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U.S. Department of Health and Human Services

Drugs

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IND Applications for Clinical Treatment (Expanded Access): Overview

Expanded access, sometimes called "compassionate use," is the use of investigational new drug products outside of clinical trials to treat patients with serious or immediately life-threatening diseases or conditions when there are no comparable or satisfactory alternative treatment options. The rules and regulations related to expanded access are intended to improve access to investigational drugs for patients who may benefit from investigational therapies.

When considering an IND application for expanded access to an investigational product with the purpose of treating a patient or a group of patients, physicians and investigators should recognize that such applications would be suitable when all of the following criteria apply:

  • Patient(s) have a serious or immediately life-threatening disease or condition, and there is no comparable or satisfactory alternative therapy to diagnose, monitor, or treat the disease or condition;

  • The potential patient benefit justifies the potential risks of the treatment and the potential risks are not unreasonable in the context of the disease or condition to be treated; and

  • The expanded use of the investigational drug for the requested treatment will not interfere with the initiation, conduct, or completion of clinical investigations that could support marketing approval of the product. 

The party who (1) submits a request to open an expanded access IND application and (2) receives FDA’s authorization to use the investigational product is considered the sponsor of the IND application. In the absence of any other sponsor (e.g. pharmaceutical company), the treating physician is the sponsor of the expanded access IND application.

The Investigators’ Responsibilities described for sponsors of IND applications intended for clinical investigations also apply to sponsors of expanded access IND applications intended for clinical treatment with investigational products.

An expanded access IND application intended for IND use in non-emergency setting will go into effect 30 days after FDA receives the application or on earlier notification by FDA that the expanded access use may begin. Expanded access IND applications submitted for the purpose of clinical treatment in Emergency Setting may begin as soon as the use of investigational drug is authorized by an FDA reviewing official.

As other IND applications, expanded access IND applications may be placed by FDA on Clinical Hold.

FDA regulations allow access to investigational drugs for treatment purposes on a case-by-case basis for:

    • individual patients (single-patient IND applications for treatment in emergency settings and non-emergency settings)

    • intermediate-size patient populations (groups of patients, n >1)

    • larger populations for use of the drug under a treatment protocol or Treatment IND Application   

For detailed explanation of the regulations related to expanded access to investigational new drug products refer to Expanded Access

For answers to questions frequently asked about the expanded access to investigational drugs, refer to the following guidance documents: