FAQs for Expanded Access & Compassionate Use
Not necessarily. Even if you meet the criteria, there may still be some obstacles, including:
- Your medical history may preclude you from taking the specific investigational drug unless the risk from your condition outweighs the risk from the drug.
- Your physician is not willing to manage the use of the therapy.
- The company may decline to allow you to access the drug. They may do so for reasons discussed below
So-called “Right-to-try” laws are state laws that allow terminally-ill patients to bypass the FDA Expanded Access Program and obtain experimental therapy (drugs, biologics, devices that have completed Phase 1 testing but have not been approved by FDA) directly from a manufacturer. Right-to-try is completely separate from the FDA Expanded Access Program and is not regulated or managed by the FDA. Because of this, there are few if any safety procedures in place to protect patients and monitor for adverse reactions to the experimental treatment. Moreover, because Right-to-try laws bypass the FDA, manufacturers are less likely to participate in them. In fact, to date, there has not been a successful use of Right-to-try to gain access to an experimental drug. By contrast, the FDA authorizes over 99% of the Expanded Access requests it receives.
Yes. The manufacturer is not required to offer the drug outside their clinical trials, and therefore may choose not to do so. For example:
- The company may not have enough of the drug available for all patients requesting expanded access and may either establish a lottery system to determine which patients will have treatment access or make the decision on a case-by-case basis.
- Oftentimes small companies do not have any extra product to give outside of the clinical trial, and expanded access requests would siphon away product from the clinical trials.
- Companies are also concerned about adverse events in patients not in the clinical trials affecting the FDA’s review.
- Companies are only allowed to charge to recoup certain research and development costs.
An institutional review board (IRB) protects the people receiving the drug and ensures that the risks are reasonable given the potential benefits before approving the use of the therapy. You will be informed of the potential risks and will be required to give consent for treatment. It is important to note that significant unknown risks may exist.
The FDA authorizes over 99% of expanded access requests it receives. If the application is denied, FDA will notify the physician. The notification will be followed by a written letter that provides the reasons for FDA's denial of the request.
f approved, 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. Typically FDA responds to these requests in a matter of days (or hours for emergency requests). You must also receive IRB approval before treatment can begin unless it’s an emergency request.