How to apply for Emergency Treatment IND (Single Patient) access of an investigational drug:
If your HIV has evidence of resistance to all commercially available anti-retrovirals (ARVs) and your viral load suggests that your HIV is not responding to your current drug regimen, have a genotypic resistance test performed. If your doctor has not done so yet, also have a tropism test done.
Once the results from the test are available, there are a number of possible options:
1- Consider repeating drug-resistance testing, using a phenotypic assay. This may include the testing of virus sensitivity to Fuzeon.
2- If you have at least one active drug to which you may show sensitivity in your genotype or phenotype test, talk to your doctor about the advantages and disadvantages of entering any phase II/III studies
3- If your health is at risk (your CD4 is under 50 cells/ml), and you have developed resistance to all commercially available or expanded access HIV medications, and you and your doctor deem that you cannot risk being on virtual monotherapy by joining research studies, ask your doctor to:
a- Call the company that carries the investigational ARV that may help you to find out if they are willing to help you by providing free drug before it gets approved. GSK carries GSK572 (new integrase inhibitor that may work for many patients with raltegravir resistance) and Taimed carries ibalizumab ( an entry inhibitor ARV in a new class). Both drugs have gone through dosing and safety studies. For GSK572, they need to know your integrase resistance mutations, so make sure that information is included in your genotype/phenotype tests.
b- After having the manufacturer agree to provide the investigational drug via Single Patient IND, the doctor should follow the procedure described here to fill the required forms and get IRB (institutional review board) approval. This procedure is hardly used by doctors in HIV care due to lack of information or concerns about its complexity (it is a little time consuming but not too complex). The link which describes the entire procedure is
Three simple forms, a signed patient consent form and IRB (Institutional Review Board) approval are needed. Many locals IRBs expedite approval of this kind of request due to its urgency. Also, single patient IND can be approved verbally by the FDA if the patient has an expected survival of less than 30 days (this is called emergency IND) so that the drug company can ship the drug in an expedited manner, but the forms will still need to be processed while drug shipment is being processed.
For a sample consent form and cover letter for IRB submission go to:
Click here for Consent form (DOC)
Click here for Cover Letter (DOC)
Physician Request for an Individual Patient IND under Expanded Access for Non-emergency or Emergency Use
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).
- 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 the appropriate Review Division.
|CDER Review Division
Division of Anti-Viral Products