Treatment of Patients with Investigational Agents
When a patient has a serious or life-threatening condition that is not addressed by current approved treatments, options may exist to use an investigational drug or device to treat. The use of an investigational product outside of a clinical trial for treatment of a patient is called “expanded access.” Most expanded access involves treatment of a single patient. However, some expanded access involves treatment protocols for multiple patients.
Single Patient Treatment Use
Use of an investigational drug or device in an individual patient is divided into 2 pathways; “Emergency use” and “Compassionate use”. Please see detailed guidance below for submission and approval of each of these pathways.
Emergency Use versus Compassionate Use
The emergency use pathway involves the following criteria. If all three criteria are not met, refer to the Compassionate Use Pathway:
- The patient has a life-threatening or serious disease or condition that needs immediate treatment; and
- No generally acceptable alternative treatment for the condition exists; and
- Because of the immediate need to use the drug/device, there is no time to use existing procedures to obtain FDA approval for the use.
*”Life-threatening” is defined as:
- The likelihood of death is high unless the course of the disease is interrupted;
- A disease or condition with a potentially fatal outcome, where the end-point is survival.
- The disease or condition causes major irreversible morbidity.
This guidance should be used by any physician who wishes to use an investigational drug, device or biologic in a non-emergency situation with a single patient. This means that there is NOT an immediate need to use the drug/device, AND there is time to use existing procedures to obtain FDA approval for the use. It is strongly recommended that submission to the FDA and IRB occur simultaneously to improve the efficiency of the submission process.
The compassionate use pathway involves the following criteria. If the criteria are not met, expanded access is likely not appropriate for the patient.
- The patient has a life-threatening or serious disease or condition; and
- No generally acceptable alternative treatment for the condition exists.
Documentation Required for Single Patient Expanded Access
Single patient expanded access submissions may be submitted via HSERA or via e-mail. In all submissions, please ensure that your submission does not contain any patient PHI. Please utilize patient initials on study documents or redact documentation. The following documents are required for approval:
Expanded Access Application
Available on the tools and templates page.
Drugs/Biologics Only: FDA form 3926
NOTE: Box 10b MUST be checked; otherwise the request will have to wait for convened board review)
Drugs/Biologics Only: Investigator’s Brochure / Package Insert
Devices Only: Device Brochure / Manual
Devices Only: Documented independent assessment from an uninvolved physician
Unsigned consent form
If a consent form is not available from the manufacturer, please utilize the Single Patient Expanded Access Consent Form (available on the tools and template page)
Regulatory documentation from FDA for the IND or IDE
OR notice that documentation is pending receipt from FDA and will be provided to the IRB upon receipt.
A complete list of documents being submitted for review (document name, version #, date)
The following are also required with your submission when applicable:
- Clinical Protocol (i.e., if available from and provided by manufacturer)
- Manufacturer authorization for the treating physician to utilize the device / drug (when required)
- If appropriate: Reference a published protocol or journal article to support proposed treatment.
Expanded Access for More Than One Patient
If an investigator anticipates more than one patient may benefit from the expanded access of a drug or device, an Intermediate-size patient population expanded access protocol, or a treatment protocol should be submitted through HSERA. These require convened review. Please see below for additional guidance regarding these types of requests.
Drugs Only: Intermediate-Size Patient Populations
Access to an investigational drug (including a biologic) for use by more than one patient, but generally fewer patients than are treated under a typical treatment protocol.
Treatment Protocol for Drugs or Devices
Access to an investigational drug or device for treatment use by a large (widespread) population, submitted as a protocol.
- FDA Regulations: §812.36, §812.47, §50.24
- FDA’s Expanded Access Program
- FDA Information Sheets for IRBs and Investigators
- Expanded Access to Investigational Drugs for Treatment Use—Questions and Answers
- IND Applications