Course Glossary

Abbreviated New Drug Application (ANDA): Termed “abbreviated” because preclinical (animal) and clinical (human) data to establish safety and effectiveness are not required. Instead, generic applicants must scientifically demonstrate that their product is bioequivalent (i.e. performs in the same manner as the innovator drug).

Adverse Event: any undesirable experience associated with the use of a medical product in a patient

Advisory Committees: provide FDA with independent advice from outside experts on issues related to human and veterinary drugs, vaccines and other biological products, medical devices, and food

Approval Letter: a written communication to an applicant from FDA approving an application or an abbreviated application

Basic Science: seeks foundational knowledge to gain new insights into biology of disease

Biologics: A biologic is a medicine that is manufacture in a living system such as a microorganism or animal cells. Most biologics are very large, complex molecules and include proteins, blood products, vaccines, and cellular therapies.

Biologics License Application (BLA): formal presentation of data collected by drug sponsor supporting the safety and effectiveness of an investigational biologic agent

Biosimilar: a biological product that is highly similar to an FDA-approved biological product, known as a reference product, and has no clinically meaningful differences in terms of safety and effectiveness.

Blinding: process of keeping study participants, those involved with their management, and those collecting and analyzing clinical data unaware of the assigned treatment, so that they should not be influenced by that knowledge

Breakthrough Therapy Designation: FDA designates certain drugs as “breakthrough therapies” that show promising early clinical trial results and that are intended to treat serious diseases. The breakthrough designation is intended to speed the development of drugs that show the most promise in improving the treatment of the disease.

Clinical Endpoints: are outcomes that directly measures the therapeutic effect of a drug, such as how a patient feels, functions, or survives. An example is the incidence of stroke in a trial for cardiovascular disease drug

Complete Response Letter: a written communication to an applicant from FDA usually describing all of the deficiencies that the agency has identified in an application or abbreviated application that must be satisfactorily addressed before it can be approved

Endpoint: an event or outcome that can be measured objectively to determine whether the intervention being studied is beneficial. Some examples of endpoints are survival, improvements in quality of life, relief of symptoms, duration of response, and disappearance of the tumor.

Generic Drug: a drug that is comparable to an FDA-approved drug, known as a reference product, in dosage form, strength, route of administration, quality and performance characteristics, and intended use.

In Vitro Study: performed outside a living organism; typically, in a test tube or culture dish

Investigational New Drug Application (IND): a request for authorization from the FDA to administer an investigational treatment to humans

New Drug Application (NDA): formal presentation of data collected by drug sponsor supporting the safety and effectiveness of an investigational small molecule drug

Off-Label Drug Use: prescribing a medicine for an indication not specified on the drug label

Patient-Focused Drug Development Meetings: a series of disease-focused meetings held at FDA, featuring patients who provide their experience with a disease and their perspective on what new treatments ought to look like

Post-Marketing Commitments: studies FDA requires upon approval; may include: studies to assess drug response in specific subpopulations, evaluation of new doses, or further characterization of rare adverse events

Pre-Market Approval: system in which manufacturers of medical products are required to demonstrate the safety and effectiveness of a product before it can be sold to consumers

Prescription Drug User Fee Act (PDUFA): 1992 law that created “user fee” program, wherein the drug sponsor or applicant provides funds to hire additional FDA reviewers in return for assurances of timely reviews of new drug applications

Protocol: document that describes how a trial will be conducted, including the objective(s), design, methodology, statistical considerations and organization, and ensures the safety of the trial subjects and integrity of the data collected

Regulatory Science: science of developing new tools, standards, and approaches to assess the safety, effectiveness, quality, toxicity, public health impact, or performance of FDA regulated products.

Request for More Information: a letter sent to an applicant during an application review to request further information or clarification that is needed or would be helpful to allow completion of the review

Rolling Review: a drug sponsor can submit separate sections of its drug application for review by FDA, rather than waiting until every section of the application is completed. When rolling review is not in effect, review usually begins only when the drug sponsor has submitted the entire application to FDA.

Single Arm Trials: the test drug is not usually compared to a control; all the participants receive the experimental therapy

Sentinel Initiative: research tool to actively identify drug-induced adverse events from electronic healthcare data

Small molecule drugs: most common medicines are small molecule drugs, meaning they are typically manufactured through chemical synthesis, which means that they generally have well-defined chemical structures. By contrast, biologics are much more complex.

Supplemental New Drug Application (sNDA): after receiving FDA approval for a new drug, sponsors may seek approval for additional indication

Surrogate Endpoint: An endpoint reasonable likely to predict how a treatment impacts how patients feel, function, or survive. In cancer, tumor shrinkage might be a surrogate endpoint that can be measure before survival outcomes. a written communication to an applicant from FDA approving an application or an abbreviated application