|
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.
|
