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:
Is the drug safe and effective (efficacy) in its proposed use(s) when used as directed, and do the benefits of the drug outweigh the risks? Is the drug’s proposed labeling (package insert) appropriate, and what should it contain? Are the methods used in manufacturing the drug (good manufacturing practice [GMP]) and the controls used to maintain the drug’s quality adequate to preserve the drug’s identity, strength, quality, and purity?
Exceptions to this process include voter driven initiatives for "medical" marijuana in certain states.
1 Before trials 2 Clinical trials 3 The actual application 4 Requirements for similar products 5 See also 6 References 7 External links
To legally test the drug on human subjects in the U.S., the maker must
first obtain an
Investigational New Drug
Phase 1: The drug is tested in a few healthy volunteers to determine if it is acutely toxic. Phase 2: The drug is tested for both efficacy and safety in a larger sample pool. The majority of drugs fail in Phase 2 clinical trials due to the drug not being as effective as anticipated. Phase 3: The drug is typically tested in double-blind, placebo controlled trials to demonstrate that it works. Sponsors typically confer with FDA prior to starting these trials to determine what data is needed, since these trials often involve hundreds of patients and are very expensive. (Phase 4): These are post-approval trials that are sometimes a condition attached by the FDA to the approval.
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
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
The actual application
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
Once the application is submitted, the FDA has 60 days to conduct a
preliminary review, which assesses whether the NDA is "sufficiently
complete to permit a substantive review." If the FDA finds the NDA
insufficiently complete (reasons can vary from a simple administrative
mistake in the application to a requirement to re-conduct testing),
then the FDA rejects the application by sending the applicant a Refuse
Drug development Inverse benefit law Investigational New Drug, FDA application to start clinical trials Kefauver Harris Amendment, a 1962 amendment to the Federal Food, Drug, and Cosmetic Act (e.g. to also require evidence of efficacy) Regulation of therapeutic goods, rules in different countries.
^ FDA and Marijuana Feb 2017
^ Food, Drug, and Cosmetic Act, Section 505; 21 USC 355]
^ 21 CFR 201.5: Labeling Requirements for Prescription Drugs and/or
^ "Daily Med:Current Medication Information". Retrieved October 10,
^ Liberti L, McAuslane JN, Walker S (2011). "Standardizing the
Benefit-Risk Assessment of New Medicines: Practical Applications of
Frameworks for the Pharmaceutical Healthcare Professional". Pharm Med.
25 (3): 139–46. doi:10.1007/BF03256855.
^ Merck KGaA Receives Refuse To
Henninger, Daniel (2002). "Drug Lag". In
David R. Henderson