After a drug manufacturer has agreed to provide an investigational drug, the next step is to obtain FDA authorization. It is a good idea to call the FDA Review Division and describe your patient’s situation prior to sending a written application. An FDA official will help you determine whether your patient’s condition requires you to follow a non-emergency (Individual Patient IND) or emergency (Emergency IND) pathway, which is used when treatment of the patient must occur within a very limited number of hours or days. FDA authorizes compassionate use in 99.5% of cases.
Your steps will be:
• Call FDA’s drug review division to discuss your planned submission.
• Send required paperwork to FDA by fax or email, followed by hard copy.
• Obtain informed consent and IRB approval.
• Receive an IND number from FDA.
• Submit this IND number to the manufacturer.
• Receive the drug from the manufacturer and begin treatment.
Application paperwork
1. Form 3926, Individual Patient Expanded Access Investigational New Drug Application (IND). Expanded Access Explained. It should take about 45 minutes to complete. Form 1571 and Instructions for Form 1571, and Form 1572 should be used for submission of an Individual Patient Protocol, as well as treatments INDs and protocols.)
2. Letter of Authorization from the drug company.
3. A cover letter specifying that this is a request for an individual patient IND or an emergency IND and your contact information.
4. A copy of the informed consent.
The information required on Form 3926 includes:
a. Brief clinical history of the patient including:
• patient’s age, gender, weight, diagnosis,
• disease status,
• prior therapy and response to prior therapy,
• rationale for requesting the proposed treatment. It can include 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.
b. Proposed treatment plan including:
• the dose,
• route of administration,
• planned duration,
• monitoring procedures and modifications (e.g., dose reduction or treatment delay) for toxicity.
c. Name of the manufacturer (drug supply reference statement) and a statement that a Letter of Authorization to cross-reference an appropriate IND of the supplier or Drug Master File (DMF) of the manufacturer is included.
d. Informed consent statement that states that informed consent and approval of an appropriate Institutional Review Board (IRB) will be obtained prior to initiating treatment.
e. 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.
Receive FDA approval
In practice, FDA will review the paperwork for oncology patients seeking compassionate use in about 2 business days, although by law it has up to 30Â days to review the application. If the request is approved, an IND number will be issued by the FDA and you will be contacted by phone or fax with a letter to follow. It will contain the name and telephone number of the FDA official to whom questions about the application should be directed.
If the unlikely situation when the treatment is not allowed to proceed (i.e., a clinical hold is placed on the application), FDA will notify you by phone and in writing.
Contact information for FDA
The initial request may be made by fax to the appropriate drug review division with hard copies of documentation to follow.
For questions about expanded access for a specific investigational drug, contact the appropriate review division, if known, or CDER’s Division of Drug Information (DDI), if not known, at phone:
301-796-3400 or 855-543-3784; fax: 301-431-6353; or e-mail: druginfo@fda.hhs.gov.
For questions about expanded access for emergency use for investigational biologics, contact CBER’s Office of Communication, Outreach, and Development at phone:
240-402-7800 or 800-835-4709; or e-mail: industry.biologics@fda.gov.
For questions about non-emergency individual expanded access for biologics, contact CBER at:
240-402-8010 or 800-835-4709.
Mailing addresses (courier service can be used)
For a drug — An original and two photocopies:
Food and Drug Administration
Center for Drug Evaluation and Research
Central Document Room
5901-B Ammendale Rd.
Beltsville, MD 20705-1266
For a biologic product — An original application and a CD with a copy:
Food and Drug Administration
Center for Biologics Evaluation and Research
Document Control Center
10903 New Hampshire Avenue
WO71, G112
Silver Spring, MD 20993-0002
Emergency requests
Your application process will be quicker if the patient needs the drug on an emergency basis because you will be able to proceed before submitting paperwork to FDA or an IRB. FDA defines an emergency as a case in which:
• the physician considers the product is immediately needed for the patient’s serious or life-threatening condition;
• no satisfactory alternative standard therapy is available;
• the patient cannot receive the product through any existing clinical trials or expanded access protocols;
• access to the drug is needed before the written application can be submitted (approximately within the next 24-72 hours).
Your steps will be:
• Call FDA at the emergency numbers below.
• Receive an IND number from FDA.
• Submit this IND number to the manufacturer.
• Obtain informed consent.
• Notify an IRB of planned emergency use if there is no time to obtain IRB approval.
• Receive the drug from the manufacturer and begin treatment.
• Apply for IRB approval within 5 days of the beginning of treatment.
• Submit required paperwork to FDA within 15 days of the beginning of treatment.
About 30% of compassionate use requests FDA receives are considered emergency requests.
Emergency contact information at FDA
Emergency requests may be submitted over the phone, by email or by fax. You will need to explain your patient’s situation and agree to submit the required paperwork within 15 working days. An FDA official may provide verbal authorization for compassionate use over the phone.
• During normal business hours contact Center for Drug Evaluation and Research, Division of Drug Information (DDI) (8am-4:30pm EST weekdays):
Phone: 301-796-3400 or 855-543-3784
Fax: 301-431-6353
E-mail: druginfo@fda.hhs.gov.
• After hours – or if you can’t reach anyone at DDI – contact the Emergency Call Center (after 4:30pm EST weekdays and all day on weekends) at:
1- 866-300-4374 or 301-796-8240.
 Use this checklist to make sure FDA requirements have been fulfilled.
Submit IND number to drug manufacturer and await shipment of the drug
Upon receipt of the IND number from the FDA, you must then submit the IND number to the manufacturer, and the company will ship the drug directly to you. The IND is considered active as soon as the number is issued.
Print all application steps
as one document (PDF)
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