Description
This guidance provides recommendations to institutional review boards (IRBs) and clinical investigators regarding the key factors and procedures IRBs should consider when reviewing individual patient expanded access submissions, including for reviews conducted by a single member of the IRB, to fulfill its obligations under 21 CFR part 56. Although FDA has issued guidance on expanded access requests, including expanded access for individual patients, the Agency is aware that IRBs seek further clarity on this topic.
Scope & Applicability
Product Classes
2Subject of expanded access submissions.
Requires analytical comparability per ICH Q5E
Stakeholders
5Governs top dose in clinical studies
Veterinarian responsible for selecting anesthetic regimens and conducting field studies
Typically submit individual patient expanded access requests; The individual responsible for treating the patient under expanded access.; Healthcare provider seeking information for patients; doctor or clinical staff whom the patient can contact
responsible for justifying omission of studies
Position title used to identify who carries out specific duties
Regulatory Context
Attributes
1Specific population requiring additional protections
Related CFR Sections (13)
- 21CFR10.115§ 10.115 Good guidance practices.
(a) What are good guidance practices? Good guidance practices (GGP's) are FDA's policies and procedures for developing, issuing, and using guidance documents.Read full regulation →
- 21CFR312.310§ 312.310 Individual patients, including for emergency use.
Under this section, FDA may permit an investigational drug to be used for the treatment of an individual patient by a licensed physician.Read full regulation →
- 21CFR312.315§ 312.315 Intermediate-size patient populations.
Under this section, FDA may permit an investigational drug to be used for the treatment of a patient population smaller than that typical of a treatment IND or treatment protocol. FDA may ask a sponsor to consolidate expanded access under this section when the agency has received a significant numbeRead full regulation →
- 21CFR312.320§ 312.320 Treatment IND or treatment protocol.
Under this section, FDA may permit an investigational drug to be used for widespread treatment use.Read full regulation →
- 21CFR312.300§ 312.300 General.
(a) Scope. This subpart contains the requirements for the use of investigational new drugs and approved drugs where availability is limited by a risk evaluation and mitigation strategy (REMS) when the primary purpose is to diagnose, monitor, or treat a patient's disease or condition. The aim of thisRead full regulation →
- 21CFR312.305§ 312.305 Requirements for all expanded access uses.
The criteria, submission requirements, safeguards, and beginning treatment information set out in this section apply to all expanded access uses described in this subpart. Additional criteria, submission requirements, and safeguards that apply to specific types of expanded access are described in §§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 →
- 21CFR56.103§ 56.103 Circumstances in which IRB review is required.
(a) Except as provided in §§ 56.104 and 56.105 , any clinical investigation which must meet the requirements for prior submission (as required in parts 312, 812, and 813) to the Food and Drug Administration shall not be initiated unless that investigation has been reviewed and approved by, and remaiRead full regulation →
- 21CFR56.105§ 56.105 Waiver of IRB requirement.
On the application of a sponsor or sponsor-investigator, the Food and Drug Administration may waive any of the requirements contained in these regulations, including the requirements for IRB review, for specific research activities or for classes of research activities, otherwise covered by these reRead full regulation →
- 21CFR56.108§ 56.108 IRB functions and operations.
In order to fulfill the requirements of these regulations, each IRB shall:Read full regulation →
- 21CFR56.111§ 56.111 Criteria for IRB approval of research.
(a) In order to approve research covered by these regulations the IRB shall determine that all of the following requirements are satisfied:Read full regulation →
- 21CFR50.55§ 50.55 Requirements for permission by parents or guardians and for assent by children.
(a) In addition to the determinations required under other applicable sections of this subpart D, the IRB must determine that adequate provisions are made for soliciting the assent of the children when in the judgment of the IRB the children are capable of providing assent.Read full regulation →
- 21CFR50.25§ 50.25 Elements of informed consent.
(a) Basic elements of informed consent. In seeking informed consent, the following information shall be provided to each subject:Read full regulation →
Related Warning Letters (10)
- 2024-11-05
Institutional Review Board (IRB)
Armstrong County Memorial Hospital
- 2024-10-08
Institutional Review Board (IRB)
Louisiana State University Health Science Center IRB
- 2024-07-16
Bioresearch Monitoring Program/IRB
Massachusetts Institute of Technology MIT
- 2024-06-18
Clinical Investigator (Sponsor)
Angela D. Ritter, M.D.
- 2024-03-26
Institutional Review Board (IRB)
New York State Psychiatric Institute IRB
- 2024-01-09
Clinical Investigator
Jeffrey W. Taub, M.D./Children's Hospital of Michigan
- 2023-09-29
Clinical Investigator
Luis Javier Pena-Hernandez, M.D., FCCP
- 2023-01-24
Bioresearch Monitoring Program/Institutional Review Board (IRB)
Larkin Community Hospital Institutional Review Board
- 2022-03-15
Clinical Investigator (CI)
Sabine S. Hazan, M.D.
- 2022-02-15
Clinical Investigator
Richard J. Obiso, PhD dba Avila Herbals, LLC
See Also (8)
- IRB Review of Stand-Alone HIPAA Authorizations Under FDA Regulations: Guidance for Industry (Status: Final)
- Acceptability of Draft Labeling to Support Abbreviated New Drug Application Approval; Guidance for Industry (Status: Draft)
- DSCSA Implementation: Product Tracing Requirements for Dispensers — Compliance Policy (Revised) Guidance for Industry (Status: Final)
- Early Clinical Trials With Live Biotherapeutic Products: Chemistry, Manufacturing, and Control Information: Guidance for Industry (Status: Final)
- Determining Donor Eligibility for Autologous Donors of Blood and Blood Components Intended Solely for Autologous Use - Compliance Policy: Guidance for Industry (Status: Final)
- Guidance for Industry: Use of the Term “Healthy” in the Labeling of Human Food Products (Status: Final)
- Revised Recommendations for Determining Eligibility of Donors of Human Cells, Tissues, and Cellular and Tissue-Based Products Who Have Received Human-Derived Clotting Factor Concentrates: Guidance for Industry (Status: Final)
- Guidance for Industry: Application of the Foreign Supplier Verification Program Regulation to the Importation of Live Animals (Status: Final)