Description
This guidance provides information for industry, researchers, physicians, institutional review boards (IRBs), and patients about the implementation of FDA’s regulations on expanded access to investigational drugs for treatment use under an investigational new drug application (IND) (21 CFR part 312, subpart I), which went into effect on October 13, 2009. Since 2009, FDA has received a number of questions concerning implementation of the regulations. As a result, FDA is providing guidance in a question and answer format, addressing the most frequently asked questions. In a separate guidance, FDA provides answers to questions concerning the implementation of the regulation on charging for investigational drugs under an IND (21 CFR 312.8). Also in a separate guidance, FDA describes Form FDA 3926 (Individual Patient Expanded Access—Investigational New Drug Application (IND)) and the process for submitting expanded access requests for individual patient INDs.
Scope & Applicability
Product Classes
2drug or biological product not yet approved for marketing; Drugs used for treatment use under expanded access categories.; Drug used for treatment under expanded access; The product being provided through expanded access; Drugs provided under expanded access.
Regulated by CDER and CBER
Stakeholders
8Institutional Review Board providing study approvals
Responsible for qualifications, training, and trial conduct; Individual responsible for trial conduct and data governance at a site.; May delegate tasks but retains overall responsibility; Person responsible for the conduct of the clinical trial at a trial site; Responsible for trial conduct and participant safety; Responsible for trial conduct, data integrity, and investigational product management.; Individual responsible for trial conduct at a site and informing the institution.; maintaining
Pharmaceutical company developing the drug for marketing
Governs top dose in clinical studies
Allowed value for reporter qualification
Entity responsible for submitting applications under section 524B
A licensed physician who submits an expanded access IND and administers the drug.; Physician acting as a sponsor for an individual patient IND; An individual who both initiates and conducts a clinical investigation.; Role that may authorize expanded access protocols
Authorized to make individual patient expanded access submissions
Regulatory Context
Attributes
1Evaluation criteria for individual patient expanded access
Identified Hazards
Hazards
1New information that may change the benefit/risk ratio
Related CFR Sections (10)
- 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 →
- 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 →
- 21CFR312.305§ 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 →
- 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 →
- 21CFR312.310§ 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 →
- 21CFR312.8§ 312.8 Charging for investigational drugs under an IND.
(a) General criteria for charging.Read 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 →
- 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 →
Related Warning Letters (10)
- 2025-12-23
In Vivo Bioavailability-Bioequivalence Studies – Clinical
Maria A. Carballosa, M.D.
- 2025-12-09
Unapproved New Drug/Unlicensed Biological Product/Biologics License Application (BLA)
Celularity, Inc
- 2025-11-18
Sponsor/Investigator
Verdure Sciences, Inc.
- 2025-09-30
Clinical Investigator (Sponsor)
Pamela K. Den Besten, DDS, MS
- 2025-09-23
Clinical Investigator/Sponsor
Ralph A. DeFronzo, M.D.
- 2025-09-09
Clinical Investigator
Shirish M. Gadgeel, M.D.
- 2025-07-15
Clinical Investigator
Mark J. Savant, M.D
- 2025-07-01
Clinical Investigator
Peter Michael, M.D.
- 2025-06-10
Clinical Investigator (Sponsor)
American Behavioral Research Institute, LLC
- 2025-06-03
Bioresearch Monitoring Program
Amy Lightner, MD
See Also (8)
- Reporting Amount of Listed Drugs and Biological Products Under Section 510(j)(3) of the FD&C Act (Status: Final)
- Enhancing Participation in Clinical Trials — Eligibility Criteria, Enrollment Practices, and Trial Designs: Guidance for Industry (Status: Final)
- Study of Sex Differences in the Clinical Evaluation of Medical Products (Status: Draft)
- CHAPTER 48 - 7348.809 Bioresearch Monitoring (Status: Final)
- Preparation of Investigational New Drug Products (Human and Animal): Guidance for Industry (Status: Final)
- Formatting, Assembling and Submitting New Drug and Antibiotic Applications* (Status: Final)
- Nuclear Pharmacy Guideline Criteria for Determining When to Register as a Drug Establishment (Status: Final)
- Guidance for Industry: Exports Under the FDA Export Reform and Enhancement Act of 1996 : Guidance for Industry (Status: Final)