U.S. flag An official website of the United States government
  1. Home
  2. Drugs
  3. Guidance, Compliance, & Regulatory Information
  4. Enforcement Activities | FDA
  5. Unapproved Drugs
  1. Enforcement Activities | FDA

Unapproved Drugs

Unapproved prescription drugs pose significant risks to patients because they have not been reviewed by FDA for safety, effectiveness or quality. Without FDA review, there is no way to know if these drugs are safe and effective for their intended use, whether they are manufactured in a way that ensures consistent drug quality or whether their label is complete and accurate. Unapproved drugs have resulted in patient harm, and the agency works to protect patients from the risks posed by these drugs.

Preserving Patient Access to Medically Necessary Drugs

The agency balances its goal to eliminate unapproved prescription drugs from the market with patient access to medically necessary drugs. FDA carefully considers the possible effects on patient access, including whether any action would likely lead to a disruption in the drug supply, before initiating an action against an unapproved drug.

The agency permits some unapproved drugs to be marketed if they are relied on by health care professionals to treat serious medical conditions when there is no FDA-approved drug to treat the condition or there is insufficient supply of FDA-approved drugs.

FDA Approval is Required by Law

Federal law requires all new prescription drugs in the U.S. be shown to be safe and effective for their intended use prior to marketing. However, some drugs are available in the U.S. even though they have never received the required FDA approval. Many health care professionals and patients are unaware that some of the drugs prescribed are not FDA approved.

FDA permits some unapproved prescription drugs to be marketed if:  

  • the drug is subject to an open drug efficacy study implementation (DESI) program proceeding,
  • health care professionals rely on the drug to treat serious medical conditions when there is no FDA-approved drug to treat the condition,
  • there is insufficient supply of an FDA-approved drug.

The law allows some unapproved prescription drugs to be lawfully marketed if they meet the criteria of generally recognized as safe and effective (GRASE) or grandfathered. However, the agency is not aware of any human prescription drug that is lawfully marketed as grandfathered.

Unapproved Drugs Program

The agency’s Unapproved Drugs Program has a two-prong approach to help assure patient safety. First, the agency encourages manufacturers of unapproved drugs to obtain approval to be legally marketed in the U.S. Second, FDA has worked to remove unapproved drugs from the market. Many potentially unsafe drugs have been removed from the market since 2006, including several drugs with significant safety concerns. The agency uses a risk-based approach, giving enforcement priority to drugs that pose the highest risk to public health, without imposing undue burden on patients or unnecessarily disrupting the availability of drugs on the market.

In 2011, FDA issued a final guidance, Guidance for FDA Staff and Industry, Marketed Unapproved Drugs – Compliance Policy Guide, Section 440.100: Marketed New Drugs Without Approved NDAs or ANDAs, which describes FDA’s enforcement priorities.

Search Marketed Drugs Listed with FDA, Including Unapproved Drugs

Drugs marketed in the U.S., with or without FDA approval, can be identified in the following databases:

  • National Drug Code (NDC) Directory publishes data derived from information submitted to the agency as part of drug listing requirements, including information on unapproved drugs. NDC numbers are provided for all listed drugs, regardless of approval status. Information in the directory does not indicate that FDA has verified the information provided. 
  • Drugs@FDA lists information on FDA-approved drugs since 1998, including patient information, labels and approval letters.
  • Orange Book identifies FDA approved drugs.

Unapproved Drugs and Drug Prices

There are many factors that contribute to drug pricing. When there is a sole source of an FDA-approved drug, market dynamics may enable the company that sought approval to set a higher price than when the drug faces competition. Patients and health care professionals can, however, have confidence that the FDA-approved version has been shown to be safe and effective for its intended use and that it is manufactured according to federal quality standards. 

While FDA does not have the authority to regulate drug prices, it is keenly aware of price fluctuations that can occur on the heels of its regulatory actions and takes steps within its authority to minimize the duration, if not the extent, of those price hikes. Although following the FDA approval process may result in cost increases for a drug over the short term, the risks to the individal patient are substantally reduced and the benefits are assured for the long term.

For More Information