Expanded Access Categories for Drugs (Including Biologics)
For each of the three expanded access categories, there are two types of regulatory submissions that can be used: a new investigational new drug application (IND) or a protocol (treatment plan) submitted as a protocol amendment to an existing IND.
Individual Patient Access
Individual Patient Expanded Access IND (also referred to as a Single Patient IND):
Access to an investigational drug (including a biologic) for use by a single patient submitted as a protocol under a new IND. The investigational product may or may not be under development. Unless FDA notifies the sponsor that treatment may begin earlier, there is a 30-day waiting period before treatment with the drug may begin.
Individual Patient Expanded Access Protocol (also referred to as a Single Patient Protocol):
Access to an investigational drug (including a biologic) for use by a single patient submitted as a new protocol to an existing IND by the sponsor of the existing IND. Typically, several patients may follow the same protocol. The investigational product may or may not be under development. There is no 30- day waiting period before treatment with the investigational product may begin, but the protocol must be received by FDA and have approval by an Institutional Review Board (IRB) before treatment may begin.
Individual Patient Access in an Emergency (also known as emergency expanded access use and emergency access):
Please note that Emergency INDs and protocols are a subset of Individual Patient Access.
(1) Emergency IND: Individual Patient Access IND for Emergency use: Access to an investigational drug (including a biologic) for use by a single patient in an emergency situation (i.e., a situation that requires a patient to be treated before a written submission can be made) submitted as a protocol under a new IND. Treatment is initially requested and authorized by telephone or other rapid means of electronic communication, and may start immediately upon FDA authorization. The written submission (i.e., the individual patient expanded access IND) must be submitted within 15 business days of the telephone authorization.
(2) Emergency Protocol: Individual Patient Expanded Access Protocol for Emergency Use: Access to an investigational drug (including a biologic) for use by a single patient in an emergency situation (i.e., a situation that requires a patient to be treated before a written submission can be made) submitted as a new protocol to an existing IND by the sponsor of the existing IND. Treatment is initially requested and authorized by telephone or other rapid means of communication, and treatment may start immediately upon FDA authorization. The written submission (i.e., the individual patient expanded access protocol) must be submitted within 15 business days of the telephone authorization.
In an emergency situation where there is not sufficient time to secure IRB review prior to beginning treatment, the emergency use of the investigational drug must be reported to the IRB within 5 working days, as required under 21 CFR 56.104(c).
Intermediate-Size Patient Population Access
Intermediate-size Patient Population Expanded Access IND:
Access to an investigational drug (including a biologic) for use by more than one patient, but generally fewer patients than are treated under a typical treatment IND or protocol, submitted as a protocol under a new IND. The investigational product may or may not be under development for marketing. Unless FDA notifies the sponsor that treatment may begin earlier, there is a 30-day waiting period before treatment may begin.
Intermediate-size Patient Population Expanded Access Protocol:
Access to an investigational drug (including a biologic) for use by more than one patient, but generally fewer patients than are treated under a typical treatment IND or protocol, submitted as a protocol to an existing IND by the sponsor of the existing IND. The investigational product may or may not be under development for marketing. There is no 30-day waiting period before treatment with the investigational product may begin, but the protocol must be received by FDA and have IRB approval before treatment may begin.
An intermediate-size patient population protocol may also be requested to allow access to treatment with an approved drug (including a biologic) or a related product that is not available through marketing channels because of failure to meet the conditions of approval or a drug shortage, provided the drug and the patient meet the general criteria for expanded access as well as the criteria specific to use in an intermediate-size patient population.
Expanded Access for Widespread Use
Treatment IND:
Access to an investigational drug (including a biologic) for treatment use by a large (widespread) population, submitted as a protocol under a new IND. The investigational product must be under active development for marketing. Unless FDA notifies the sponsor that treatment may begin earlier, there is a 30-day waiting period before treatment may begin.
Treatment Protocol:
Access to an investigational drug (including a biologic) for treatment use by a large (widespread) population, submitted as a protocol to an existing IND by the sponsor of the existing IND. The investigational product must be under development for marketing. Unlike other access protocols submitted to existing INDs, there is a 30-day waiting period before treatment may begin, unless FDA notifies the sponsor that treatment may begin earlier.
Visit the Expanded Access (Compassionate Use) Main Page
Related Information
- 21 CFR part 312 subpart I
- FDA's Final Guidance: Expanded Access to Investigational Drugs for Treatment Use—Questions
- Expanded Access: Criteria for an Investigational Product to be Made Available For Widespread Use Under a Treatment Protocol or Treatment IND/ Treatment IDE
- Individual Patient Expanded Access Applications: Form FDA 3926