Investigational New Drug Applications Prepared and Submitted by Sponsor-Investigators

Description

The purpose of this guidance is to assist sponsor-investigators in preparing and submitting complete investigational new drug applications (INDs) to the Center for Drug Evaluation and Research (CDER) and the Center for Biologics Evaluation and Research (CBER) at the Food and Drug Administration (FDA). Sponsor-investigators seeking to do clinical research often do not have the regulatory knowledge or the resources to hire experts to help them with the IND submission process. Although not an exhaustive step-by-step instruction manual, this guidance highlights certain elements of this process to facilitate a sponsor-investigator’s successful submission of an IND. This guidance also discusses the IND review process and general responsibilities of sponsor-investigators related to clinical investigations.

Scope & Applicability

Regulatory Context

• 21CFR312.33§ 312.33 Annual reports.

  A sponsor shall within 60 days of the anniversary date that the IND went into effect, submit a brief report of the progress of the investigation that includes:Read full regulation →

• 21CFR312.32§ 312.32 IND safety reporting.

  (a) Definitions. The following definitions of terms apply to this section:Read full regulation →

• 21CFR312.57§ 312.57 Recordkeeping and record retention.

  (a) A sponsor shall maintain adequate records showing the receipt, shipment, or other disposition of the investigational drug. These records are required to include, as appropriate, the name of the investigator to whom the drug is shipped, and the date, quantity, and batch or code mark of each such Read full regulation →

• 21CFR312.110§ 312.110 Import and export requirements.

  (a) Imports. An investigational new drug offered for import into the United States complies with the requirements of this part if it is subject to an IND that is in effect for it under § 312.40 and:Read full regulation →

• 21CFR312.31§ 312.31 Information amendments.

  (a) Requirement for information amendment. A sponsor shall report in an information amendment essential information on the IND that is not within the scope of a protocol amendment, IND safety reports, or annual report. Examples of information requiring an information amendment include:Read full regulation →

• 21CFR312.30§ 312.30 Protocol amendments.

  Once an IND is in effect, a sponsor shall amend it as needed to ensure that the clinical investigations are conducted according to protocols included in the application. This section sets forth the provisions under which new protocols may be submitted and changes in previously submitted protocols maRead full regulation →

• 21CFR312.42§ 312.42 Clinical holds and requests for modification.

  (a) General. A clinical hold is an order issued by FDA to the sponsor to delay a proposed clinical investigation or to suspend an ongoing investigation. The clinical hold order may apply to one or more of the investigations covered by an IND. When a proposed study is placed on clinical hold, subjectRead full regulation →

• 21CFR50.24§ 50.24 Exception from informed consent requirements for emergency research.

  (a) The IRB responsible for the review, approval, and continuing review of the clinical investigation described in this section may approve that investigation without requiring that informed consent of all research subjects be obtained if the IRB (with the concurrence of a licensed physician who is Read full regulation →

• 21CFR312.22§ 312.22 General principles of the IND submission.

  (a) FDA's primary objectives in reviewing an IND are, in all phases of the investigation, to assure the safety and rights of subjects, and, in Phase 2 and 3, to help assure that the quality of the scientific evaluation of drugs is adequate to permit an evaluation of the drug's effectiveness and safeRead full regulation →

• 21CFR312.23§ 312.23 IND content and format.

  (a) A sponsor who intends to conduct a clinical investigation subject to this part shall submit an “Investigational New Drug Application” (IND) including, in the following order:Read full regulation →

• 21CFR312.3§ 312.3 Definitions and interpretations.

  (a) The definitions and interpretations of terms contained in section 201 of the Act apply to those terms when used in this part:Read full regulation →

• 21CFR312.55§ 312.55 Informing investigators.

  (a) Before the investigation begins, a sponsor (other than a sponsor-investigator) shall give each participating clinical investigator an investigator brochure containing the information described in § 312.23(a)(5) .Read full regulation →

• 21CFR56.104§ 56.104 Exemptions from IRB requirement.

  The following categories of clinical investigations are exempt from the requirements of this part for IRB review:Read full regulation →

• 21CFR312.6§ 312.6 Labeling of an investigational new drug.

  (a) The immediate package of an investigational new drug intended for human use shall bear a label with the statement “Caution: New Drug—Limited by Federal (or United States) law to investigational use.”Read full regulation →

• 21CFR25.40§ 25.40 Environmental assessments.

  (a) As defined by CEQ in 40 CFR 1508.9 , an EA is a concise public document that serves to provide sufficient evidence and analysis for an agency to determine whether to prepare an EIS or a FONSI. The EA shall include brief discussions of the need for the proposal, of alternatives as required by secRead full regulation →

• 21CFR312.10§ 312.10 Waivers.

  (a) A sponsor may request FDA to waive applicable requirement under this part. A waiver request may be submitted either in an IND or in an information amendment to an IND. In an emergency, a request may be made by telephone or other rapid communication means. A waiver request is required to contain Read full regulation →

• In Vivo Bioavailability-Bioequivalence Studies – Clinical

  Maria A. Carballosa, M.D.

  2025-12-23

• Unapproved New Drug/Unlicensed Biological Product/Biologics License Application (BLA)

  Celularity, Inc

  2025-12-09

• Sponsor/Investigator

  Verdure Sciences, Inc.

  2025-11-18

• Clinical Investigator (Sponsor)

  Pamela K. Den Besten, DDS, MS

  2025-09-30

• Clinical Investigator/Sponsor

  Ralph A. DeFronzo, M.D.

  2025-09-23

• Clinical Investigator

  Shirish M. Gadgeel, M.D.

  2025-09-09

• Clinical Investigator

  Mark J. Savant, M.D

  2025-07-15

• Clinical Investigator

  Peter Michael, M.D.

  2025-07-01

• Clinical Investigator (Sponsor)

  American Behavioral Research Institute, LLC

  2025-06-10

• Bioresearch Monitoring Program

  Amy Lightner, MD

  2025-06-03

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