Description
This guidance provides information for industry, researchers, physicians, institutional review boards, and patients about the implementation of FDA’s regulations on expanded access to investigational drugs for treatment use under an investigational new drug application (21 CFR part 312, subpart I), which went into effect on October 13, 2009. FDA received numerous questions concerning implementation of the regulatory requirements for expanded access. As a result, FDA issued the guidance for industryExpanded Access to Investigational Drugs for Treatment Use: Questions and Answers(June 2016, updated October 2017), providing recommendations in a question-and-answer format, addressing the most frequently asked questions. Since 2017, FDA has received additional questions concerning implementation of the regulatory and statutory requirements of expanded access to investigational drugs, including those added by the 21st Century Cures Act and the FDA Reauthorization Act of 2017.
Scope & Applicability
Product Classes
1drug 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.
Stakeholders
10Governs top dose in clinical studies
Clinical staff member responsible for medical care and informing the patient of risks.
Person authorized to provide consent on behalf of the patient if needed.
individual receiving expanded access treatment; The individual receiving the investigational treatment and providing consent.
Entity involved in investigational drug supply chain
Entity required to conduct postmarket surveillance; entity responsible for submitting postmarket surveillance plans; Entity holding the approved PMA, HDE, or cleared 510(k); The entity responsible for conducting postmarket surveillance and submitting reports.; Entity that can use the checklist as a reference for submissions.
Entity responsible for submitting applications under section 524B
Allowed value for reporter qualification
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
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
Regulatory Context
Attributes
6Waiting period before an IND goes into effect
reasonably foreseeable risks or side effects
Time required before treatment may begin after submission
Evaluation used to determine if expanded access is appropriate
Timeframe to report emergency use to IRB
Timeframe to submit written IND after emergency authorization
Related CFR Sections (20)
- 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 →
- 21CFR312.21§ 312.21 Phases of an investigation.
An IND may be submitted for one or more phases of an investigation. The clinical investigation of a previously untested drug is generally divided into three phases. Although in general the phases are conducted sequentially, they may overlap. These three phases of an investigation are a follows:Read full regulation →
- 21CFR312.7§ 312.7 Promotion of investigational drugs.
(a) Promotion of an investigational new drug. A sponsor or investigator, or any person acting on behalf of a sponsor or investigator, shall not represent in a promotional context that an investigational new drug is safe or effective for the purposes for which it is under investigation or otherwise pRead full regulation →
- 21CFR201.128§ 201.128 Meaning of “intended uses”.
The words intended uses or words of similar import in §§ 201.5 , 201.115 , 201.117 , 201.119 , 201.120 , 201.122 , and 1100.5 of this chapter refer to the objective intent of the persons legally responsible for the labeling of an article (or their representatives). The intent may be shown by such peRead 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 →
- 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 →
- 21CFR312.40§ 312.40 General requirements for use of an investigational new drug in a clinical investigation.
(a) An investigational new drug may be used in a clinical investigation if the following conditions are met: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 →
- 21CFR312.320§ 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 →
- 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 →
- 21CFR50.3§ 50.3 Definitions.
As used in this part:Read 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 →
- 21CFR312.8§ 312.8 Charging for investigational drugs under an IND.
(a) General criteria for charging.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 →
- 21CFR312.3§ 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 →
- 21CFR312.315§ 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 →
- 21CFR312.32§ 312.32 IND safety reporting.
(a) Definitions. The following definitions of terms apply to this section:Read 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 →
- 21CFR312.62§ 312.62 Investigator recordkeeping and record retention.
(a) Disposition of drug. An investigator is required to maintain adequate records of the disposition of the drug, including dates, quantity, and use by subjects. If the investigation is terminated, suspended, discontinued, or completed, the investigator shall return the unused supplies of the drug tRead full regulation →
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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)