Sponsor Investigator Studies

This section should help guide an investigator looking into using an FDA-unapproved drug, device, or biologic in a clinical investigation with human subjects.   

Definitions

The FDA has the following definitions under 21 CFR 312.3 and 21 CFR 812.3:

Clinical investigation means any experiment in which a drug is administered or dispensed to, or used involving, one or more human subjects. For the purposes of this part, an experiment is any use of a drug except for the use of a marketed drug in the course of medical practice.

IND means an investigational new drug application.

Investigational new drug means a new drug or biological drug that is used in a clinical investigation. The term also includes a biological product that is used in vitro for diagnostic purposes. The terms "investigational drug" and "investigational new drug" are deemed to be synonymous for purposes of this part.

Investigational device means a device, including a transitional device, that is the object of an investigation.

Investigator means an individual who actually conducts a clinical investigation (i.e., under whose immediate direction the drug is administered or dispensed to a subject or under whose immediate direction the test article is administered or dispensed to, or used involving, a subject, or, in the event of an investigation conducted by a team of individuals, is the responsible leader of that team). 

Sponsor means a person who takes responsibility for and initiates a clinical investigation. The sponsor may be an individual or pharmaceutical company, governmental agency, academic institution, private organization, or other organization. The sponsor does not actually conduct the investigation unless the sponsor is a sponsor-investigator. A person other than an individual that uses one or more of its own employees to conduct an investigation that it has initiated is a sponsor, not a sponsor-investigator, and the employees are investigators.

Sponsor-Investigator means an individual who both initiates and conducts an investigation, and under whose immediate direction the investigational drug or device is administered or dispensed. 

Monitor, when used as a noun, means an individual designated by a sponsor or contract research organization to oversee the progress of an investigation. The monitor may be an employee of a sponsor or a consultant to the sponsor, or an employee of or consultant to a contract research organization. Monitor, when used as a verb, means to oversee an investigation.

Background

When using a drug or device for research, a principal investigator should consider if the drug or device is FDA approved for the proposed use. If not, the use of the drug or device for research may need a special FDA authorization called an Investigational New Drug (IND) or Investigational Device Exemption (IDE), respectively.

An IND is required if a drug (or biologic) is not FDA approved and an IND may be needed if approved for a different indication or in a different patient population.  For more information, please consult the FDA website at this link.

An IDE is required if a device is not FDA approved and an IDE may be needed if approved for a different indication or in a different patient population.   A device which is determined to be a non-significant risk device generally does not need an IDE submission to the FDA, but it is considered to have an abbreviated IDE once the protocol and risk assessment are approved by the IRB. For more information, consult the FDA website at this link.

Investigators who hold an IND or IDE are considered to be Sponsor-Investigators (S-I) and have responsibilities both as the investigator and as the sponsor. In particular S-Is have additional FDA regulatory, record keeping and safety reporting requirements.

The IRB works closely with the Office of Research Integrity and Compliance (ORIC) to ensure that Emory S-Is are compliant with federal regulations and Emory IRB requirements. ORIC provides educational resources and is available to assist investigators in determining which regulations apply to their research.

When the IRB identifies a study as one having an Emory investigator as S-I, the S-I will be referred to OC for training about specific S-I responsibilities. The S-I will be requested to complete Emory S-I IDE or IND Responsibility Checklists. The ORIC website contains Emory S-I IDE or IND Responsibility Checklists and other important information about IND or IDE submissions under the Drug and Device sections, respectively.

Submitting a S-I study to the IRB

The Emory IRB requires SIs to provide the IRB with the following:

  • IND or IDE number assigned by the FDA and FDA IND or IDE ‘may proceed’ letter or documentation that establishes that the drug is exempt from IND requirements or that the device is non-significant risk or exempt from IDE
    • A study can be reviewed by the IRB analyst and placed on a Full Board agenda while the IND or IDE is pending. If the IRB does not receive the IND or IDE may proceed letter or information from the FDA indicating the date of the IND or IDE submission (to verify that more than 30 days have passed), the study will be removed from the agenda. 
  • Completion of S-I training
  • Completion of IND or IDE S-I Responsibility Checklist,
  • Protocol with adequate Data and Safety Monitoring Plan (DSMP) and site monitoring (more details below).
Other study requirements per current Emory policies and procedures.

Data Safety Monitoring Plan (DSMP) and Study Monitoring

All studies need a DSMP.  If a research protocol involves an increased level of risk, a Data and Safety Monitoring Board (DSMB) may be required to ensure that subjects are protected, reporting requirements are met, and that the data is kept confidential. For more information about DSMBs, follow this link.

Sponsors and S-Is are responsible for site monitoring to ensure that the investigation is conducted according to the investigational plan & protocol.  Site monitoring is an essential part of study oversight and is not done by a DSMB. Monitoring demonstrates that the S-I is taking steps to ensure the adequate protection of the rights, welfare, and safety of human subjects and the quality of the clinical trial data submitted to FDA1. The IRB will review the DSMP and site monitoring plan and may consider a monitoring plan which utilizes self- monitoring adequate for a study conducted only at Emory.

Multi-site Studies

If the S-I has more than one site (making him/her a sponsor at an external site), a CRO or other monitoring alternatives may be required before IRB approval. For an overview of monitoring methods and more information, please go to this FDA guidance. ORIC is available to provide assistance with the development of a monitoring plan.

S-I IDE Responsibilities Checklist Multi-site Trials

S-I IND Responsibilities Checklist Multi-site Trials

 

After IRB submission

When all S-I requirements are met, the study will be placed on a Full Board Meeting agenda for review.

Continuing Review

At the continuing review, the IRB will request that the Sponsor/S-I complete the appropriate Emory Sponsor-Investigator Responsibility Checklist Continuing Review Update and forward to the ORIC. ORIC will make the IRB aware of significant concerns with S-I processes.

Modifications to add non-Emory study sites or change sponsor and/or S-I

Modification may require that the Sponsor/S-I complete S-I training, the appropriate Emory Sponsor-Investigator Responsibility Checklist (if changing Investigator or Sponsor), and/or Multi-Site trials information checklist(s) (if adding a non-Emory site when Emory IRB is the IRB of record).