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  1. Cellular & Gene Therapy Products

OTP Meetings Glossary

Biologics License Application (BLA) - BLA is a request for permission to introduce, or deliver for introduction, a biologic product into interstate commerce (21 CFR 601.2).

BLA supplement - A request to FDA to approve a change to an approved license application

BLA efficacy supplement - A supplement to an approved application proposing to make one or more related changes from among the following changes to product labeling: 1) Add or modify an indication or claim; 2) Revise the dose or dose regimen; 3) Provide for a new route of administration; 4) Make a comparative efficacy claim naming another drug product; 5) Significantly alter the intended patient population; 6) Incorporate other information based on at least one adequate and well-controlled clinical study.

Clinical Investigation or Clinical Study or Clinical Trial - An experiment in which a drug is administered, dispensed, or used involving one or more human subjects. For the purposes of this webpage, an experiment is any use of a drug except for the use of a marketed drug in the course of medical practice (21 CFR 312.3(b)).

Human Subject- An individual who participates in a clinical investigation, either as a recipient of an investigational new drug or as a control. A subject may be a healthy human or a patient with a disease or condition. (21 CFR 312.3(b)).

Investigational New Drug - A drug or biological product that is used in a clinical investigation. The term also includes a biological product that is used in vitro for diagnostic purposes (21 CFR 312.3(b)).

Investigational New Drug Application (IND) - A request from a sponsor to FDA seeking permission to use an unapproved drug or biological product for the purpose of clinical investigation or clinical treatment.

IND Amendment - Additional information submitted to an IND to clarify, revise, or modify previously submitted information.

Master file (MF) - For CBER’s purposes, master files (MFs) are voluntary submissions of information to the FDA that may be used to provide confidential, detailed information to the FDA about the facilities, processes, or articles used in the manufacture, processing, packaging, or storing of one or more regulated products. The person holding the MF (MF holder) may authorize one or more sponsors or applicants to reference information contained in the MF to support a submission to FDA without having to disclose that information to the sponsors or applicants. MF holders submit amendments to FDA when changes occur that might affect the product.

Prescription Drug User Fee Act (PDUFA) – The PDUFA was created by Congress in 1992 and authorizes FDA to collect fees from companies that produce certain human drug and biological products.

Rolling review - Submission of Portions of an Application (SoPA). If FDA determines, after preliminary evaluation of clinical data submitted by a sponsor, that a fast track / breakthrough therapy / regenerative medicine advanced therapy designated product may be effective, the Agency may consider reviewing portions of a marketing application before the sponsor submits the complete application.

Sponsor - A person who takes responsibility for and initiates a clinical investigation. The sponsor may be an individual or pharmaceutical company, governmental agency, academic institution, private organization, or other organization. The sponsor does not actually conduct the investigation unless the sponsor is a sponsor-investigator. A person other than an individual that uses one or more of its own employees to conduct an investigation that it has initiated is a sponsor, not a sponsor-investigator, and the employees are investigators (21 CFR 312.3(b))

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