Expanded Access

In certain situations seriously ill patients can be allowed access to drugs that have not yet been cleared through the U.S. Food and Drug Administration approval process. This is done by means of a program known as “expanded access to investigational drugs.”

Overview

Expanded access, also referred to as “compassionate use,” is the use of an investigational drug outside of a clinical trial. Expanded access allows patients with serious or life-threatening diseases and illnesses access to these investigational or experimental drugs if they have no other acceptable medical options. This type of treatment should not be considered standard. The drugs offered through expanded access are currently under testing, meaning they could offer therapeutic benefits, or they could be ineffectual. Other things to consider are unknown side effects, ranging from mild to severe or life-threatening. Expanded access is not approved under every circumstance and depends on the following factors:
  • Does the patient have a serious or life-threatening disease and no treatment options?
  • What possible side effects could be associated with the investigational drug and how much is known concerning safe use and dosage?
  • What is the patient’s medical history, including any complications and other medicines, as well as the stage of the illness and their overall health.
  • Is the drug’s manufacturer willing to make the drug for treatment purposes?
  • Could expanded access interfere with the drug’s development?

Expanded Access Categories

Expanded access also consists of different levels which are based on the number of patients treated and what knowledge has already been gained about the drug.
  • Individual patient access is where a single patient is granted accessibility to the investigational drug.
  • Intermediate-size group access can be anywhere between a single patient up to a large group.
  • Hundreds to thousands of patients may receive access to these drugs through large scale programs sometimes referred to as a “treatment IND” or “treatment protocol.”
These categories vary depending on the number of patients, as the more patients become involved, the more evidence required concerning the drug’s safety and potential efficacy when treating a disease.

Expanded Access Protection

Patient interested in an expanded access program must complete an informed consent application process. Through these steps patients must sign an informed consent document, acknowledging both known and unknown risks, and their understanding that the drug has yet to be proven safe and effective. At this time the expanded access protocol must be reviewed by an Institutional Review Board (IRB). This is a special committee responsible for ensuring patient rights and safety; and that the patient is aware of the potential risks associated with using an experimental drug.

How Can Patients Locate Expanded Access Programs?

Patients interested in expanded access to a particular drug should first consult with their doctor. Most programs available can be found via an internet search of clinicaltrials.gov. Another option is to contact the company studying the drug directly to inquire about availability outside of clinical trials. If a patient is attempting to gain access to an unlisted drug their doctor must contact both the drug’s manufacturer and the FDA as part of the approval process.

Who are these Programs For?

Expanded access programs were developed for patients who have exhausted all other options for treatment of their disease. The program is not approved for everyone and patients should consult their doctor or other licensed specialist concerning further information and applications. Reference:
U.S. Food and Drug Administration