Experimental drug

An experimental drug is a medicinal product (a drug or vaccine) that has not yet received approval from governmental regulatory authorities for routine use in human or veterinary medicine. A medicinal product may be approved for use in one disease or condition but still be considered experimental for other diseases or conditions.

In the United States, the body responsible for approval is the U.S. Food and Drug Administration (FDA), which must grant the substance Investigational New Drug (IND) status before it can be tested in human clinical trials. IND status requires the drug's sponsor to submit an IND application that includes data from laboratory and animal testing for safety and efficacy.

In Canada a Clinical Trial Application (CTA) must be filed before starting a clinical trial. Health Canada will review the application and will notify the sponsor within 30 days if it is found to be deficient.

In Europe, the application process to perform a clinical trial takes place on a country-by-country basis. A sponsor must apply for approval in each country in which it intends to have study sites. A sponsor also must register their proposed clinical trial in EudraCT, a database of all in European clinical trials that is operated by the European Medicines Agency.