Drug Laws & Development

Question 1

What act allowed for the marketing of drugs for administration in uncommon disorders and tax credits?

Answer 1

1983 Orphan Drug Act

Question 2

A new molecule has been produced following cell culture studies. What is the next step in drug generation?

Answer 2

In vivo testing in animal models

Question 3

What is the risk of addiction for Schedule IV drugs?

Answer 3

Low

Question 4

How many refills are permitted with Schedule III - V drugs?

Answer 4

5x or 6 months

Question 5

What is the risk of addiction for Schedule III drugs?

Answer 5

Moderate

Question 6

When was the first attempt by the US to regulate drug production?

Answer 6

1848

Question 7

True/False. Only FDA approval is needed to begin clinical trials.

Answer 7

False. Most organizations have an IRB that must approve all research protocols, in addition to the FDA (when applicable)

Question 8

A physician prescribes their patient a drug to treat a disease not listed on the drug’s intended treatment. What is this an example of?

Answer 8

Off-label usage

Question 9

True/False. An IND must be submitted to the FDA when a physician wishes to use a drug for off-label usage.

Answer 9

False. So long as the intent is the practice of medicine, an individual provider does not need to submit an application to the FDA for off-label usage.

Question 10

In what clinical trials phase is the drug administered to patients with the target disease?

Answer 10

Phase 2

Question 11

What is the lifetime of a US drug patent?

Answer 11

20 years - this begins from the original creation of the molecule/drug

Question 12

What schedule of controlled substances has the lowest risk of addiction?

Answer 12

Schedule V

Question 13

What caused the US to enact the Federal Food, Drug, & Cosmetic Act in 1938?

Answer 13

Manufacture of a drug that included anti-freeze, leading to 40 deaths

Question 14

What occurs in Phase 3 of clinical trials?

Answer 14

The drug is administered to a large number of patients across a wide area to determine effective dose information

Question 15

Drug efficacy was not addressed by any US laws until when?

Answer 15

1962 - Kefauver-Harris Amendments

Question 16

What is a BLA?

Answer 16

BLA is a biologics application. The application is submitted to the FDA for any biological that is introduced to the market. This includes antibodies, cytokines, proteins, etc. and not drugs

Question 17

How does an IND differ from an NDA?

Answer 17

An IND is submitted after the creation of the new drug to request approval to begin clinical trials. An NDA (New Drug Application) is submitted after clinical trials are ended in order to introduce the new drug to the market.

Question 18

What are the ethical guidelines for research as established by the Nuremberg Code?

Answer 18

1. Voluntary consent
2. Intended for the good of society
3. Design based on knowledge
4. Avoid unnecessary injury
5. No believed risk of death or disabling injury
6. Risk should not outweigh the benefits
7. Proper protections
8. Conducted by qualified persons
9. Subject can stop at any time
10. PI must be prepared to end the study at any time

Question 19

What is the only schedule drugs not permitted for medical use?

Answer 19

Schedule I

Question 20

What schedule drugs have the highest risk of addiction?

Answer 20

Schedule I & II

Question 21

When does an IND or BLA not need to be submitted to the FDA?

Answer 21

When the substance is not intended to support a new indication for use, support change in advertising, or support changes in dose or target population (generally, this includes educational research by universities and NGOs)

Question 22

True/False. Animal testing of new drugs is stopped once clinical trials begin.

Answer 22

False. Testing in animals continues to evaluate chronic and toxic effects of the drug.

Question 23

Phase 2 of clinical trials evaluates what aspects of the new drug?

Answer 23

Efficacy & safety

Question 24

What schedule drugs are not permitted any refills?

Answer 24

Schedule I & II

Question 25

What is an Investigational New Drug (IND)?

Answer 25

An IND is a drug approved by the FDA to begin clinical trials. This is after preclinical investigation shows pharmacological efficacy and low toxicity of the drug

Question 26

What is the purpose of breakthrough therapy?

Answer 26

To speed up delivery of limited drugs to the market that show exceptional results for patients

Question 27

Why may a drug produce an ADR not seen during Phase 1-3 of clinical trials?

Answer 27

In clinical trials, the patient population is homogenous, specialists administer treatment, the dose is continuously monitored, and subjects are not on other drugs. In the real world, these criteria are not always maintained.

Question 28

A researcher began clinical trials one month ago. Following preliminary results, the researcher wants to collect a blood sample from future participants for analysis. Is this permitted?

Answer 28

No. Once a study begins, the protocol cannot be altered in any way. If the researcher wants to collect blood samples, the new protocol must be approved and the study restarted.

Question 29

What are the for primary requirements for an effective clinical trial?

Answer 29

Careful planning, control group, double-blind format, randomization?

Question 30

True/False. Data shows the drugs undergoing faster review have more ADRs.

Answer 30

True.

Question 31

Young, healthy individuals are treated with a new drug to gather pharmacokinetic data (absorption, biotransformation, elimination). What phase is this clinical trial in?

Answer 31

Phase 1

Question 32

What is the role of healthcare providers in clinical trials?

Answer 32

Healthcare providers play an active role in Phase IV of clinical trials. This is after the drug is introduced to the market. Providers share information regarding ADRs with the FDA.