Drug Info Rounds Video: Expanded Access
Expanded access allows patients to use investigational drugs outside of clinical trials. FDA Drug Info Rounds pharmacists discuss the requirements that must be met before FDA can authorize Expanded Access and discuss the safeguards in place to avoid exposing patients to unnecessary risks.
CAPT Kremzner: Expanded access allows patients to use investigational drugs outside of clinical trials.
Hi, I’m Captain Mary Kremzner, and this is Drug Info Rounds, brought to you by the pharmacists in FDA’s Division of Drug Information.
I’m joined by two drug information specialists, Captain Catherine Chew and Lieutenant Lindsay Davison, to tell you more about expanded access to investigational drugs. Captain Chew, how would you describe Expanded Access?
CAPT Chew: Expanded access is the use of an investigational drug outside of a clinical trial. Because of the risks inherent to using an investigational drug outside of a clinical trial, many requirements must be met before FDA can authorize Expanded Access. The patient, their doctor, and the drug company must all be willing to participate.
LT Davison: Eligible patients are those with a serious or immediately life-threatening disease or condition who have no comparable or satisfactory alternative treatment options. In most circumstances, these patients should have already explored all available FDA-approved options to diagnose, monitor, or treat their disease or condition.
The patient’s physician must be willing to manage the use of the investigational drug. For individual patient expanded access, this includes a consideration of the risks and benefits; and in some cases, submitting the necessary paperwork to FDA, such as an Investigational New Drug application or IND.
Before the physician submits an IND, the drug company should be consulted to ensure they are willing to provide the drug product under these circumstances. These companies may not always be willing or able to provide access to a drug outside of the clinical trials intended to support marketing approval.
CAPT Kremzner: Let’s go over the safeguards in place to avoid exposing patients to unnecessary risks.
CAPT Chew: Safeguards in place include that FDA must determine that the potential benefit justifies the potential risks of treatment with the investigational drug. Federal regulations generally require that an Institutional Review Board review the use of the investigational drug to determine, among other things, that risks are minimized and reasonable, and there is appropriate informed consent.
LT Davison: Even with these safeguards, there may be significant unknowns. Investigational drugs may not have been approved by the FDA as safe and effective. These investigational drugs may be effective in the treatment of a disease or condition, or they may not. The risks associated with the use of the drug may also be unknown. Patients should understand and be willing to accept the possible risks and unknown effectiveness associated with the investigational drug.
CAPT Kremzner: Will you review the resources available for Expanded Access?
LT Davison: In some cases, companies have well-established expanded access programs that are easy to find. You can use ClinicalTrials.gov to view a list of expanded access studies or search for specific expanded access programs. Providers can also call or go online to inquire about expanded access programs a drug company might offer.
CAPT Chew: Patient advocacy groups can sometimes help patients find and explore expanded access options, and healthcare professionals can also visit FDA’s many online resources.
CAPT Kremzner: If you have questions about Expanded Access, call or email FDA’s Division of Drug Information.