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  3. Development & Approval Process | Drugs
  4. How Drugs are Developed and Approved
  5. Types of Applications
  6. Investigational New Drug (IND) Application
  7. IND Applications for Clinical Treatment: Contents and Format
  1. Investigational New Drug (IND) Application

IND Applications for Clinical Treatment: Contents and Format

An expanded access submission may be a new IND application or a Protocol Amendment to an existing IND application. Each expanded access submission is expected to be clearly identified as “Expanded Access Submission” with an applicable box in Section 12 on Form FDA 1571 (PDF - 830KB) checked.

Some information required for application submission may be supplied by referring to pertinent information contained in an existing IND application if the sponsor of the existing IND application for the same investigational product grants a right of reference to their application through a Letter of Authorization. A Letter of Authorization is not required when an expanded access protocol is part of an existing IND application.

An IND application submitted for the purposes of clinical treatment to obtain access to an investigational product is recommended to include all the elements described in the table below. There is no template for an expanded access IND application. However, if the organization of the table below is followed, it will help to assure that all the needed information has been submitted.
  

Cover Letter

(typically 1 page or less)

The Cover Letter is used for triaging and routing of an IND application within FDA and is expected to include the following:  

  • Submission Identifier: “Expanded Access Submission”

  • Brief explanation of the intended clinical use

  • Investigational new drug product’s name and proposed formulation

  • IND manufacturer’s name and contact information (if applicable)

  • Reference to an existing IND application (if applicable) 

The Cover Letter is typically addressed to the Director of the Review Division in the Office of New Drugs. The Cover Letter would be expected to be signed by the sponsor of the expanded access IND application or their legal representative.
Form 1571 
(PDF  - 830KB) 		 This form includes administrative information pertinent to the IND application submission. Instructions for completion (PDF - 151KB)
Form 1572
(PDF - 718KB) 		 This form represents Statement of the Investigator conducting clinical research under the IND application. For frequently asked questions, refer to Information Sheet Guidance for Sponsors, Clinical Investigators, and IRBs (PDF - 105KB). 
Table of Contents   The Table of Contents for an IND application is expected to be detailed enough to permit FDA reviewers to locate items in the application quickly and easily. It is helpful if location of information is provided by volume and page. 
Letter of Authorization  

A Letter of Authorization from the IND product’s manufacturer for the right of reference to the information contained in their existing IND application. Such information may include, for example, the following:

  • A description of the facility where the drug is manufactured

  • Chemistry, manufacturing, and controls information adequate to ensure the proper identification, quality, purity, and strength of the investigational drug

  • Pharmacology and toxicology information adequate to conclude that the drug is reasonably safe at the dose and duration proposed for expanded access use (ordinarily, information that would be adequate to permit clinical testing of the drug in a single patient or in a population of the size expected to be treated)
Clinical protocol

A clinical protocol for treatment of a single patient or a group of patients (i.e. intermediate-size and large-size patient populations) is expected to include:

  • Rationale for the intended use of the drug, including a list of available therapeutic options that would ordinarily be tried before resorting to the investigational drug or an explanation of why the use of the investigational drug is preferable to the use of available therapeutic options

  • Description of the disease or condition for the expanded access use. For single patient protocols, include patient’s recent medical history and previous treatments. For protocols intended to treat groups of patients, specify inclusion and exclusion criteria enabling to enroll patients for whom the proposed treatment would be appropriate.

  • Proposed method of administration of the drug, dose, and duration of therapy

  • Description of clinical procedures, laboratory tests, or monitoring necessary to evaluate the effects of the drug and to minimize its risks 
Informed Consent   A copy of the Informed Consent for the investigational treatment. 
Investigator’s Brochure  A copy of the Investigator’s Brochure, if it was not included in the materials granted for reference in the Letter of Authorization. 

Statement about Product Development 

In addition to the information described above, an expanded access IND application proposing to treat a group of patients should note whether the drug is being developed or is not being developed. If the drug is not being actively developed, the expanded access IND application sponsor is expected to explain why the drug cannot currently be developed for the expanded access use and under what circumstances the drug could be developed. If the drug is being studied in a clinical trial, the sponsor is expected to explain why the patients to be treated cannot be enrolled in the clinical trial and under what circumstances the sponsor would conduct a clinical trial in these patients.

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