Expanded Access Use of Investigational Drugs and Biologics
For drug products, expanded access is defined as "the use of an investigational drug when the primary purpose is to diagnose, monitor, or treat a patient rather than to obtain the kind of information about the drug that is generally derived from clinical trials." See https://www.fda.gov/media/85675/download.
Under the FDA's current regulations, there are three categories of expanded access for investigational drugs and biologics:
- For individual patients (single patient), including for emergency use (21 CFR 312.310)
- For intermediate-size patient populations (>1 and generally smaller than those typical of a treatment IND or treatment protocol) (21 CFR 312.315)
- For widespread use (commonly referred to as treatment use) (21 CFR 312.320)
For each category of expanded access noted above, there are two different types of regulatory pathways that can be followed for FDA review:
- An expanded access protocol submitted to the FDA as a protocol amendment to an existing IND (i.e., an expanded access protocol) or
- A new IND submission, which is separate and distinct from any existing INDs and is intended only to make a drug available for treatment use (i.e., an expanded access IND).
Note that 21 CFR 312 Subpart D (Responsibility of Sponsors and Investigators) is applicable to expanded access use of investigational drugs. Please contact the IAP in the CTO if you are submitting an application request to the FDA.
For additional information, see FDA Guidance on Expanded Access to Investigational Drugs for Treatment Use at https://www.fda.gov/media/85675/download and the Expanded Access Navigator http://navigator.reaganudall.org
The following is a quick guide to the submission of expanded access use of drugs and biologics:
Requirements for Expanded Access Submissions of Drugs and Biologics
- FDA Submission
(contact CTO IAP) - Single Patient
- Cover letter
- Form FDA 3926
- CV of treating physician
- Medical history of patient
- Proposed treatment plan
- Informed consent form
- Investigator's Brochure (IB)
- Letter of Authorization (LOA)
- Intermediate Population or Treatment (Widespread)
- Cover letter
- Form FDA 1571
- Form FDA 1572
- CV of investigator
- Description of population to treat
- Treatment protocol
- Informed consent form
- Investigator's Brochure (IB)
- Letter of Authorization (LOA)
- Contractual Requirements
(contact CTO Contracts) - Single Patient
- CDA/NDA
- Agreement (may not be needed)
- Review of ICF (confidentiality, subject injury and compensation)
- Intermediate Population or Treatment (Widespread)
- CDA/NDA
- Agreement
- Review of ICF (confidentiality, subject injury and compensation)
- Research Pharmacy
- Single Patient
- Completion of Cost Estimate Form
- Intermediate Population or Treatment (Widespread)
- Completion of Cost Estimate Form
- IRB Submission
- Single Patient
- Rascal submission for each patient
(A copy of the FDA submission should be uploaded into Rascal at the time of submission)
- Rascal submission for each patient
- Intermediate Population or Treatment (Widespread)
- Rascal submission for the population
(A copy of the FDA submission should be uploaded into Rascal at the time of submission)
- Rascal submission for the population
Updated February 2024