As public health officials accelerate their efforts to develop a vaccine to combat Ebola—one that the U.S. Food and Drug Administration (FDA) has not yet approved for use—some may ask if there is ever an option for critically ill patients to gain access to unapproved drugs. These drugs can be obtained in very limited circumstances, but patients and their healthcare providers must clear a number of high hurdles.
Typically, an unapproved drug may only be administered through an FDA-approved clinical study in which patients meet certain criteria, and the drug is used in accordance with an approved protocol by a clinical investigator participating in the clinical trial. However, a few narrowly defined exceptions exist.
One of these exceptions is when a patient seeks "emergency use" of an unapproved drug. This exception is intended to benefit a single patient who is not eligible to participate in a study approved at the institution conducting the clinical trial. Typically, use of an unapproved drug requires an Investigational New Drug application (IND), approval by an institutional review board (IRB) and the patient's informed consent. But the "emergency use" exception provides means to expedite or bypass these requirements.
An IND is required under the "emergency use" exception, but an investigator can request an emergency IND through the FDA, which has 24-hour emergency contact information available on its website.
IRB approval can be expedited or waived if certain criteria are
satisfied. Regulations detailed at
21 C.F.R. §§ 56.104(c), 56.102(d) provide the
circumstances where IRB approval is not required:
- The situation is life-threatening;
- No standard acceptable treatment is available; and
- There is insufficient time to obtain IRB approval.
While notice of the "emergency use" may not be required in advance, some sponsors may require advance notice to the IRB or documentation from the IRB concerning the "emergency use." Additionally, the investigator must notify the IRB within five days after the drug's use.
"Emergency use" requires informed consent, but an exception is available here, too, in limited circumstances. Informed consent can be waived if both the investigator and a physician who is not otherwise directly involved with the patient's treatment or the clinical trial certify:
- The situation is life-threatening;
- Informed consent cannot be obtained because of an inability to communicate with or obtain a legally effective consent from the patient;
- There is insufficient time to obtain consent from the patient's legal representative; and
- There is not an alternative method of approved or generally recognized therapy providing an equal or better likelihood of saving the patient's life.
While "emergency use" of unapproved drugs is available, it is a narrow exception and few are likely to satisfy all the criteria required by the FDA. That said, it may be a critically important option to those few facing life-threatening situations. Healthcare providers may wish to consider this option when considering using an FDA unapproved product in the treatment of a patient infected with a life-threatening disease, such as the Ebola virus. Despite the high bar for satisfying all the criteria, the "emergency use" exception may be a viable means for seeking treatment under such dire circumstances.
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