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Biomarker: The measure of something in the patient, such as blood levels of VEGF or LDH, that can indicates drug activity or clinical benefit.
CBER: Center for Biologics Evaluation and Research- FDA agency whose job it is to ensure that drugs are safe and effective.
CDER: Center for Drug Evaluation and Research- FDA agency whose job it is to ensure that drugs are safe and effective.
Controlled study: A clinical trial that compares the outcome of a group of randomly assigned patients who receive the experimental treatment to the outcome of a group of randomly assigned patients who receive a standard treatment or an inactive placebo dose.
Dose-finding study: a study, usually a phase I study, that compares the action of varying amounts of the experimental treatment to determine the optimum dose
Efficacy: evidence that a drug is effective by indications of clinical benefit or surrogate markers.
Endpoints: A variable (such as time to progression) used to judge the effectiveness of a treatment
Fast-track: This FDA section mandates the Agency to facilitate the development and expedite review of drugs and biologics intended to treat serious or life-threatening conditions and that demonstrate the potential to address unmet medical needs
IND: Investigational New Drug - an application to the FDA by the sponsor of a drug. IND applications must contain Animal Pharmacology and Toxicology Studies Manufacturing Information, Clinical Protocols and Investigator Information. INDs also refer to the emergency use of an investigational drug or biologic by an individual.
NDA: New Drug Application
Phase I trial: small studies that try to identify the best ways to administer a therapy and identify dose tolerances
Phase II trial: larger studies that test for efficacy of a treatment, usually at various doses.
Phase III trial: Studies designed to test the efficacy of a new intervention, usually against standard therapies; often randomized to minimize selection bias.
PDUFA: Prescription Drug User Fee Act of 1992 - agreement to incorporate tough performance deadlines fo decisions on NDA in exchange for fees from drug sponsors?
Quality of life: End points that may be used to determine the impact of a treatment on the patient that are not directly related to clinical benefit, such as fatigue, pain, ability to go to work and function normally, etc.
Randomized studies: compare outcomes of similar patients recruited and randomly assigned to receive one of two interventions.
Surrogate endpoint: An endpoint that is used to infer clinical benefit (survival advantage), such as tumor regression or time to progression.
Survival benefit: Considered to be the ultimate endpoint that proves clinical benefit. In some cases the meaning of this endpoint is confounded when patients crossover to other treatments.
Time to progression: A surrogate endpoint used to infer clinical benefit. It's the measure of the amount of time the disease stays dormant or stable.
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