Expanded Access
A program that provides an investigational drug to a group of patients with serious conditions outside of a clinical trial, often while the drug is being reviewed for approval.
In Detail
Expanded access is an FDA-regulated program that allows groups of patients with serious or life-threatening conditions to receive investigational drugs, biologics, or medical devices outside of clinical trials when no satisfactory alternatives exist. Unlike compassionate use (which typically involves individual patients), expanded access programs can serve intermediate-sized patient populations or large groups through treatment protocols, treatment INDs, or treatment protocols. These programs are most commonly established when a drug has shown promising results in Phase 2 or Phase 3 trials and the sponsor is preparing or has already submitted a marketing application to the FDA. Expanded access serves a dual purpose: it provides treatment to patients who need it while also generating additional safety data that can support the drug's approval. The FDA has three categories of expanded access: individual patient access (for a single patient), intermediate-size patient populations (typically a few dozen to a few hundred patients), and widespread treatment use (for larger populations, often when the drug is close to approval). Drug manufacturers are not required to provide expanded access programs, and some have been criticized for declining requests. Expanded access programs must have IRB approval and informed consent procedures, just like clinical trials. Participants in expanded access programs may be asked to pay for the cost of the drug or treatment, though some sponsors provide the drug at no charge. For patients considering expanded access, it is important to understand that the treatment is still investigational, may have unknown risks, and insurance coverage varies.
Frequently Asked Questions
What does "Expanded Access" mean in clinical trials?
A program that provides an investigational drug to a group of patients with serious conditions outside of a clinical trial, often while the drug is being reviewed for approval.
Why is "expanded access" important for patients?
Understanding expanded access helps patients and caregivers navigate clinical trial participation with confidence. It is part of the broader clinical research process that ensures treatments are safe and effective before reaching patients.
Related Terms
Compassionate Use
A pathway that allows seriously ill patients to access experimental treatments outside of clinical trials when no other options exist.
FDA Approval Process
The regulatory pathway through which the U.S. Food and Drug Administration evaluates and approves new drugs, biologics, and medical devices for public use.
Clinical Trial
A research study that tests a medical treatment, drug, device, or intervention in human volunteers to determine whether it is safe and effective.
Investigational New Drug (IND)
An application submitted to the FDA before a new drug can be tested in humans, containing preclinical data demonstrating safety and a plan for clinical trials.