7. US FDA Regulations

Question 1

What is an IND?

Answer 1

Investigational New Drug

Any UNAPPROVED drug including biological product being tested or used in a clinical research study

Includes using an existing drug for new indication that is not FDA approved

Question 2

What is an IDE?

Answer 2

Investigational Device Exemption

An exemption granted by the FDA to permit the investigational use of an unapproved device in human subjects by qualified clinical investigators

Question 3

What is a Significant Risk (SR) Device?

Answer 3

Means an investigational device that presents a potential for serious risk to the health, safety, welfare of a subject, because it is (at least one of the following):

1. Intended as an IMPLANT
2. Purported or represented to be for a use in supporting or sustaining human life
3. For a use of substantial importance in diagnosing, curbing, mitigating, or treating disease, or otherwise preventing impairment of human health

Question 4

Who is responsible for making the initial risk determination of a device?

Answer 4

SPONSOR

But this prelim determination can be challenged by IRB - subject to approval or review

Question 5

What is the definition of an investigator?

Answer 5

An individual who actually CONDUCTS a clinical investigation (ie under whose immediate direction an investigational drug is administered or dispensed to a subject)

Question 6

What is the definition of a sponsor?

Answer 6

An individual who takes responsibility for and INITIATES a clinical investigation. the sponsor does not actually conduct the investigation

Question 7

What is the definition of a sponsor-investigator?

Answer 7

Is an individual who both INITIATES and CONDUCTS a clinical research project and under whose immediate direction the investigational product is administered or dispensed to or used by subject

Question 8

What is the IRB responsible for?

Answer 8

1. Initial review and approval of proposed study
2. Continuing review and approval
3. Make SR device determination (DEVICE STUDY ONLY)

Question 9

What are the 7 general responsibilities of the SPONSOR

Answer 9

1. Select qualified investigators
2. Ensure proper monitoring
3. Ensure compliance with protocol
4. Proper record keeping
5. Maintain effective IND/IDE
6. Prompt reporting of SAEs to FDA and all participating PIs
7. Disposition of unused drug/device

Question 10

What are the 5 general responsibilities of the Investigator?

Answer 10

1. Conduct study according to signed investigator statement, protocol, and applicable regulations
2. Protect rights, safety, and welfare of subjects
3. Obtain informed consent
4. Control of the investigational drugs/devices
5. Assure IRB review and approval

(Financial disclosure)

Question 11

What conversation does the Investigator facilitate?

Answer 11

Link between sponsor and IRB. Sponsor speaks to IRB THROUGH Investigator.

Investigator talks to the IRB

Question 12

What conversation does the Sponsor facilitate?

Answer 12

Between the Investigator and FDA

Talks directly to the FDA

Question 13

How and what should the IRB document a SR/NSR determination?

Answer 13

In the meeting minutes

1. IRBs reason for an SR or NSR determination
2. The documentation used to establish IDE status (ex FDA letter of IDE approval or conditional approval)

Question 14

Is the submission of an Investigator’s Brochure or Device Manual to the IRB a regulatory requirement?

Answer 14

No, regulations DO REQUIRE submission of INFORMATION CONTAINED in the IB/DM

Question 15

What is the IRBs responsibility in media ads?

Answer 15

1. Lack of coercion or promise of certain cure
2. No claims of safety/effectiveness

Question 16

Guidelines for payment to subjects

Answer 16

Amount should be
1. Pro rated
2. Disclosed in the consent form (under recruitment incentive, NOT BENEFIT)
3. Not so high that it exerts undue influence

Question 17

When can Sponsors charge for an investigational drug or biological?

Answer 17

Only if it’s a clinical trial or treatment IND

Must be indicated in informed consent form

Written FDA approval required for clinical trials

30 day FDA notices required for treatment IND

Question 18

T/F IDE regulations allow for Sponsors to charge for an investigational device

Answer 18

True

Sponsor justifies the proposed charges for the device in the IDE application

FDA generally allows sponsors to charge INVESTIGATORS for investigational devices; cost usually passed on to the subjects

Question 19

Guidelines for emergency use of investigational drug/biologic

Answer 19

1. Requires an IND (and emergency one from the FDA)
2. If no approved protocol or subject ineligible contact manufacturer regarding emergency use under company IND
3. If no time, FDA may authorize shipment prior to IND submission (can approve without an established protocol)
4. Prospective IRB approval is NOT required (but need to submit for IRB within 5 days of starting)

Question 20

What are the 4 pre reqs of the Treatment IND?

Answer 20

1. Condition must be serious/life-threatening
2. No satisfactory alternative
3. Already under study, or trials completed
4. Sponsor actively pursuing marketing approval

Protocol is required for submission. Must go through full prospective IND application still.

Question 21

Does Treatment IND require IRB?

Answer 21

Yes requires prospective IRB approval (or waiver) and informed consent

IRB may opt to review study even if FDA grants waiver

Question 22

What are surveillance inspections?

Answer 22

Periodic scheduled inspections to review the overall operations and procedures of the IRB by the FDA

Question 23

What are direct inspections?

Answer 23

Unscheduled inspections generally result from a complaint, investigator noncompliance, or test article safety issues

Question 24

What documents of the IRB are reviewed during inspection?

Answer 24

1. IRB written procedures and guidelines
2. IRB meeting minutes and member roster
3. Documents submitted by the investigator and reviewed by the IRB

Question 25

T/F consent must be obtained by a licensed physician investigator for studies involving more than minimal risk and/or involving investigational drugs/devices

Answer 25

True

Question 26

When can a study for investigational drug or device begin?

Answer 26

When IDE obtained for device if SR

When IND obtained for drug

IRB can approve the study but cannot start until FDA ruling/paperwork down.