21 CFR Part 312 Investigational New Drug Application

Question 1

This is the Code of Federal Regulations that focuses on trials for pharmaceutical products.

Answer 1

21 CFR Part 312

Question 2

Clinical Investigation

Answer 2

Any experiment in which a drug is administered or dispensed to, or used involving, one or more human subjects.

Question 3

Contract research organization

Answer 3

a person that assumes, as an independent contractort with the sponsor, one or more of the obligations of a sponsor, e.g., design of a protocol, selection or monitoring of investigations, evaluation or reports, and preparation of materials to be submitted to the FDA.

Question 4

IND

Answer 4

Investigational new drug application

Question 5

Independent ethics committee (IEC)

Answer 5

A review panel that is responsible for ensuring the protection of the rights, safety, and well-being of human subjects involved in a clinical investigation and is adequately constituted to provide assurance of that protection.

Question 6

Investigational new drug

Answer 6

A new drug or biological drug that is used in a clinical investigation. Also includes a biological product that is used in vitro for diagnostic purposes.

Question 7

Investigator

Answer 7

An individual who actually conducts a clinical investigation (i.e., under whose immediate direction the drug is administered or dispensed to a subject). In the event an investigation is conducted by a team of individuals, the ____________ is the responsible leader of the team. Subinvestigator includes any other individual member of that team.

Question 8

Marketing application

Answer 8

An application for a new drug submitted under section 505(b) of the act or a biologics license application for a biological product submitted under the Public Health Service Act.

Question 9

Sponsor

Answer 9

A person who takes responsibility for and initiates a clinical investigation. Can be an individual or pharmaceutical company, governmental agency, academic institution, private organization, or other organization. Does not actually conduct the investigation,

Question 10

Sponsor-Investigator

Answer 10

An individual who both initiates and conducts an investigation, and under whose immediate direction the investigational drug is administered or dispensed.

Question 11

Subject

Answer 11

A human who participates in an investigation, either as a recipient of the investigational new drug or as a control. May be a healthy human or a patient with a disease.

Question 12

What is and is not on the label of an investigational new drug?

Answer 12

On the label: “Caution: New Drug - Limited by Federal (or United States) Law to Investigational Use”

Not on the label:

• Shall not bear any statement that is false or misleading
• Shall not represent the IND as safe or effective

Question 13

Promotion of investigational drugs.

Answer 13

• Shall not promote IND as safe or effective
• Precludes commercialization of the drug before it is approved for commercial distribution
• Sponsor may not commercially distribute or test market an IND
• A sponsor shall not prolong an investigation after finding that the results appear to establish sufficient data to support a marketing application.

Question 14

Charging for a Drug Under an IND - What sponsor must demonstrate

Answer 14

A sponsor must:

• Provide evidence has potential clinical benefit, provide significant advantage.
• Demonstrate data from trial would be essential to establish safety and effectiveness
• Demonstrate trial could not be conducted without charging because of cost of drug

Question 15

Charging for a Drug Under an IND - FDA

Answer 15

• Sponsor must justify amount and obtain written authorization from FDA
• FDA may withdraw authorization if charging is interfering with drug development
• FDA expanded access charging must not interfere with drug development

Question 16

Charging for a Drug Under an IND - Costs

Answer 16

-Charge is limited to number of subjects authorized to receive drug (expanded access)
-A sponsor may recover only the direct costs of making investigational drug available.
Direct Costs:
-Cost per unit to manufacturer of the drug
-Cost to acquire drug from another manufacturing source
-Cost to ship and handle
Indirect Costs:
-Primarily to produce drug for commercial sale