancy test uses the antibodies of what 2 animals? a. pig and goat b. dog and pig c. goat and mouse d. dog and mouse

c. goat and mouse

Which phase of clinical trails have the highest fail rate? a. Preclinical b. Phase I c. Phase II d. Phase III

c. phase 2

4. Who initiates a clinical investigation? a. human subjects b. institutional review board (IRB) c. sponsor d. principal investigator

c. sponsor

Which is TRUE regarding guidances? a. they are legally binding b. pharmaceutical companies cannot be fined for not adhering c. they are strong suggestions from the FDA d. specify details and requirements necessary to implement and enforce legislation enacted by congress

c. they are strong suggestions from the FDA

Approximately how long does it take to bring a new molecule to market? a. 2-5 years b. 20-30 years c. 1-3 years d. 12-15 years

d. 12-15 years

16. Which doctor accidentally discovered penicillin? a. Charles Best b. Frederick Banting c. John McLeod d. Alexander Fleming

d. Alexander Fleming

13. Which of the following is a step of rational drug design? a. target selection b. medicinal chemistry c. lead discovery d. all of the above

d. all of the above

15. Which of the following is a method of excretion? a. urine b. saliva c. sweat d. all of the above

d. all of the above

7. Which of the following statements is TRUE regarding generic drugs? a. they are less expensive b. they are more cost effective c. they have the same active ingredient as the branded drug d. all of the above

d. all of the above

The formulation of a drug can effect...? a. how fast it works b. where it goes c. how long it lasts d. all of the above e. none of the above

d. all of the above

Why was the Tuskegee Sysphilis experiment unethical? a. Researchers did not tell the subjects the true purpose of the study b. Patients could not give informed consent c. Patient never received treatment d. All of the above

d. all of the above

8. Generic names stem from all of the following EXCEPT...? a. structure b. function c. molecular target d. color

d. color

5. Who would be held responsible if anything were to go wrong during a clinical trial? a. human subjects b. institutional review board (IRB) c. sponsor d. principal investigator

d. principal investigator

1. Which of these is false in regards to active ingredients? a. its the component of the medicine that provides the actual pharmacological activity b. it can be the component that has a direct effect on the diagnosis, cure, mitigation, treatment or prevention of a disease c. it can be the component that affects the structure or function of the body d. they are included to help stabilize the drug and may include flavorings, binding agents, or preservatives

d. they are included to help stabilize the drug and may include flavorings, binding agents, or preservatives

All of the following are true about human subjects in clinical studies but: a. subjects must volunteer to be a part of the study b. subjects cannot be forced to participate c. they are individuals who are or become a participant in research d. they don't have the option to drop out of the study at any time

d. they don't have the option to drop out of the study at any time

Which of the following about generic drug names is TRUE?

drug makers usually propose names according to their compound's chemical make-up

The FDA writes guidances and regulations for the drug industry, and takes into consideration pharmaceutical company input. Guidances are__________ and regulations are __________; companies who do not follow guidances or regulations are subject to __________.

not legally binding ; legally binding ; fines

Which of the following would be considered a surrogate endpoint?

progression-free survival

The FDA must evaluate the NDA and weigh the effectiveness of the drug against the potential safety concerns. This is known as:

risk-benefit ratio