New drug application

The Food and Drug Administration's new drug application (NDA) is the vehicle in the United States through which drug sponsors formally propose that the FDA approve a new pharmaceutical for sale and marketing. The goals of the NDA are to provide enough information to permit FDA reviewers to establish the following:

Exceptions to this process include voter driven initiatives for "medical" marijuana in certain states.

To legally test the drug on human subjects in the U.S., the maker must first obtain an Investigational New Drug (IND) designation from FDA. This application is based on nonclinical data, typically from a combination of in vivo and in vitro laboratory safety studies, that shows the drug is safe enough to test in humans

Often the "new" drugs that are submitted for approval include new molecular entities or old medications that have been chemically modified to elicit differential pharmacological effects or reduced side-effects.

The legal requirement for approval is "substantial" evidence of effectiveness demonstrated through controlled clinical trials. This standard lies at the heart of the regulatory program for drugs. It means that the clinical experience of doctors, the opinion of experts, or testimonials from patients, even if they have experienced a miraculous recovery, have minimal weight in this process. Data for the submission must come from rigorous clinical trials.

The trials are typically conducted in three phases:

The legal requirements for safety and effectiveness have been interpreted as requiring scientific evidence that the benefits of a drug outweigh the risks and that adequate instructions exist for use, since many drugs are toxic and technically not "safe" in the usual sense.

Many approved medications for serious illnesses (e.g., cancer) have severe and even life-threatening side effects. Even relatively safe and well understood OTC drugs such as aspirin can be dangerous if used incorrectly.

The results of the testing program are codified in an FDA-approved public document that is called the product label, package insert or Full Prescribing Information. The prescribing information is widely available on the web, from the FDA, drug manufacturers, and frequently inserted into drug packages. The main purpose of a drug label is to provide healthcare providers with adequate information and directions for the safe use of the drug.

The documentation required in an NDA is supposed to tell the drug’s whole story, including what happened during clinical tests, what the ingredients of the drug formulation are, results of animal studies, how the drug behaves in the body, and how the company manufactures, processes and packages it. Currently, the FDA decision process lacks transparency, however, efforts are underway to standardize the benefit-risk assessment of new medicines. Once approval of an NDA is obtained, the new drug can be legally marketed starting that day in the U.S.

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