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The U.S. Food and Drug Administration is responsible for protecting the public health by, among other things, ensuring the safety and effectiveness of drugs, biological products and medical devices. In certain circumstances, the FDA allows the emergency use of medical products that do not yet have agency approval.

The language used to describe potential treatments can be confusing. Here’s what some of those terms mean.

What ‘FDA Approved’ Means

American consumers depend on the FDA to provide independent scientific reviews of medical products, including therapeutic drugs and vaccines. Before the FDA can approve a medicine, such as a drug or biological product, the agency must determine whether the clinical data and other scientific information show that:

The medicine is safe and effective for its intended use. For example, to prevent or treat a certain disease.
The product can be made according to federal quality standards.

When the FDA approves a drug, it means the agency has determined that:

The drug is safe and effective for its intended use.
The benefits of the drug outweigh its known and potential risks when used according to its approved labeling.

The FDA works with manufacturers and researchers to make sure the agency has the information needed to complete drug evaluations as quickly as possible.

Investigational Treatments

Scientists conduct clinical trials to study investigational drugs to see whether they can safely and effectively prevent or treat a specific disease or condition. As part of those clinical trials, they might try to discover:

How the drug might be used for that disease or condition.
If the drug is safe for people.
How much of the drug is needed.
Information about whether the drug works against the disease.
The potential benefits and risks of taking the drug.

Expanded Access

Expanded access is a possible way for a patient with a serious or immediately life-threatening disease or condition to gain access to an investigational medical product outside of clinical trials for the purposes of treatment when there are no comparable or satisfactory alternative therapies available. These products may include drugs, biological products and medical devices. Expanded access is sometimes called “compassionate use.”

The FDA may authorize treatment with an investigational medical product under expanded access if all the criteria and requirements for expanded access in the FDA’s regulations are met.

Emergency Use Authorization (EUA)

An emergency use authorization (EUA) is one of several tools the FDA can use to help make certain medical products available quickly in certain emergencies or potential emergencies to diagnose, treat or prevent a serious or life-threatening disease or condition caused by biological, chemical, radiological or nuclear agents.

Only certain types of emergency determinations enable the FDA to issue EUAs. During such times, the FDA can issue an EUA to authorize the use of unapproved medical products or unapproved uses of approved medical products when, among other things, there are no adequate, approved and available alternatives.

The evidence of effectiveness required for issuing an EUA is different than an FDA approval. Under an EUA, the FDA authorizes uses of medical products based on a reasonable belief that the product may be effective for the intended use based on the totality of the evidence available at the time, which might not include all the information that would be needed for an FDA approval.

When evaluating whether an EUA is appropriate, the FDA carefully balances the known and potential risks and benefits of the products based on the information currently available. EUAs terminate when the emergency declaration ends and can also be revised or revoked by the FDA at any time as the agency continues to evaluate the available information.

The FDA’s EUA page has information about products currently authorized under an EUA, as well as product Fact Sheets and additional information for health care professionals and patients.

‘Off-Label’ Use: Unapproved Uses of Approved Drugs

Once the FDA has approved a drug for a disease or medical condition, health care professionals generally may prescribe or administer the drug in clinical practice for an unapproved use (also called “off-label” use) based on their medical judgment, recognizing that the FDA has not approved the drug for such use.