FDA regulation · 16 pages
FDA & drug/device regulation
Answer-first explainers of the FDA regulatory pathways and instruments — applications, clearances, designations and enforcement records — written as neutral process definitions, never clinical or treatment advice.
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All 16 fda & drug/device regulation pages
Biologics License Application (BLA)
A Biologics License Application (BLA) is the application a sponsor submits to the FDA to obtain a licence to market a biological product in the United States. It is filed under section 351 of the Public Health Service Act and must demonstrate the product is safe, pure and potent. The FDA, through CBER or CDER, approves it by issuing a biologics licence.
DefinitionNew Drug Application (NDA)
A New Drug Application (NDA) is the application a sponsor files with the FDA to obtain approval to market a new drug in the United States. It is submitted under section 505 of the Federal Food, Drug, and Cosmetic Act and compiles the full record of non-clinical and clinical studies, labelling and manufacturing information. The Center for Drug Evaluation and Research (CDER) reviews and approves it.
DefinitionInvestigational New Drug (IND)
An Investigational New Drug (IND) application is the submission a sponsor makes to the FDA to begin testing an unapproved drug in humans. Federal law generally prohibits shipping unapproved drugs across state lines, so an effective IND provides the exemption that permits clinical investigation. It contains animal pharmacology and toxicology data, manufacturing information and the clinical trial protocols.
Definition510(k) clearance
510(k) clearance is an FDA premarket notification in which a manufacturer demonstrates that a medical device is "substantially equivalent" to a legally marketed predicate device. Named after section 510(k) of the Federal Food, Drug, and Cosmetic Act, it is the most common pathway for Class II devices. The FDA "clears" the device rather than "approving" it.
DefinitionPremarket Approval (PMA)
Premarket Approval (PMA) is the FDA’s most stringent pathway for medical devices, required for high-risk Class III devices that support or sustain human life or present a potential unreasonable risk. Unlike 510(k) clearance, a PMA must contain valid scientific evidence — usually including clinical data — that independently establishes the device is safe and effective. The FDA "approves" a PMA.
DefinitionDe Novo classification
De Novo classification is an FDA pathway for novel medical devices of low to moderate risk that have no legally marketed predicate to support a 510(k). Rather than being automatically treated as high-risk Class III, the device is granted a new classification — usually Class I or II — with appropriate controls. A granted De Novo can then serve as a predicate for future 510(k) submissions.
DefinitionWarning Letters and Form FDA 483
A Form FDA 483 is the document an FDA investigator issues at the end of an inspection to list observations of conditions that may violate FDA-administered laws. An FDA Warning Letter is a separate, formal notice the agency sends when it identifies significant violations requiring prompt correction. The 483 is observational; the Warning Letter is a graded enforcement communication.
DefinitionOrange Book
The Orange Book is the FDA’s official list of approved drug products, formally titled "Approved Drug Products with Therapeutic Equivalence Evaluations". It identifies drugs approved as safe and effective, assigns therapeutic-equivalence (TE) codes indicating whether a generic can be substituted for a brand product, and records related patent and exclusivity information used in generic-drug approval.
DefinitionSupplemental NDA (sNDA)
A Supplemental NDA (sNDA) is an application to the FDA to make a change to a drug that already has an approved New Drug Application. Sponsors use it to add a new indication, change the formulation or dosage, modify the manufacturing process, or update the labelling. The FDA, through CDER, reviews the supplement against the original NDA, with the depth of review scaled to the change.
DefinitionDrug Supply Chain Security Act (DSCSA)
The Drug Supply Chain Security Act (DSCSA) is US legislation enacted in 2013, as Title II of the Drug Quality and Security Act, that builds an interoperable electronic system to identify and trace certain prescription drugs through the supply chain. It introduces product serialisation, transaction information exchange, verification and authorised-trading-partner requirements, phased in over a multi-year timeline.
DefinitionOrphan drug designation
Orphan Drug Designation is a status the FDA grants to a drug or biologic intended to treat a rare disease or condition — generally one affecting fewer than 200,000 people in the United States. Established by the Orphan Drug Act of 1983, the designation provides incentives such as tax credits, fee waivers and a period of marketing exclusivity to encourage development of treatments for rare diseases.
DefinitionBreakthrough therapy designation
Breakthrough Therapy Designation is an FDA programme to expedite the development and review of a drug that treats a serious or life-threatening condition where preliminary clinical evidence indicates it may offer substantial improvement over available therapies on a clinically significant endpoint. It provides intensive FDA guidance and organisational commitment, on top of Fast Track features, but is not itself an approval.
DefinitionFast track designation
Fast Track Designation is an FDA programme that facilitates the development and expedites the review of drugs treating serious or life-threatening conditions where there is an unmet medical need. It provides benefits such as more frequent FDA communication, eligibility for rolling review of an application, and potential eligibility for Accelerated Approval and Priority Review. It is a process designation, not an approval.
DefinitionAccelerated approval
Accelerated Approval is an FDA pathway that lets a drug for a serious or life-threatening condition with unmet need be approved on the basis of a surrogate endpoint, or an intermediate clinical endpoint, that is reasonably likely to predict clinical benefit. Sponsors must conduct confirmatory trials to verify the benefit, and the FDA can withdraw approval if those trials fail to confirm it.
Comparison510(k) clearance vs PMA
The key difference is the evidence standard. A 510(k) clearance shows a device is substantially equivalent to a legally marketed predicate, and the FDA "clears" it — typically for moderate-risk Class II devices. A PMA requires independent valid scientific evidence, usually including clinical data, that the device is safe and effective, and the FDA "approves" it — for high-risk Class III devices.
DefinitionFood and Drug Administration (FDA)
The Food and Drug Administration (FDA) is the US regulatory agency charged with ensuring the safety, efficacy, and security of human and veterinary drugs, biological products, medical devices, cosmetics, and the nation’s food supply. Organized into specialized centers like CDER, CBER, and CDRH, the FDA regulates clinical trials, reviews drug and device marketing applications (NDAs, BLAs, PMAs), and enforces compliance with Good Clinical Practice (GCP) and Good Manufacturing Practice (GMP).
