When a physician would like to submit an Investigational New Drug application (IND) to obtain an unapproved drug for an individual patient, he or she should first ensure that the manufacturer of the unapproved drug is willing to provide the drug. If the manufacturer agrees to provide the drug, the physician should submit an IND to the appropriate review division.
In an emergency situation, the request to use the drug may be made via telephone or other rapid means of communication, and authorization to ship and use the drug may be given by the FDA official over the telephone. In these situations, known as emergency INDs, shipment of and treatment with the drug may begin prior to FDA’s receipt of the written IND submission that is to follow the initial request.
In a non-emergency situation, a written request (IND) for individual patient use of an investigational drug must be received by the FDA before shipment of and treatment with the drug may begin. These non-emergency requests are known as individual patient INDs. The IND should include the following information:
- Statement that this is a request for an individual patient IND for treatment use (specifying whether it is an emergency IND or individual patient IND) should be at the top of the correspondence and on the mailing cover.
- Brief Clinical History of the patient including:
- the diagnosis
- the disease status
- prior therapy
- response to prior therapy
- the rationale for requesting the proposed treatment, including a list of available therapeutic options that would ordinarily be tried before the investigational drug or an explanation of why use of the investigational drug is preferable to use of available therapeutic options.
- Proposed Treatment Plan including:
- the dose
- planned duration
- monitoring procedures
- modifications (e.g. dose reduction or treatment delay) for toxicity.
- Reference a published protocol or journal article if appropriate.
- Chemistry, Manufacturing, and Controls Information and Pharmacology and Toxicology Information, including a description of the manufacturing facility. The requirement for this information may be met by providing a Letter of Authorization (LOA) to refer to this information if it has been previously submitted to FDA (for example, to an existing IND or NDA). The treating physician should contact the sponsor of the previously submitted information for such authorization and letter. The letter of authorization should include relevant identifying information, such as the sponsor’s relevant application (e.g., IND) number.
- Informed Consent Statement that states that informed consent and approval of the use by an appropriate Institutional Review Board (IRB) will be obtained prior to initiating treatment. In the case of an emergency, treatment may begin without prior IRB approval, provided the IRB is notified of the emergency treatment within 5 working days of treatment.
- Investigator Qualification Statement that specifies the training, experience, and licensure of the treating physician. The first two pages of a Curriculum Vitae typically contain this information and are usually sufficient.
- FDA Form 1571 completed with the treating physician listed as the sponsor. You can download Form 1571 and other forms from the FDA Web site. For forms and instructions please see: Information for Sponsor-Investigators Submitting Investigational New Drug Applications (INDs).
- On February 4, 2015, the FDA released a draft guidance to facilitate streamlining the individual patient expanded access application process, Individual Expanded Access Applications: Form FDA 3926. Draft form FDA 3926 is a short form that will significantly decrease the amount time it takes to request expanded access for individual patients.
- Contact telephone number and facsimile number.
A number will be assigned to the application. The IND sponsor (treating physician) should provide this IND number to the drug supplier, so the supplier may ship the drug to the treating physician. The FDA will either allow the treatment use to proceed or not allow it to proceed (put the application on clinical hold). The IND is considered active (treatment with the drug may proceed) 30 days after FDA receives the IND submission or upon earlier notification of the physician by FDA. If the treatment use is not allowed to proceed (i.e., a clinical hold is placed on the application), FDA will notify the physician of this decision initially via a telephone call. The call will be followed by a written letter that provides the reasons for FDA's denial of the request.
For further information, please contact CDER’s Division of Drug Information (DDI) at phone: 301-796-3400 or 855-543-3784; fax: 301-431-6353; or e-mail: email@example.com or the appropriate CDER Review Division.
|CDER Review Division||Telephone Number||FAX Number|
|Division of Anti-Viral Products|
|Division of Oncology Products 1|
|Division of Oncology Products 2|
|Division of Hematology Products||301-796-7550||301-796-9849|
|Division of Reproductive and Urologic Products|
|Division of Medical Imaging Products|
|Division of Gastroenterology and Inborn Errors Products|
|Division of Anesthesia, Analgesia, and Addiction Products|
|Division of Metabolism and Endocrinology Products|
|Division of Pulmonary, Allergy and Rheumatology Products|
|Division of Dermatology and Dental Products|
|Division of Anti-Infective Products|
|Division of Transplant and Ophtalmology Products|
|Division of Cardiovascular and Renal Products|
|Division of Neurology Products|
|Division of Psychiatry Products|
Contact Information for Emergency Individual Patient INDs
During normal business hours (8 am – 4:30 pm EST weekdays)
For general questions about expanded access for emergency use for investigational drugs, contact CDER’s Division of Drug Information (DDI) at phone: 301-796-3400 or 855-543-3784; fax: 301-431-6353; or e-mail: firstname.lastname@example.org.
For questions about expanded access for emergency use for a specific investigational drug, contact the appropriate review division, if known, or DDI, if not known.
If DDI and the review division are not available, contact the CDER Emergency Coordinator (CEC) of the Counter-Terrorism and Emergency Coordination Staff (CTECS) at phone: 301-796-9900 or 301-796-2210; fax*: 301-431-6356; or e-mail at : email@example.com . * Please call or e-mail the CDER Emergency Coordinator before faxing documents.
After hours (after 4:30 pm EST weekdays and all day on weekends)
All questions about and requests for expanded access for emergency use for drugs, biologics and medical devices should be directed to the FDA Emergency Call Center, telephone: 866-300-4374.