U.S. Food and Drug Administration • Center for Drug Evaluation and Research
Access to Unapproved Drugs
Access to unapproved drugs can occur through many legal mechanisms, including enrolling in clinical trials, a special exception or compassionate exemption, an emergency Investigational New Drug (IND), and a Treatment IND.
One of the most common methods to have access to an unapproved drug is to enroll in a clinical trial. There are many sources of information about clinical trials. One of the best methods is to ask a physician who specializes in the care of cancer patients. There are two pages in this Oncology Tools site where there are listings of clinical trials, Regulatory Tools and Oncology Web Sites. These listings are provided as a convenience, and no FDA endorsement should be implied.. Other resources are the Cancer Information Service of the National Cancer Institute (NCI) at 1-800-4CANCER (1-800-422-6237) or visit the NCI online at Cancer.gov and look at the Clinical Trials section on the first page.
If the eligibility criteria in a study protocol are not suitable for a particular patient, it may still be possible to be treated according to the study protocol as a special exception (sometimes called compassionate exemption). Treating a patient as an exception is at the discretion of the investigator and sponsor, and usually requires filing extra paperwork, including sending a request to the FDA, modifying the consent form and obtaining permission from the local Institutional Review Board (IRB). The request letter should state the rationale for requesting the exception and provide a brief patient history. It is sent as general correspondence to the appropriate Investigational New Drug (IND) application. Once an investigator and sponsor agree, further advice may be obtained by contacting the FDA review division where the IND is filed. The drug supplier should be able to provide the name of the appropriate review division. FDA review divisions are prohibited from divulging proprietary information such as whether a sponsor has filed an IND or the status of an IND.
Not all protocols are listed on the internet lists, and if there is interest in a particular drug (or biological or device), contacting the sponsor or manufacturer directly may help locate a facility that can provide access.
Another alternative is for a physician to file a single patient or emergency IND directly with the FDA. This must be done with the cooperation and permission of the drug supplier. Follow these links for general procedures and guidelines, and instructions on formatting a request for a single patient IND, although it is generally helpful to get specific advice by contacting the FDA review division where the drug supplier's IND is to be filed. The drug supplier should be able to provide the name of the appropriate review division. FDA review divisions are prohibited from divulging proprietary information such as whether a sponsor has filed an IND or the status of an IND. Filing a single patient or emergency IND has the same responsibilities as any IND in that the local Institutional Review Board (IRB) must be notified and approve, and adverse events, clinical updates, and an annual report must be filed with the FDA.
Sponsors may not charge for unapproved drugs, although there may be charges by a treating facility for costs associated with the administration of the drug and the general care of the patient. There is an exception through a mechanism called a treatment IND. Under this program, a sponsor of a drug that has shown clinical promise and is currently under review by the FDA may charge for the drug during the review process if permission has been obtained from the FDA. The intent of the program is to reimburse a sponsor for costs associated with providing expanded access to the drug prior to commercial distribution.
FDA/Center for Drug Evaluation and Research