FDA Glossary

FDA submission required for approval of biological products including vaccines, blood products, gene therapy, and other complex molecules derived from living organisms.

The formal submission to FDA requesting approval to market a new pharmaceutical drug. Contains all preclinical and clinical data demonstrating safety and efficacy.

An amendment to an existing NDA for changes like new indications, dosage forms, manufacturing changes, or new patient populations.

An amendment to an existing BLA, similar to an sNDA but for biological products.

Application for generic drug approval. Does not require clinical trials but must demonstrate bioequivalence to the reference drug.

Application submitted to FDA before a company can begin clinical trials in humans. Allows the drug to be shipped across state lines for testing.

FDA communication indicating that the review cycle is complete but the application is not ready for approval. Lists deficiencies that must be addressed.

FDA determination within 60 days of submission that an application is too incomplete to permit a substantive review. The clock stops until issues are resolved.

Historical FDA response (no longer used) indicating approval would be granted once specified conditions are met. Replaced by CRL in 2008.

Safety strategy required for certain drugs with serious risks. May include medication guides, patient registries, or prescriber certifications.

Law allowing FDA to collect fees from drug companies to fund the review process. Establishes target review timelines - the "PDUFA date" is the FDA's deadline to complete review.

Independent expert panel that reviews NDAs/BLAs and provides non-binding recommendations to FDA. Meetings are public and include a vote on approval.

Expedited FDA review (6 months vs standard 10 months) for drugs offering significant improvements in treatment, diagnosis, or prevention of serious conditions.

Default FDA review timeline of 10 months for new molecular entities or 6 months for applications not qualifying for priority review.

Allows companies with Fast Track designation to submit completed portions of their application for review before the entire submission is complete.

FDA designation for drugs treating serious conditions with unmet need. Enables more frequent FDA communication and rolling submission.

For drugs showing substantial improvement over existing treatments. Includes Fast Track features plus intensive FDA guidance on development.

Allows approval based on surrogate endpoints (like tumor shrinkage) rather than clinical outcomes (like survival). Requires post-marketing confirmatory trials.

Temporary FDA authorization during public health emergencies for products not yet fully approved. Used extensively during COVID-19.

Status for drugs treating rare diseases (<200,000 US patients). Provides tax credits, fee waivers, and 7 years market exclusivity upon approval.

First-in-human studies, typically 20-100 healthy volunteers. Primary goal is assessing safety, dosing, and identifying side effects.

Studies in 100-300 patients with the target disease. Evaluates efficacy and further assesses safety. Often determines optimal dosing.

Large-scale studies (1,000-3,000+ patients) providing definitive evidence of efficacy and safety. Required for NDA/BLA submission.

Post-marketing studies conducted after approval. May be required by FDA or voluntary. Monitors long-term safety and real-world effectiveness.

A drug containing an active ingredient that has never been approved by FDA. Distinct from new formulations or combinations of existing drugs.

The main outcome measure used to evaluate a drug's effectiveness in a clinical trial. Statistical significance here typically required for approval.

A measurable marker (like blood pressure or tumor size) used as a substitute for clinical outcomes (like heart attack or death). Enables faster trials.

Analysis method including all randomized patients regardless of whether they completed treatment. Preserves randomization and reflects real-world effectiveness.